299. 嚢胞性線維症
[臨床試験数:1,592,薬物数:1,539(DrugBank:255),標的遺伝子数:81,標的パスウェイ数:162

Searched query = "Cystic fibrosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03435939
(ClinicalTrials.gov)
February 1, 20215/2/2018Effect of Losartan in Cystic Fibrosis (CF)-NIH Grant #133240Anti-Inflammatory Therapy to Augment CFTR Rescue in CF PatientsCystic FibrosisDrug: LosartanUniversity of Kansas Medical CenterNULLNot yet recruiting18 YearsN/AAll16Early Phase 1United States
2NCT03931252
(ClinicalTrials.gov)
February 202125/4/2019Thermic Effect of Feeding in Cystic FibrosisThermic Effect of Feeding in Cystic FibrosisCystic FibrosisDietary Supplement: Boost VHC;Dietary Supplement: Ensure High ProteinVirginia Commonwealth UniversityNULLEnrolling by invitation18 YearsN/AAll10N/AUnited States
3NCT04656275
(ClinicalTrials.gov)
January 25, 20211/12/2020A Study in Patients With Non-cystic Fibrosis Bronchiectasis to Test How Well Different Doses of BI 1323495 Are Tolerated and How BI 1323495 Affects Biomarkers of InflammationSafety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Different Oral Doses of BI 1323495 Bid Versus Placebo in Patients With Non-cystic Fibrosis Bronchiectasis (Randomised, Double-blind, Placebo-controlled, Parallel Group Trial)Non-cystic Fibrosis BronchiectasisDrug: BI 1323495;Drug: PlaceboBoehringer IngelheimNULLNot yet recruiting18 Years80 YearsAll36Phase 1NULL
4NCT04580368
(ClinicalTrials.gov)
January 4, 20212/10/2020Testing Drug Efficacy in Cystic Fibrosis Through N-of-1 TrialsTesting Drug Efficacy in Cystic Fibrosis Through N-of-1 TrialsCystic FibrosisDrug: CFTR ModulatorsChildren's Hospital Medical Center, CincinnatiNULLNot yet recruiting6 YearsN/AAll50N/ANULL
5NCT04599465
(ClinicalTrials.gov)
January 202121/10/2020A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF)A Phase 3b Open-label Study to Assess the Effect of Elexacaftor/Tezacaftor/Ivacaftor on Glucose Tolerance in Cystic Fibrosis Subjects With Abnormal Glucose MetabolismCystic FibrosisDrug: ELX/TEZ/IVA;Drug: IVAVertex Pharmaceuticals IncorporatedNULLNot yet recruiting12 YearsN/AAll60Phase 3NULL
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT04545515
(ClinicalTrials.gov)
January 20213/9/2020A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Subjects 6 Years and Older and F/MF GenotypesA Phase 3b Open-Label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects Ages 6 Years and Older Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)Cystic FibrosisDrug: ELX/TEZ/IVA;Drug: IVAVertex Pharmaceuticals IncorporatedNULLNot yet recruiting6 YearsN/AAll108Phase 3NULL
7NCT04315311
(ClinicalTrials.gov)
December 31, 202018/3/2020Study Of Effects Of Oral CREON Capsules In Adult Participants With Exocrine Pancreatic Insufficiency Not Due To Cystic Fibrosis, Chronic Pancreatitis, Pancreatectomy, Or Pancreatic CancerA Phase 4 Open-Label Single-Arm Study To Evaluate The Use Of CREON In Subjects With EPI Due To Etiologies Other Than Cystic Fibrosis, Chronic Pancreatitis, Pancreatectomy, Or Pancreatic CancerExocrine Pancreatic Insufficiency (EPI)Drug: CREONAbbVieNULLNot yet recruiting18 YearsN/AAll50Phase 4United States
8NCT04322929
(ClinicalTrials.gov)
December 30, 202024/3/2020Roflumilast in Non-CF Bronchiectasis Study (2019)Anti-inflammatory Effects of Roflumilast Treatment for 12 Weeks in Stable-state Non-cystic Fibrosis BronchiectasisNon-cystic Fibrosis BronchiectasisDrug: Roflumilast Oral TabletThe University of Hong KongNULLNot yet recruiting18 YearsN/AAll27Phase 2Hong Kong
9EUCTR2019-002840-26-GB
(EUCTR)
22/12/202003/09/2020Study of safety, tolerability, pharmacokinetics and pharmacodynamics of QBW251 in subjects with bronchiectasisA randomized, subject- and investigator-blinded, placebo-controlled, parallel group study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of QBW251 in patients with bronchiectasis Bronchiectasis
MedDRA version: 23.0;Level: LLT;Classification code 10083611;Term: Non-cystic fibrosis bronchiectasis;System Organ Class: 100000004855;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Code: QBW251
INN or Proposed INN: Icenticaftor
Other descriptive name: CFTR potentiator
Novartis Pharma AGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
72Phase 2Spain;Germany;China;United Kingdom
10EUCTR2020-003170-44-CZ
(EUCTR)
03/12/202022/10/2020A study Evaluating the effect of Elexacaftor/Tezacaftor/Ivacaftor on Glucose Tolerance in Cystic Fibrosis Subjects with Abnormal Glucose MetabolismA Phase 3b Open-label Study to Assess the Effect of Elexacaftor/Tezacaftor/Ivacaftor on Glucose Tolerance in Cystic Fibrosis Subjects with Abnormal Glucose Metabolism - A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Subjects With Cystic Fibrosis Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: ELX (VX-445)/TEZ (VX-661)/IVA (VX-770)
INN or Proposed INN: Elexacaftor
INN or Proposed INN: Tezacaftor
INN or Proposed INN: IVACAFTOR
Trade Name: Ivacaftor
Product Name: 150mg IVACAFTOR
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 3France;Czech Republic;Spain;Belgium;Australia;Netherlands;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2016-004558-13-DE
(EUCTR)
01/12/202009/04/2020A double-blind, placebo controlled, multicentre, clinical trial to investigate the efficacy and safety of 12 months of therapy with inhaled colistimethate sodium in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa)A double-blind, placebo controlled, multicentre, clinical trial to investigate the efficacy and safety of 12 months of therapy with inhaled colistimethate sodium in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa) - PROMIS II Non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa
MedDRA version: 21.0;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]
Trade Name: Promixin, 1 million International Units (IU) Powder for Nebuliser Solution
Product Name: Colistimethate sodium
INN or Proposed INN: COLISTIMETHATE SODIUM
Other descriptive name: Colistimethate Sodium
Zambon S.p.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
420Phase 3Portugal;United States;France;Greece;Canada;Argentina;Poland;Germany
12NCT03698448
(ClinicalTrials.gov)
December 1, 20203/10/2018A Dose-finding Study of Inhaled OligoG vs Placebo in Patients With Cystic FibrosisA Randomized, Double Blind, Dose-finding Study of Inhaled Oligosaccharide (OligoG) vs Placebo in Patients With Cystic FibrosisCystic FibrosisDrug: OligoG Dry powder for inhalationAlgiPharma ASImperial College London;Cystic Fibrosis Foundation;European Cystic Fibrosis Society;University Hospital of Cologne;Cystic Fibrosis EuropeNot yet recruiting12 YearsN/AAll120Phase 2;Phase 3Germany
13NCT04533646
(ClinicalTrials.gov)
December 1, 202023/8/2020Comparison of Meal-Time Dosing of Insulin in Cystic Fibrosis Related DiabetesComparison of Meal-Time Dosing of Rapid Acting Insulin Using Carbohydrate Counting vs. Fixed Doses Utilizing Continuous Glucose Monitoring In Patients With Cystic Fibrosis Related DiabetesCystic Fibrosis-related DiabetesDrug: Insulin;Device: Continuous glucose monitor (CGM)Jagdeesh UllalWake Forest University Health SciencesNot yet recruiting18 Years80 YearsAll20Phase 4NULL
14NCT04594369
(ClinicalTrials.gov)
December 1, 202014/10/2020A Study to Assess the Efficacy, Safety, and Tolerability of Brensocatib in Participants With Non-Cystic Fibrosis BronchiectasisA Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of Brensocatib Administered Once Daily for 52 Weeks in Subjects With Non-Cystic Fibrosis Bronchiectasis - The ASPEN StudyNon-Cystic Fibrosis BronchiectasisDrug: Brensocatib 10 mg;Drug: Brensocatib 25 mg;Drug: PlaceboInsmed IncorporatedNULLRecruiting18 Years85 YearsAll1620Phase 3United States
15NCT02547116
(ClinicalTrials.gov)
December 20209/9/2015Epidemiology and Treatment of Small-colony Variant Staphylococcus Aureus in Cystic FibrosisEpidemiology and Treatment of Small-colony Variant Staphylococcus Aureus in Cystic FibrosisCystic Fibrosis;MRSADrug: RifampinJohns Hopkins UniversityNULLSuspended12 YearsN/AAll16Phase 4United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT04596319
(ClinicalTrials.gov)
December 202015/10/2020Ph 1/2 Study Evaluating Safety and Tolerability of Inhaled AP-PA02 in Subjects With Chronic Pseudomonas Aeruginosa Lung Infections and Cystic FibrosisA Phase 1b/2a, Multi-Center, Double-Blind, Randomized, Placebo-Controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of AP-PA02 Multi-Phage Therapeutic Candidate for Inhalation in Subjects With Cystic Fibrosis and Chronic Pulmonary Pseudomonas Aeruginosa (Pa) InfectionCystic Fibrosis;Pseudomonas Aeruginosa;Pseudomonas;Lung Infection;Lung Infection PseudomonalBiological: AP-PA02;Other: PlaceboArmata Pharmaceuticals, Inc.Cystic Fibrosis FoundationRecruiting18 YearsN/AAll48Phase 1;Phase 2United States
17NCT03058068
(ClinicalTrials.gov)
December 20206/2/2017Human MesenchymAl Stem Cells Infusion in Patients With Cystic FibrosisA Phase I, Randomized and Placebo-controlled Trial to Evaluate the Safety, Tolerability, and Potential Efficacy of Allogeneic Human MesenchymAl Stem Cells Infusion in Patients With Cystic Fibrosis - HAPICystic FibrosisBiological: Allo-hMSCs;Biological: PlaceboJoshua M HareNULLWithdrawn20 Years45 YearsAll0Phase 1United States
18NCT04643587
(ClinicalTrials.gov)
December 202019/11/2020Study to Assess CSL787 in Non-cystic Fibrosis Bronchiectasis (NCFB)A Phase 1, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Exploratory Efficacy of Nebulized CSL787 in Healthy Subjects and Subjects With Non-Cystic Fibrosis Bronchiectasis (NCFB)Noncystic Fibrosis Bronchiectasis (NCFB)Biological: CSL787;Drug: PlaceboCSL BehringNULLNot yet recruiting18 YearsN/AAll64Phase 1NULL
19NCT03921060
(ClinicalTrials.gov)
December 202026/3/2019Markers of Osteoporosis in Cystic FibrosisMarkers of Osteoporosis in Cystic FibrosisCystic FibrosisDrug: DenosumabUniversity of Texas Southwestern Medical CenterNULLNot yet recruiting12 Years64 YearsAll100Phase 4United States
20NCT04509050
(ClinicalTrials.gov)
November 18, 20206/8/2020Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young ChildrenA Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)Cystic FibrosisDrug: Ivacaftor or elexacaftor/tezacaftor/ivacaftorSonya HeltsheCystic Fibrosis Foundation;University of Washington;University of Alabama at BirminghamRecruitingN/A5 YearsAll210United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT04624490
(ClinicalTrials.gov)
November 2, 202031/10/2020Hyperpolarized 129Xe MR Imaging of Lung Function in Healthy Volunteers and Subjects With Pulmonary DiseaseHyperpolarized 129Xe MR Imaging of Lung Function in Healthy Volunteers and Subjects With Pulmonary DiseaseAsthma;COPD;Interstitial Lung Disease;Cystic Fibrosis;Pulmonary Hypertension;Pulmonary Infection;Other Lung DiseaseDrug: Hyperpolarized Xe129Mario Castro, MD, MPHNULLNot yet recruiting18 YearsN/AAll260Phase 1NULL
22NCT04537793
(ClinicalTrials.gov)
November 202028/8/2020Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 YearsA Phase 3 Study Evaluating the Safety, Tolerability, and Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Through 5 Years of AgeCystic FibrosisDrug: ELX/TEZ/IVA;Drug: IVAVertex Pharmaceuticals IncorporatedNULLNot yet recruiting2 Years5 YearsAll84Phase 3NULL
23NCT04579939
(ClinicalTrials.gov)
November 202029/9/2020Oral Glucose Tolerance Testing Using Candy for Cystic FibrosisOral Glucose Tolerance Testing Using Candy: A Sweet Solution to Improve Screening Compliance in Cystic FibrosisCystic FibrosisDiagnostic Test: Candy Glucose Tolerance TestMayo ClinicNULLNot yet recruiting10 Years21 YearsAll10N/AUnited States
24EUCTR2019-003554-86-FR
(EUCTR)
29/10/202002/04/2020A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 through 11 Years Old With Cystic Fibrosis and F/MF genotypesA Phase 3b, Randomized, Placebo-Controlled Study Evaluating the Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 6 Through 11 Years of Age Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 50 mg ELX/25 mg TEZ/37.5 mg IVA fixed dose combination
Product Code: VX-445 / TEZ / IVA
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: elexacaftor
Other descriptive name: ELX
INN or Proposed INN: Tezacaftor
Other descriptive name: TEZ
Product Name: 150-mg IVACAFTOR
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Product Name: 100 mg ELX/50 mg TEZ/75 mg IVA fixed dose combination
Product Code: VX-445 / TEZ / IVA
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: elexacaftor
Other descriptive name: ELX
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
108Phase 3France;Canada;Spain;Denmark;Australia;Israel;Netherlands;Germany;United Kingdom;Switzerland
25EUCTR2019-004511-31-IT
(EUCTR)
21/10/202007/10/2020Prospective validation and clinical evaluation of a new posaconazole dosing regimen for children and adolescents with cystic fibrosis and Aspergillus infection.Prospective validation and clinical evaluation of a new posaconazole dosing regimen for children and adolescents with cystic fibrosis and Aspergillus infection. - cASPerCF Cystic Fibrosis and Aspergillus infection.
MedDRA version: 20.0;Level: LLT;Classification code 10003488;Term: Aspergillosis;System Organ Class: 100000004862;Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]
Trade Name: NOXAFIL - 40 MG/ML SOSPENSIONE ORALE USO ORALE FLACONE (VETRO) 105 ML 1 FLACONE
Product Name: Noxafil
Product Code: [NA]
INN or Proposed INN: Posaconazolo
Trade Name: Noxafil
Product Name: Noxafil
Product Code: [NA]
INN or Proposed INN: POSACONAZOLO
Trade Name: Noxafil
Product Name: Noxafil
Product Code: [NA]
INN or Proposed INN: POSACONAZOLO
Product Name: Noxafil
Product Code: [na]
INN or Proposed INN: POSACONAZOLO
Trade Name: NOXAFIL - 40 MG/ML SOSPENSIONE ORALE USO ORALE FLACONE (VETRO) 105 ML 1 FLACONE
Product Name: Noxafil
Product Code: [na]
INN or Proposed INN: POSACONAZOLO
Trade Name: Noxafil
Product Name: Noxafil
Product Code: [na]
INN or Proposed INN: Posaconazolo
IRCCS, OSPEDALE PEDIATRICO BAMBINO GESÙ DI ROMANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
135Phase 2;Phase 3Portugal;France;Czech Republic;Greece;Spain;Ireland;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26EUCTR2020-001404-42-GB
(EUCTR)
12/10/202025/08/2020A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Subjects 6 Years and Older and F/MF genotypesA Phase 3b Open label Study Evaluating the Long term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects Ages 6 Years and Older Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) - Phase 3b Open-label Study of ELX/TEZ/IVA in subjects 6 years & older Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 50 mg ELX/25 mg TEZ/37.5 mg IVA fixed dose combination
Product Code: VX-445 / TEZ / IVA
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: Tezacaftor
Other descriptive name: TEZ
INN or Proposed INN: Elexacaftor
Other descriptive name: ELX
Product Name: 150mg IVACAFTOR
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Product Name: 100 mg ELX/50 mg TEZ/75 mg IVA fixed dose combination
Product Code: VX-445 / TEZ / IVA
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: Tezacaftor
Other descriptive name: TEZ
INN or Proposed INN: Elexacaftor
Other descriptive name: ELX
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
108Phase 3Israel;Netherlands;Germany;Switzerland;United Kingdom;France;Canada;Spain;Denmark;Australia
27NCT04467957
(ClinicalTrials.gov)
October 1, 202019/6/2020Non-contrast Lung Perfusion Mapping Applied for New Insights in Cystic FibrosisNon-contrast Lung Perfusion Mapping Applied for New Insights in Cystic FibrosisCystic FibrosisDrug: Initiation of CFTR Modulator;Drug: Hyperpolarized Xenon 129Children's Hospital Medical Center, CincinnatiNULLNot yet recruiting6 Years21 YearsAll26Phase 4NULL
28NCT03925194
(ClinicalTrials.gov)
October 1, 202019/4/2019A Study to Evaluate Safety and Efficacy of Subcutaneous Administration of Anakinra in Patients With CFA Phase IIa, Randomized, Placebo-controlled, Double-blind, Cross-over Study to Evaluate Safety and Efficacy of Subcutaneous Administration of Anakinra in Patients With Cystic FibrosisCystic Fibrosis, 10011762Drug: AnakinraHeidelberg UniversityNULLNot yet recruiting12 YearsN/AAll52Phase 2Germany
29NCT04658277
(ClinicalTrials.gov)
October 1, 202031/8/2020Effect of Long Term Clarithromycin for Prevention of Exacerbations in Non-cystic Fibrosis Bronchiectasis in Asian PopulationsEffect of Long Term Clarithromycin for Prevention of Exacerbations in Non-cystic Fibrosis Bronchiectasis in Asian PopulationsBronchiectasis AdultDrug: Clarithromycin;Drug: PlaceboChinese University of Hong KongNULLRecruiting50 YearsN/AAll136N/AHong Kong
30EUCTR2020-001762-11-GB
(EUCTR)
17/09/202023/07/2020A research study to collect long term safety information from subjects who have received study treatment from a qualifying lenabasum Corbus trial.An Observational Long-Term Safety Surveillance of Participants from Corbus Sponsored Lenabasum Pivotal Clinical Trials Dermatomyositis (DM) is a rare and serious autoimmune disease.An overactive immune response causes chronic inflammation,which results in growth of scar tissue in the skin,muscles,and many internal organs.Cystic Fibrosis (CF) is a genetic disorder which results in thick mucus formation on the airways leading to increased lung infections,fibrosis of the lungs and digestive tract and abnormal immune function
MedDRA version: 20.0;Level: PT;Classification code 10012503;Term: Dermatomyositis;System Organ Class: 10040785 - Skin and subcutaneous tissue disorders
MedDRA version: 20.0;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Not possible to specify
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
100Phase 2;Phase 3United States;Serbia;Portugal;Slovakia;Greece;Spain;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland;Romania;Bulgaria;Germany;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31EUCTR2019-000750-63-PL
(EUCTR)
15/09/202021/07/2020Cystic Fibrosis: A Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in subjects who have two copies of the F508del mutationA Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in Cystic Fibrosis Subjects Who Are Homozygous for the F508del Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ABBV-2222
Product Code: ABBV-2222
INN or Proposed INN: Galicaftor
Other descriptive name: ABBV-2222
Product Name: ABBV-2222
Product Code: ABBV-2222
INN or Proposed INN: Galicaftor
Other descriptive name: ABBV-2222
Product Name: ABBV-2222
Product Code: ABBV-2222
INN or Proposed INN: Galicaftor
Other descriptive name: ABBV-2222
Product Name: ABBV-3067
Product Code: ABBV-3067
Other descriptive name: ABBV-3067
AbbVie Deutschland GmbH & Co. KGNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
189Phase 2United States;Serbia;Slovakia;Spain;United Kingdom;France;Czech Republic;Hungary;Canada;Puerto Rico;Poland;Belgium;Netherlands
32NCT04531410
(ClinicalTrials.gov)
September 1, 202018/8/2020NETwork of Linoleic Acid Supplementation in Cystic FibrosisDouble-blind Randomized Controlled Study of Linoleic Acid Supplementation for 1 Year in Patients With Cystic Fibrosis - Influence on Clinical Status and MetabolismCystic FibrosisDietary Supplement: linoleic acid supplementation;Dietary Supplement: oleic acid supplementationKarolinska InstitutetEuropean Society of Pediatric Gastroenterology, Hepatology and NutritionEnrolling by invitation5 Years15 YearsAll90N/AItaly;Norway;Poland
33NCT04460586
(ClinicalTrials.gov)
September 1, 20201/7/2020Pharmacokinetics of Omadacycline in Cystic FibrosisPharmacokinetics of Omadacycline in Patients With Cystic FibrosisCystic FibrosisDrug: Omadacycline Injection [Nuzyra];Drug: Omadacycline Oral Tablet [Nuzyra]Paul BeringerParatek Pharmaceuticals IncNot yet recruiting18 YearsN/AAll12Phase 4United States
34NCT04509661
(ClinicalTrials.gov)
September 1, 202022/4/2020Efficacy and Safety of Inhaled Bronchodilator in Non-CF Bronchiectasis With Airflow LimitationEfficacy and Safety of Inhaled Bronchodilator in Non-cystic Fibrosis Bronchiectasis Patients With Airflow Limitation: a Multicenter, Open-label Randomized Controlled TrialBronchiectasis AdultDrug: LABA/LAMA or Placebo inhalationShanghai Pulmonary Hospital, Shanghai, ChinaNULLNot yet recruiting18 YearsN/AAll200Phase 4NULL
35NCT04496921
(ClinicalTrials.gov)
September 202021/7/2020Vitamin K Supplementation to Reduce Deficiencies in Adults With Cystic FibrosisVitamin K Supplementation to Reduce Deficiencies in Adults With Cystic Fibrosis : A Pilot StudyCystic FibrosisDietary Supplement: Vitamin K supplementation, dose #1;Dietary Supplement: Vitamin K supplementation, dose #2Institut de Recherches Cliniques de MontrealUniversité de MontréalNot yet recruiting18 YearsN/AAll40N/ACanada
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36EUCTR2020-001762-11-HU
(EUCTR)
26/08/202002/07/2020A research study to collect long term safety information from subjects who have received study treatment from a qualifying lenabasum Corbus trial.An Observational Long-Term Safety Surveillance of Participants from Corbus Sponsored Lenabasum Pivotal Clinical Trials Dermatomyositis (DM) is a rare and serious autoimmune disease.An overactive immune response causes chronic inflammation,which results in growth of scar tissue in the skin,muscles,and many internal organs.Cystic Fibrosis (CF) is a genetic disorder which results in thick mucus formation on the airways leading to increased lung infections,fibrosis of the lungs and digestive tract and abnormal immune function
MedDRA version: 20.0;Level: PT;Classification code 10012503;Term: Dermatomyositis;System Organ Class: 10040785 - Skin and subcutaneous tissue disorders
MedDRA version: 20.0;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Not possible to specify
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
100Phase 2;Phase 3United States;Serbia;Portugal;Slovakia;Greece;Spain;Austria;Russian Federation;United Kingdom;Italy;France;Hungary;Czech Republic;Canada;Belgium;Poland;Romania;Bulgaria;Germany;Sweden
37NCT04378153
(ClinicalTrials.gov)
August 25, 20204/5/2020Impact of Discontinuing Chronic Therapies in People With Cystic Fibrosis on Highly Effective CFTR Modulator TherapyA Master Protocol to Test the Impact of Discontinuing Chronic Therapies in People With Cystic Fibrosis on Highly Effective CFTR Modulator Therapy (SIMPLIFY)Cystic FibrosisOther: Discontinuation of hypertonic saline (HS);Other: Continuation of hypertonic saline (HS);Other: Discontinuation of dornase alfa (dnase);Other: Continuation of dornase alfa (dnase)David Nichols, MDCystic Fibrosis Foundation;Dartmouth-Hitchcock Medical Center;University of WashingtonRecruiting12 YearsN/AAll800N/AUnited States
38NCT04530383
(ClinicalTrials.gov)
August 22, 202024/8/2020A Trial of Metformin for Improving CFTR Function in CFRDAn Open Label Phase 2 Trial of Metformin for Improving CFTR Function in CFRDCystic Fibrosis-related Diabetes;Cystic FibrosisDrug: Metformin HydrochlorideUniversity of Kansas Medical CenterNULLActive, not recruiting18 YearsN/AAll20Phase 2United States
39EUCTR2019-003554-86-DK
(EUCTR)
19/08/202027/03/2020A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 through 11 Years Old With Cystic Fibrosis and F/MF genotypesA Phase 3b, Randomized, Placebo-Controlled Study Evaluating the Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 6 Through 11 Years of Age Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 50 mg ELX/25 mg TEZ/37.5 mg IVA fixed dose combination
Product Code: VX-445 / TEZ / IVA
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: elexacaftor
Other descriptive name: ELX
INN or Proposed INN: Tezacaftor
Other descriptive name: TEZ
Product Name: 150-mg IVACAFTOR
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Product Name: 100 mg ELX/50 mg TEZ/75 mg IVA fixed dose combination
Product Code: VX-445 / TEZ / IVA
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: elexacaftor
Other descriptive name: ELX
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
108Phase 3France;Canada;Spain;Australia;Denmark;Israel;Netherlands;Germany;United Kingdom;Switzerland
40NCT04381429
(ClinicalTrials.gov)
August 17, 202030/4/2020Effect of Postprandial Insulin Administration of Faster-acting Insulin Analogue Versus Pre-prandial Administration of Acting-insulin Analogue in Cystic Fibrosis Related DiabetesEffect of Postprandial Insulin Administration of Faster-acting Insulin Analogue Versus Pre-prandial Administration of Acting-insulin Analogue in Cystic Fibrosis Related Diabetes : MIRE TrialCystic Fibrosis-related DiabetesDrug: A-F-A-F (NovoRapid-FIASP-NovoRapid-FIASP);Drug: F-A-F-A (FIASP-NovoRapid-FIASP-NovoRapid)University Hospital, Strasbourg, FranceNULLRecruiting18 YearsN/AAll40Phase 4France
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
41EUCTR2019-003554-86-DE
(EUCTR)
14/08/202012/03/2020A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 through 11 Years Old With Cystic Fibrosis and F/MF genotypesA Phase 3b, Randomized, Placebo-Controlled Study Evaluating the Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 6 Through 11 Years of Age Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 50 mg ELX/25 mg TEZ/37.5 mg IVA fixed dose combination
Product Code: VX-445 / TEZ / IVA
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: elexacaftor
Other descriptive name: ELX
INN or Proposed INN: Tezacaftor
Other descriptive name: TEZ
Product Name: 150-mg IVACAFTOR
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Product Name: 100 mg ELX/50 mg TEZ/75 mg IVA fixed dose combination
Product Code: VX-445 / TEZ / IVA
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: elexacaftor
Other descriptive name: ELX
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
108Phase 3Switzerland;Germany;United Kingdom;France;Canada;Spain;Denmark;Australia;Israel;Netherlands
42NCT04375514
(ClinicalTrials.gov)
August 10, 202030/4/2020Study of ARO-ENaC in Healthy Volunteers and in Patients With Cystic FibrosisA Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability and Pharmacokinetic Effects of ARO-ENaC in Normal Healthy Volunteers and Safety, Tolerability and Efficacy in Patients With Cystic FibrosisCystic Fibrosis, PulmonaryDrug: ARO-ENaC;Drug: PlaceboArrowhead PharmaceuticalsNULLRecruiting18 Years55 YearsAll54Phase 1;Phase 2Australia;New Zealand
43EUCTR2016-004558-13-PT
(EUCTR)
27/07/202020/04/2020A double-blind, placebo controlled, multicentre, clinical trial to investigate the efficacy and safety of 12 months of therapy with inhaled colistimethate sodium in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa)A double-blind, placebo controlled, multicentre, clinical trial to investigate the efficacy and safety of 12 months of therapy with inhaled colistimethate sodium in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa) - PROMIS II Non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa
MedDRA version: 21.0;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]
Trade Name: Promixin, 1 million International Units (IU) Powder for Nebuliser Solution
Product Name: Colistimethate sodium
INN or Proposed INN: COLISTIMETHATE SODIUM
Other descriptive name: Colistimethate Sodium
Zambon S.p.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
420Phase 3United States;France;Portugal;Greece;Canada;Argentina;Poland
44NCT04553419
(ClinicalTrials.gov)
July 27, 202011/9/2020Antibiotic Treatment Of Staphylococcus Aureus In Stable People With CFAntibiotic Treatment Of Staphylococcus Aureus In Stable People With CF (ASAP-CF) Clinical Research ProtocolCystic FibrosisDrug: Cephalexin;Drug: PlaceboUniversity of British ColumbiaThe Hospital for Sick ChildrenRecruiting3 Years16 YearsAll86Phase 3Canada
45NCT04058210
(ClinicalTrials.gov)
July 21, 202013/8/2019VX-445/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients Heterozygous for F508del Mutation and a Minimal Function Mutation (F/MF Genotypes)VX-445/TEZ/IVA Triple Combination Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis Who Are Heterozygous for F508del and a Minimal Function CFTR MutationCystic FibrosisDrug: ELX/TEZ/IVA;Drug: IVAVertex Pharmaceuticals IncorporatedNULLApproved for marketing12 YearsN/AAllNULL
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
46NCT04375878
(ClinicalTrials.gov)
July 20, 202030/4/2020OPTION 2: A Trial to Assess the Safety and Efficacy of MS1819 in Enteric Capsules in Patients With Cystic FibrosisOPTION 2: A Phase 2, Open-Label, Multicenter, 2x2 Crossover Trial to Assess the Safety and Efficacy of MS1819 in Enteric Capsules in Patients With Exocrine Pancreatic Insufficiency Due to Cystic FibrosisExocrine Pancreatic Insufficiency (EPI);Cystic Fibrosis (CF)Drug: MS1819;Drug: Porcine PERTAzurRx BioPharma, Inc.NULLRecruiting18 YearsN/AAll30Phase 2United States;Poland
47NCT04458129
(ClinicalTrials.gov)
July 8, 202030/6/2020Polyethylene Glycol and Intestinal Inflammation in Cystic FibrosisEFFECT OF POLYETHYLENE GLYCOL TREATMENT ON INTESTINAL INFLAMMATION ASSOCIATED WITH CYSTIC FIBROSIS IN CHILDRENCystic FibrosisDrug: Treatment with polyethylene glycol (Macrogol 4000)University Hospital, BordeauxNULLNot yet recruiting4 Years17 YearsAll23Phase 2NULL
48EUCTR2016-004558-13-GR
(EUCTR)
07/07/202014/05/2020Treatment for bronchiectasis not due to cystic fibrosis.A double-blind, placebo controlled, multicentre, clinical trial to investigate the efficacy and safety of 12 months of therapy with inhaled colistimethate sodium in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa) - PROMIS II Non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa
MedDRA version: 21.0;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]
Trade Name: Promixin, 1 million International Units (IU) Powder for Nebuliser Solution
Product Name: Colistimethate sodium
INN or Proposed INN: COLISTIMETHATE SODIUM
Other descriptive name: Colistimethate Sodium
Zambon S.p.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
420Phase 3United States;France;Canada;Greece;Argentina;Poland
49NCT04279769
(ClinicalTrials.gov)
July 3, 202010/2/2020Study to Evaluate the Safety of CB-280 in Patients With Cystic FibrosisA Phase 1b Randomized, Double-blind, Placebo-Controlled Trial to Evaluate the Safety of CB-280 in Patients With Cystic FibrosisCystic FibrosisDrug: CB-280;Drug: PlacebosCalithera Biosciences, IncNULLRecruiting18 YearsN/AAll32Phase 1United States
50NCT04353817
(ClinicalTrials.gov)
June 19, 202016/4/2020A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF GenotypesA Phase 3b, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 6 Through 11 Years of Age Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)Cystic FibrosisDrug: ELX/TEZ/IVA;Drug: IVA;Other: PlaceboVertex Pharmaceuticals IncorporatedNULLRecruiting6 Years11 YearsAll108Phase 3Australia;Canada;Denmark;France;Germany;Israel;Netherlands;Spain;Switzerland;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
51NCT04415268
(ClinicalTrials.gov)
June 9, 202028/5/2020Combined Effect of CFTR Protein Modulator Drugs and Exercise in Cystic FibrosisCombined Effect of CFTR Protein Modulator Drugs and Exercise on Pulmonary Function, Fitness, Sweat Test and Quality of Life in Children With Cystic FibrosisCystic Fibrosis in ChildrenBehavioral: Exercise;Behavioral: Unsupervised exercise;Drug: CFTR ModulatorsUniversidad Europea de MadridNULLRecruiting6 Years18 YearsAll11N/ASpain
52NCT04362761
(ClinicalTrials.gov)
May 4, 202022/4/2020A Study Evaluating the Long-Term Safety of Elexacaftor Combination TherapyA Phase 3b Open-label Extension Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis SubjectsCystic FibrosisDrug: ELX/TEZ/IVA;Drug: IVAVertex Pharmaceuticals IncorporatedNULLActive, not recruiting12 YearsN/AAll172Phase 3Australia;Belgium;Germany;United Kingdom
53EUCTR2019-003554-86-GB
(EUCTR)
01/05/202001/04/2020A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 through 11 Years Old With Cystic Fibrosis and F/MF genotypesA Phase 3b, Randomized, Placebo-Controlled Study Evaluating the Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 6 Through 11 Years of Age Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) - Phase 3b Study of ELX/TEZ/IVA in CF subjects 6 Through 11 Years of Age Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 50 mg ELX/25 mg TEZ/37.5 mg IVA fixed dose combination
Product Code: VX-445 / TEZ / IVA
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: elexacaftor
Other descriptive name: ELX
INN or Proposed INN: Tezacaftor
Other descriptive name: TEZ
Product Name: 150-mg IVACAFTOR
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Product Name: 100 mg ELX/50 mg TEZ/75 mg IVA fixed dose combination
Product Code: VX-445 / TEZ / IVA
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: elexacaftor
Other descriptive name: ELX
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
108Phase 3France;Canada;Spain;Denmark;Australia;Israel;Netherlands;Germany;Switzerland;United Kingdom
54NCT04294043
(ClinicalTrials.gov)
May 1, 20201/3/2020IV Gallium Study for Patients With Cystic Fibrosis Who Have NTM (ABATE Study)A Phase 1b, Multi-center Study of Intravenous (IV) Gallium Nitrate in Patients With Cystic Fibrosis (CF) Who Are Colonized With Nontuberculous Mycobacteria (NTM) (The ABATE Study)Nontuberculous Mycobacterium InfectionDrug: Gallium nitrateChris GossCystic Fibrosis FoundationNot yet recruiting18 YearsN/AAll40Phase 1United States
55ChiCTR2000031817
2020-04-302020-04-11Evaluation and comparison of quality of life between acetylcysteine tablets and placebo in patients with non cystic fibrosis bronchiectasisA Randomized, Double-Blinded, Placebo-Controlled, Multiple-Center Clinical Trial to Compare the Quality of Life of Acetylcysteine Tablets 600 Mg BID Treatment With Placebo in Patients With Non-Cystic Fibrosis Bronchiectasis bronchiectasiaexperimental group:Acetylcysteine;control group:placebo;West China Hospital, Sichuan UniversityNULLPending18Bothexperimental group:79;control group:40;Phase 4China
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
56EUCTR2019-003455-11-DE
(EUCTR)
08/04/202009/12/2019Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis SubjectsA Phase 3b Open-label Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg ELX / 50-mg TEZ / 75 -mg IVA FDC
Product Code: ELX/TEZ/IVA
INN or Proposed INN: SUB185183
Other descriptive name: VX-445
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
158Phase 3Belgium;Australia;Germany;United Kingdom
57EUCTR2019-004958-29-FR
(EUCTR)
07/04/202004/06/2020Polyethylene glycol and intestinal inflammation in cystic fibrosisEFFECT OF POLYETHYLENE GLYCOL TREATMENT ON INTESTINAL INFLAMMATION ASSOCIATED WITH CYSTIC FIBROSIS IN CHILDREN - MUCOLAX Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: FORLAX 4 g, poudre pour solution buvable en sachetCHU de BordeauxNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
23Phase 2France
58EUCTR2019-004940-31-DK
(EUCTR)
24/03/202024/03/2020Effect of timing of enzyme replacement therapy (Creon®) at meals on fat malabsorption in adult patients with cystic fibrosis - a cross-over trialEffect of timing of enzyme replacement therapy (Creon®) at meals on fat malabsorption in adult patients with cystic fibrosis - a cross-over trial GI pains and fat malabsorption in cysticfibrosis patients with pancreas insufficiency
MedDRA version: 20.1;Level: LLT;Classification code 10025479;Term: Malabsorption syndrome;System Organ Class: 100000004856;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: Creon 10.000, hårde enterokapsler
INN or Proposed INN: Pankreatin
Other descriptive name: PANCREATIN
Trade Name: Creon 25.000, hårde enterokapsler
INN or Proposed INN: Pankreatin
Other descriptive name: PANCREATIN
Trade Name: Creon 40.000, hårde enterokapsler
INN or Proposed INN: Pankreatin
Other descriptive name: PANCREATIN
Terese KatzensteinNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 4Denmark
59NCT04118010
(ClinicalTrials.gov)
March 13, 20204/10/2019Vitamin D and Prebiotics for Intestinal Health in Cystic FibrosisVitamin D and Prebiotics for Intestinal Health in Cystic FibrosisCystic Fibrosis;DysbiosisDrug: Vitamin D3;Drug: Placebo vitamin D3;Drug: Inulin;Drug: Placebo InulinEmory UniversityNULLRecruiting18 YearsN/AAll40Phase 4United States
60EUCTR2018-000378-30-DE
(EUCTR)
04/03/202006/06/2019A dose finding study of OligoG in patients with CF.A randomised, double-blind, dose finding study of inhaled alginate oligosaccharide (OligoG) vs placebo in patients with Cystic Fibrosis (CF). Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011763;Term: Cystic fibrosis lung;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
AlgiPharma ASNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
168Phase 2Poland;Ireland;Austria;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
61EUCTR2018-000378-30-GB
(EUCTR)
03/03/202026/06/2019A dose finding study of OligoG in patients with CF.A randomised, double-blind, dose finding study of inhaled alginate oligosaccharide (OligoG) vs placebo in patients with Cystic Fibrosis (CF). Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011763;Term: Cystic fibrosis lung;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: OligoG CF-5/20 - 17.5 mg
INN or Proposed INN: OLIGOG CF-5/20
Other descriptive name: OLIGOG CF-5/20
Product Name: OligoG CF/20 - 27.5 mg
INN or Proposed INN: OLIGOG CF-5/20
Other descriptive name: OLIGOG CF-5/20
Product Name: OligoG CF/20 - 37.5 mg
INN or Proposed INN: OLIGOG CF-5/20
Other descriptive name: OLIGOG CF-5/20
AlgiPharma ASNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
168Phase 2Poland;Ireland;Austria;Germany;United Kingdom
62NCT04235140
(ClinicalTrials.gov)
February 24, 202015/1/2020Long-term Safety of Lumacaftor/Ivacaftor in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment InitiationA Phase 3, Open-label, and Rollover Study to Evaluate the Long-term Safety and Tolerability of Lumacaftor/Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment InitiationCystic FibrosisDrug: LUM/IVAVertex Pharmaceuticals IncorporatedNULLEnrolling by invitation12 MonthsN/AAll50Phase 3United States;Canada
63NCT04166396
(ClinicalTrials.gov)
February 17, 202014/11/2019Non-pulmonary Contributors of Exercise Intolerance in Patients With Cystic FibrosisNon-pulmonary Contributors of Exercise Intolerance in Patients With Cystic FibrosisCystic FibrosisDietary Supplement: Resveratrol;Dietary Supplement: NR;Dietary Supplement: PlaceboVirginia Commonwealth UniversityNULLActive, not recruiting18 YearsN/AAll36N/AUnited States
64NCT04183790
(ClinicalTrials.gov)
February 17, 202028/11/2019Evaluation of Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and OlderA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445/TEZ/IVA Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and OlderCystic FibrosisDrug: ELX/TEZ/IVA;Drug: IVAVertex Pharmaceuticals IncorporatedNULLActive, not recruiting6 YearsN/AAll64Phase 3United States;Australia;Canada;Ireland;United Kingdom
65EUCTR2019-000955-16-AT
(EUCTR)
28/01/202007/01/2020Characterization of lung concentrations of the antibiotics piperacillin/tazobactam and levofloxacinPulmonary pharmacokinetics of piperacillin/tazobactam and levofloxacin in patients with chronic obstructive pulmonary disease or cystic fibrosis: Comparison of epithelial lining fluid, in-vivo microdialysis and tissue biopsy Pneumonia;Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]Trade Name: Piperacillin/Tazobactam Kabi - Fresenius Kabi Austria
INN or Proposed INN: piperacillin
Other descriptive name: PIPERACILLIN
INN or Proposed INN: tazobactam
Other descriptive name: TAZOBACTAM
Trade Name: Levofloxacin Kabi
INN or Proposed INN: levofloxacin
Other descriptive name: LEVOFLOXACIN HEMIHYDRATE
Medical University of ViennaNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
18Phase 4Austria
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
66EUCTR2018-003831-31-ES
(EUCTR)
24/01/202002/12/2019MS1819-SD phase II clinical trial for Exocrine Pancreatic Insufficiency caused by Cystic Fibrosis.A multicenter, open-label Phase 2 study with escalating doses of MS1819-SD on top of a stable dose of PPEs, to investigate the efficacy and safety of this combination for the compensation of severe exocrine pancreatic insufficiency in CF patients not fully compensated with only PPEs Exocrine Pancreatic Insufficiency
MedDRA version: 20.0;Level: LLT;Classification code 10033628;Term: Pancreatic insufficiency;System Organ Class: 100000004856 ;Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
AzurRxNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
24Phase 2Hungary;Spain
67EUCTR2019-000833-37-DE
(EUCTR)
23/01/202027/06/2019A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445 / 50-mg TEZ / 75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: elexacaftor
Other descriptive name: VX-445
Trade Name: Kalydeco 150 mg film-coated tablets
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Trikafta
Product Name: 100-mg ELX (VX-455) / 50-mg TEZ / 75-mg IVA FDC
Product Code: ELX/TEZ/IVA
INN or Proposed INN: Elexacaftor
Other descriptive name: ELX
INN or Proposed INN: Tezacaftor
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
250Phase 3United States;France;Canada;Spain;Belgium;Ireland;Denmark;Australia;Netherlands;Germany;United Kingdom;Italy
68EUCTR2018-002835-76-NL
(EUCTR)
22/01/202017/09/2019A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445 / 50-mg TEZ / 75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: elexacaftor
Other descriptive name: VX-445
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Symkevi 100mg/150mg Film-coated tablets
Product Code: VX-661 / VX-770
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Kalydeco 150 mg film-coated tablets
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
250Phase 3United States;France;Canada;Spain;Belgium;Ireland;Denmark;Australia;Germany;Netherlands;United Kingdom;Italy
69EUCTR2019-000833-37-NL
(EUCTR)
22/01/202017/09/2019A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445 / 50-mg TEZ / 75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: elexacaftor
Other descriptive name: VX-445
Trade Name: Kalydeco 150 mg film-coated tablets
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
250Phase 3United States;France;Canada;Spain;Belgium;Ireland;Denmark;Australia;Germany;Netherlands;United Kingdom;Italy
70EUCTR2019-003455-11-GB
(EUCTR)
17/01/202009/12/2019Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis SubjectsA Phase 3b Open-label Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
158Phase 3Belgium;Australia;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
71EUCTR2018-004652-38-IE
(EUCTR)
15/01/202017/06/2019A Study Evaluating the Long-term Safety of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis - A Study Evaluating the Long-term Safety of VX-445 Combination Therapy Cystic Fibrosis
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
481Phase 3United States;Canada;Poland;Spain;Ireland;Denmark;Australia;Israel;Germany;United Kingdom;Switzerland
72NCT04259970
(ClinicalTrials.gov)
January 13, 202015/1/2020Hyperpolarized Imaging for New TreatmentsHyperpolarized Imaging for New Treatments (HyPOINT) StudyCystic FibrosisDrug: Initiation of CFTR ModulatorChildren's Hospital Medical Center, CincinnatiUniversity of Virginia;University of Wisconsin, Madison;The Hospital for Sick ChildrenRecruiting6 Years18 YearsAll64Phase 4United States;Canada
73NCT04301856
(ClinicalTrials.gov)
January 1, 202021/2/2020Response to CFTR Modulators in CF Patients Under 18 YearsEvaluation of the Response to CFTR Modulators in Patients With Cystic Fibrosis Less Than 18 Years of AgeCystic Fibrosis in ChildrenDrug: CFTR ModulatorsSociete Francaise de la MucoviscidoseNULLRecruitingN/A18 YearsAll600France
74NCT04206436
(ClinicalTrials.gov)
December 18, 201918/12/2019CFTR Modulator Effects on Bone and Muscle in Adults With Cystic FibrosisCFTR Modulator Effects on Bone and Muscle in Adults With Cystic FibrosisCystic Fibrosis;Bone Loss;Muscle LossDrug: Cftr ModulatorsIndiana UniversityCystic Fibrosis FoundationEnrolling by invitation18 YearsN/AAll60United States
75NCT03894657
(ClinicalTrials.gov)
December 18, 201912/2/2019Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®.Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®. Pathway to Personalized Therapy in Cystic FibrosisCystic Fibrosis;Homozygous F508del MutationDiagnostic Test: Nasal brushing;Other: Sputum sample;Other: blood sample;Drug: OrkambiAssistance Publique - Hôpitaux de ParisNULLRecruiting5 YearsN/AAll104N/AFrance
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
76NCT04210427
(ClinicalTrials.gov)
December 12, 201918/12/2019Cystic Fibrosis and Gut Dysmotility: The Effect of Polyethylene Glycol (PEG) on Intestinal TransitCystic Fibrosis and Gut Dysmotility: The Effect of Polyethylene Glycol (PEG) on Intestinal TransitCystic Fibrosis Gastrointestinal DiseaseDrug: Polyethylene Glycol 3350;Device: SmartPill Motility System & PillCam Patency CapsuleSt. Louis UniversityNULLRecruiting18 Years40 YearsAll15Phase 4United States
77NCT03969888
(ClinicalTrials.gov)
December 11, 201930/5/2019A Phase 2 Study of ABBV-3067 Alone and in Combination With ABBV-2222A Phase 2 Study of ABBV-3067 Alone and in Combination With ABBV-2222 in Cystic Fibrosis Subjects Who Are Homozygous for the F508del MutationCystic FibrosisDrug: ABBV-3067;Drug: Placebo ABBV-3067;Drug: ABBV-2222;Drug: Placebo ABBV-2222AbbVieNULLRecruiting18 YearsN/AAll189Phase 2United States;Belgium;Canada;Czechia;France;Hungary;Netherlands;New Zealand;Poland;Romania;Serbia;Slovakia;Spain;United Kingdom
78NCT03988816
(ClinicalTrials.gov)
December 6, 201914/6/2019Effect of Roflumilast on Quality of Life, Lung Function and Mucus Properties in Patients With BronchiectasisEffect of Roflumilast on Quality of Life, Lung Function and Mucus Properties in Patients With Non-cystic Fibrosis Bronchiectasis: a Cross-over, Unicentric, Double-blind and Placebo-controlled StudyBronchiectasis AdultDrug: Roflumilast;Drug: Placebo oral tabletUniversity of Sao Paulo General HospitalFAPESP - Fundação de Apoio à Pesquisa do Estado de São PauloRecruiting18 YearsN/AAll30Phase 2Brazil
79NCT04058366
(ClinicalTrials.gov)
December 5, 201914/8/2019Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)Cystic FibrosisDrug: ELX/TEZ/IVA;Drug: IVAVertex Pharmaceuticals IncorporatedNULLActive, not recruiting12 YearsN/AAll251Phase 3United States;Australia;Belgium;Canada;Denmark;France;Germany;Ireland;Italy;Netherlands;Spain;United Kingdom
80NCT04135495
(ClinicalTrials.gov)
November 25, 201916/8/2019A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD of ELX-02 in Cystic Fibrosis Patients With G542X AlleleA Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients With Cystic Fibrosis With at Least One G542X AlleleCystic FibrosisDrug: ELX-02Eloxx Pharmaceuticals, Inc.NULLRecruiting18 YearsN/AAll8Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
81EUCTR2019-003178-25-HU
(EUCTR)
19/11/201920/11/2019A study of safety, tolerability and effect of SNSP113 in Cystic FibrosisA Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Safety, Tolerability, and Effect of Inhaled SNSP113 in Adult Subjects with Cystic Fibrosis Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: SNSP113
Product Code: SNSP113
INN or Proposed INN: not assigned
Other descriptive name: PAAG15
Synspira Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
56Phase 2Hungary
82EUCTR2019-000833-37-ES
(EUCTR)
19/11/201909/08/2019A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
250Phase 3France;United States;Canada;Belgium;Spain;Ireland;Denmark;Australia;Netherlands;Germany;Italy;United Kingdom
83EUCTR2018-000966-12-DE
(EUCTR)
18/11/201925/06/2019A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients with Cystic FibrosisA Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients with Cystic Fibrosis with at Least One G542X Allele Cystic fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Code: ELX-02
INN or Proposed INN: Not Assigned
Eloxx Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
16Phase 2Israel;Germany
84EUCTR2019-000750-63-FR
(EUCTR)
15/11/201922/08/2019Cystic Fibrosis: A Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in subjects who have two copies of the F508del mutationA Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in Cystic Fibrosis Subjects Who Are Homozygous for the F508del Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
AbbVie DeutschlandNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
189Phase 2Serbia;United States;France;Hungary;Czech Republic;Slovakia;Puerto Rico;Canada;Belgium;Spain;Netherlands;United Kingdom
85EUCTR2018-004652-38-GB
(EUCTR)
06/11/201919/06/2019A Study Evaluating the Long-term Safety of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis - A Study Evaluating the Long-term Safety of VX-445 Combination Therapy Cystic Fibrosis
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445 /50-mg TEZ/75-mg
Product Code: VX-445 /TEZ/IVA
INN or Proposed INN: TEZACAFTOR
Other descriptive name: VX-661
INN or Proposed INN: IVACAFTOR
INN or Proposed INN: VX-445
Other descriptive name: VX-445
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
481Phase 3United States;Canada;Poland;Spain;Ireland;Denmark;Australia;Israel;Germany;Switzerland;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
86NCT04126473
(ClinicalTrials.gov)
November 5, 201916/8/2019A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X AlleleA Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients With Cystic Fibrosis With at Least One G542X AlleleCystic FibrosisDrug: ELX-02Eloxx Pharmaceuticals, Inc.NULLRecruiting16 YearsN/AAll16Phase 2Germany;Israel;Australia
87EUCTR2019-000261-21-GB
(EUCTR)
05/11/201905/06/2019A 4-week study to test different doses of BI 1265162 in adolescents and adults with cystic fibrosis using the Respimat® inhaler - BALANCE - CF(TM) 1A randomised, double-blind, placebo-controlled and parallel group trial to evaluate efficacy and safety of twice daily inhaled doses of BI 1265162 delivered by Respimat® inhaler as add-on therapy to standard of care over 4 weeks in patients with cystic fibrosis - BALANCE - CF(TM) 1 Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: BI 1265162 10 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Product Name: BI 1265162 25 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Product Name: BI 1265162 50 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Product Name: BI 1265162 100 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Boehringer Ingelheim LimitedNULLNot RecruitingFemale: yes
Male: yes
98Phase 2United States;France;Canada;Spain;Belgium;Ireland;Germany;United Kingdom;Sweden
88EUCTR2018-000378-30-AT
(EUCTR)
04/11/201905/07/2019A dose finding study of OligoG in patients with CF.A randomised, double-blind, dose finding study of inhaled alginate oligosaccharide (OligoG) vs placebo in patients with Cystic Fibrosis (CF). Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011763;Term: Cystic fibrosis lung;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
AlgiPharma ASNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
168Phase 2Poland;Ireland;Austria;Germany;United Kingdom
89EUCTR2019-000750-63-GB
(EUCTR)
31/10/201926/06/2019Cystic Fibrosis: A Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in subjects who have two copies of the F508del mutationA Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in Cystic Fibrosis Subjects Who Are Homozygous for the F508del Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ABBV-2222
Product Code: ABBV-2222
INN or Proposed INN: Galicaftor
Other descriptive name: ABBV-2222
INN or Proposed INN: Galicaftor
Other descriptive name: ABBV-2222
INN or Proposed INN: Galicaftor
Other descriptive name: ABBV-2222
Product Name: ABBV-3067
Product Code: ABBV-3067
Other descriptive name: ABBV-3067
AbbVie DeutschlandNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
189Phase 2Serbia;France;United States;Hungary;Czech Republic;Slovakia;Puerto Rico;Canada;Belgium;Spain;Netherlands;United Kingdom
90EUCTR2019-000750-63-NL
(EUCTR)
31/10/201905/08/2019Cystic Fibrosis: A Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in subjects who have two copies of the F508del mutationA Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in Cystic Fibrosis Subjects Who Are Homozygous for the F508del Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
AbbVie DeutschlandNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
189Phase 2Serbia;France;United States;Hungary;Czech Republic;Slovakia;Puerto Rico;Canada;Belgium;Spain;Netherlands;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
91EUCTR2018-002835-76-GB
(EUCTR)
29/10/201927/06/2019A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445 / 50-mg TEZ / 75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: elexacaftor
Other descriptive name: VX-445
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Symkevi 100mg/150mg Film-coated tablets
Product Code: VX-661 / VX-770
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Kalydeco 150 mg film-coated tablets
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
250Phase 3United States;France;Canada;Spain;Belgium;Ireland;Denmark;Australia;Netherlands;Germany;Italy;United Kingdom
92EUCTR2019-000833-37-GB
(EUCTR)
29/10/201927/06/2019A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445 / 50-mg TEZ / 75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: elexacaftor
Other descriptive name: VX-445
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
250Phase 3France;United States;Canada;Belgium;Spain;Ireland;Denmark;Australia;Netherlands;Germany;Italy;United Kingdom
93NCT04176328
(ClinicalTrials.gov)
October 25, 20198/11/2019Open-label, Dose-escalation Study to Evaluate the Pharmacokinetics of Inhaled Teicoplanin in Cystic Fibrosis PatientsAn Open-label, Dose-escalation Study to Evaluate the Pharmacokinetics of Inhaled Teicoplanin in Cystic Fibrosis PatientsCystic FibrosisDrug: Teicoplanin Sandoz 200 mg powder and solvent for solution for injection or infusion or oral solution.Neupharma SrlSintesi Research Srl;Aptuit Srl;Pari Pharma GmbHCompleted18 YearsN/AAll12Phase 1Italy
94NCT03924947
(ClinicalTrials.gov)
October 23, 201918/4/2019A Study to Compare US Marketed Pancrelipase Drug Product With Drug Product Manufactured With a Modernized Process at an Alternate Manufacturing Site, in Participants With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis.A Phase 4 Study to Compare US Marketed Creon Drug Product With Drug Product Manufactured With a Modernized Process at an Alternate Manufacturing Site, in Subjects With Exocrine Pancreatic Insufficiency (EPI) Due to Cystic FibrosisCystic FibrosisDrug: PancrelipaseAbbVieNULLRecruiting12 YearsN/AAll28Phase 4United States;Spain
95NCT04437641
(ClinicalTrials.gov)
October 22, 201926/3/2020Impact of Pediatrician Intervention on the Smoking Habits of Parents of Sick ChildrenImpact of Pediatrician Intervention on the Smoking Habits of Parents of Children With Cystic Fibrosis, Type 1 Diabetes or Children Hospitalized for a First Episode of Bronchiolitis.Tobacco Consumption;BronchiolitisOther: Tobacco questionnaireUniversity Hospital, ToulouseNULLRecruiting18 Years65 YearsAll600France
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
96EUCTR2019-000833-37-IE
(EUCTR)
21/10/201927/06/2019A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
250Phase 3France;United States;Canada;Belgium;Spain;Ireland;Denmark;Australia;Netherlands;Germany;Italy;United Kingdom
97EUCTR2018-002835-76-DE
(EUCTR)
21/10/201903/07/2019A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445 / 50-mg TEZ / 75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: elexacaftor
Other descriptive name: VX-445
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Symkevi 100mg/150mg Film-coated tablets
Product Code: VX-661 / VX-770
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Kalydeco 150 mg film-coated tablets
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
250Phase 3United States;France;Canada;Spain;Belgium;Ireland;Denmark;Australia;Netherlands;Germany;United Kingdom;Italy
98EUCTR2018-002835-76-IE
(EUCTR)
21/10/201928/06/2019A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445 / 50-mg TEZ / 75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: elexacaftor
Other descriptive name: VX-445
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Symkevi 100mg/150mg Film-coated tablets
Product Code: VX-661 / VX-770
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Kalydeco 150 mg film-coated tablets
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
250Phase 3United States;France;Canada;Spain;Belgium;Ireland;Denmark;Australia;Netherlands;Germany;United Kingdom;Italy
99EUCTR2018-004652-38-DK
(EUCTR)
18/10/201928/06/2019A Study Evaluating the Long-term Safety of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis - A Study Evaluating the Long-term Safety of VX-445 Combination Therapy Cystic Fibrosis
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445 /50-mg TEZ/75-mg
Product Code: VX-445 /TEZ/IVA
INN or Proposed INN: TEZACAFTOR
Other descriptive name: VX-661
INN or Proposed INN: IVACAFTOR
INN or Proposed INN: VX-445
Other descriptive name: VX-445
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
506Phase 3United States;Canada;Poland;Spain;Ireland;Australia;Denmark;Israel;Germany;United Kingdom;Switzerland
100EUCTR2019-000833-37-DK
(EUCTR)
17/10/201909/07/2019A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445 / 50-mg TEZ / 75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: elexacaftor
Other descriptive name: VX-445
Trade Name: Kalydeco 150 mg film-coated tablets
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
250Phase 3France;United States;Canada;Belgium;Spain;Ireland;Australia;Denmark;Netherlands;Germany;Italy;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
101EUCTR2018-002835-76-DK
(EUCTR)
16/10/201909/07/2019A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445 / 50-mg TEZ / 75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: elexacaftor
Other descriptive name: VX-445
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Symkevi 100mg/150mg Film-coated tablets
Product Code: VX-661 / VX-770
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Kalydeco 150 mg film-coated tablets
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
250Phase 3France;United States;Canada;Belgium;Spain;Ireland;Australia;Denmark;Netherlands;Germany;Italy;United Kingdom
102EUCTR2019-000261-21-ES
(EUCTR)
10/10/201905/07/2019A 4-week study to test different doses of BI 1265162 in adolescents and adults with cystic fibrosis using the Respimat® inhalerA randomised, double-blind, placebo-controlled and parallel group trial to evaluate efficacy and safety of twice daily inhaled doses of BI 1265162 delivered by Respimat® inhaler as add-on therapy to standard of care over 4 weeks in patients with cystic fibrosis Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: BI 1265162 10 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Product Name: BI 1265162 25 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Product Name: BI 1265162 50 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Product Name: BI 1265162 100 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Boehringer Ingelheim, S.A.NULLNot RecruitingFemale: yes
Male: yes
98Phase 2Spain;United States;France;Canada;Belgium;Ireland;Germany;United Kingdom;Sweden
103EUCTR2018-001695-38-GB
(EUCTR)
09/10/201919/08/2019Evaluation of VX-445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 YearsA Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-445/TEZ/IVA Triple Combination Therapy in Cystic Fibrosis Subjects 6 Through 11 Years of Age Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-445 / TEZ / IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: elexacaftor
Other descriptive name: VX-445
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: IVACAFTOR
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Product Name: VX-445 / TEZ / IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: elexacaftor
Other descriptive name: VX-445
Product Name: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
56Phase 3United States;Canada;Ireland;Australia;United Kingdom
104EUCTR2019-000261-21-SE
(EUCTR)
08/10/201929/04/2019A 4-week study to test different doses of BI 1265162 in adolescents and adults with cystic fibrosis using the Respimat®inhalerA randomised, double-blind, placebo-controlled and parallel group trial to evaluate efficacy and safety of twice daily inhaled doses of BI 1265162 delivered by Respimat® inhaler as add-on therapy to standard of care over 4 weeks in patients with cystic fibrosis Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: BI 1265162 10 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Product Name: BI 1265162 25 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Product Name: BI 1265162 50 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Product Name: BI 1265162 100 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Boehringer IngelheimNULLNot RecruitingFemale: yes
Male: yes
98Phase 2United States;France;Canada;Spain;Belgium;Ireland;Germany;United Kingdom;Sweden
105EUCTR2019-000261-21-BE
(EUCTR)
07/10/201929/05/2019A 4-week study to test different doses of BI 1265162 in adolescents and adults with cystic fibrosis using the Respimat® inhaler – BALANCE – CF(TM)1A randomised, double-blind, placebo-controlled and parallel group trial to evaluate efficacy and safety of twice daily inhaled doses of BI 1265162 delivered by Respimat® inhaler as add-on therapy to standard of care over 4 weeks in patients with cystic fibrosis – BALANCE – CF(TM)1 Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: BI 1265162 10 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Product Name: BI 1265162 25 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Product Name: BI 1265162 50 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Product Name: BI 1265162 100 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
SCS Boehringer Ingelheim Comm.VNULLNot RecruitingFemale: yes
Male: yes
98Phase 2United States;France;Canada;Spain;Belgium;Ireland;Germany;United Kingdom;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
106NCT04105972
(ClinicalTrials.gov)
October 3, 201924/9/2019A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508delA Phase 3b, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508delCystic FibrosisDrug: ELX/TEZ/IVA;Drug: TEZ/IVA;Drug: IVAVertex Pharmaceuticals IncorporatedNULLCompleted12 YearsN/AAll176Phase 3Australia;Belgium;Germany;United Kingdom
107NCT04090294
(ClinicalTrials.gov)
October 1, 201921/8/2019Assessment of Early Outcomes of Roflumilast in Patients With Non Cystic Fibrosis BronchiectasisAssessment of Early Outcomes of Roflumilast in Patients With Non Cystic Fibrosis BronchiectasisBronchiectasisDrug: RoflumilastAssiut UniversityNULLNot yet recruiting18 Years75 YearsAll35Early Phase 1NULL
108EUCTR2019-000750-63-BE
(EUCTR)
27/09/201907/08/2019Cystic Fibrosis: A Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in subjects who have two copies of the F508del mutationA Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in Cystic Fibrosis Subjects Who Are Homozygous for the F508del Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
AbbVie DeutschlandNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
189Phase 2Serbia;France;United States;Hungary;Czech Republic;Slovakia;Puerto Rico;Canada;Spain;Belgium;Netherlands;United Kingdom
109NCT04059094
(ClinicalTrials.gov)
September 16, 201915/8/2019A 4-week Study to Test Different Doses of BI 1265162 in Adolescents and Adults With Cystic Fibrosis Using the Respimat® Inhaler - BALANCE - CF™1A Randomised, Double-blind, Placebo-controlled and Parallel Group Trial to Evaluate Efficacy and Safety of Twice Daily Inhaled Doses of BI 1265162 Delivered by Respimat® Inhaler as add-on Therapy to Standard of Care Over 4 Weeks in Patients With Cystic Fibrosis - BALANCE - CF™ 1Cystic FibrosisDrug: BI 1265162;Drug: PlaceboBoehringer IngelheimNULLTerminated12 YearsN/AAll52Phase 2United States;Belgium;Canada;France;Germany;Spain;Sweden;United Kingdom
110EUCTR2019-001735-31-GB
(EUCTR)
13/09/201924/06/2019A study to evaluate the safety and efficacy of VX-445 / Tezacaftor / Ivacaftor in patients suffering from Cystic FibrosisA Phase 3b, Randomized, Double blind, Controlled Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del - A Study Evaluating the Efficacy & Safety of VX-445/Tezacaftor/Ivacafto in Cystic Fibrosis Subjects Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445 / 50-mg TEZ / 75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: ELEXACAFTOR
Other descriptive name: VX-445
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Symkevi 100mg/150mg Film-coated tablets
Product Code: VX-661 / VX-770
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
158Phase 3Belgium;Australia;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
111EUCTR2018-003970-28-NL
(EUCTR)
12/09/201909/05/2019A Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic FibrosisA Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Deutivacaftor
Product Code: VX-561
INN or Proposed INN: DEUTIVACAFTOR
INN or Proposed INN: DEUTIVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: Ivacaftor
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
88Phase 2United States;Belgium;Ireland;Australia;Germany;Netherlands;United Kingdom
112NCT04056702
(ClinicalTrials.gov)
September 5, 201927/7/2019Impact of Triple Combination CFTR Therapy on Sinus Disease.Impact of Elexacaftor-tezacaftor-ivacaftor Triple Combination CFTR Therapy on Sinus Disease: Quantitative Sinus Computed Tomography, Patient Reported Outcomes and Cellular and Molecular ChangesCystic FibrosisDrug: Elexacaftor-tezacaftor-ivacaftor exposureJennifer Taylor-CousarCystic Fibrosis FoundationActive, not recruiting18 Years89 YearsAll30United States
113NCT04038710
(ClinicalTrials.gov)
September 5, 201926/7/2019Clinical Outcomes of Triple Combination Therapy in Severe Cystic Fibrosis Disease.An Observational Study of the Effects on Clinical Outcomes of Expanded Access Program of Vertex Triple Combination Therapy.Cystic FibrosisDrug: Triple combination therapyNational Jewish HealthNULLActive, not recruiting12 YearsN/AAll8United States
114NCT04039087
(ClinicalTrials.gov)
September 5, 201928/7/2019Sildenafil Exercise: Role of PDE5 InhibitionMechanisms of Exercise Intolerance in Cystic Fibrosis: Role of PDE5 InhibitionCystic FibrosisDrug: Sildenafil 40mg oral capsule;Drug: Placebo Oral capsuleNational Jewish HealthAugusta University;Cystic Fibrosis FoundationRecruiting9 YearsN/AAll40Phase 2;Phase 3United States
115NCT03506061
(ClinicalTrials.gov)
September 4, 201913/4/2018Trikafta in Cystic Fibrosis PatientsiPS Cell Response to CFTR Modulators: Study of Trikafta in CF Patients Carrying Partial Function MutationsCystic FibrosisDrug: TrikaftaEmory UniversityNational Heart, Lung, and Blood Institute (NHLBI)Recruiting12 YearsN/AAll22Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
116NCT04006873
(ClinicalTrials.gov)
September 3, 20192/7/2019Gut Imaging for Function & Transit in Cystic Fibrosis Study 2A Randomised Crossover Pilot Study of the Effects of Tezacaftor/Ivacaftor and Ivacaftor on Gastrointestinal Function Using Magnetic Resonance Imaging Parameters in People With Cystic FibrosisCystic FibrosisDrug: Tezacaftor/Ivacaftor + Ivacaftor;Drug: Placebo oral tabletNottingham University Hospitals NHS TrustVertex Pharmaceuticals Incorporated;Cystic Fibrosis Foundation;Cystic Fibrosis Trust;University of Nottingham;Nottingham University Hospitals CharityActive, not recruiting12 Years40 YearsAll12Phase 2United Kingdom
117EUCTR2019-000750-63-HU
(EUCTR)
03/09/201901/07/2019Cystic Fibrosis: A Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in subjects who have two copies of the F508del mutationA Phase 2 Study of ABBV-3067 Alone and in Combination with ABBV-2222 in Cystic Fibrosis Subjects Who Are Homozygous for the F508del Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: ABBV-2222
Product Code: ABBV-2222
INN or Proposed INN: Galicaftor
Other descriptive name: ABBV-2222
INN or Proposed INN: Galicaftor
Other descriptive name: ABBV-2222
INN or Proposed INN: Galicaftor
Other descriptive name: ABBV-2222
Product Name: ABBV-3067
Product Code: ABBV-3067
Other descriptive name: ABBV-3067
AbbVie DeutschlandNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
189Phase 2Serbia;France;United States;Czech Republic;Hungary;Slovakia;Puerto Rico;Canada;Belgium;Spain;Netherlands;United Kingdom
118EUCTR2019-000833-37-FR
(EUCTR)
02/09/201912/07/2019A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445 / 50-mg TEZ / 75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
INN or Proposed INN: elexacaftor
Other descriptive name: VX-445
Trade Name: Kalydeco 150 mg film-coated tablets
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
250Phase 3United States;France;Canada;Belgium;Spain;Ireland;Denmark;Australia;Netherlands;Germany;Italy;United Kingdom
119NCT04302428
(ClinicalTrials.gov)
August 29, 20196/3/2020Zinc Status and Growth in Cystic FibrosisZinc Status and Growth in Cystic FibrosisCystic;FibrosisBiological: Serum Zn;Biological: Red Blood Cell ZnIndiana UniversityNULLRecruiting3 Months3 YearsAll38United States
120NCT04058353
(ClinicalTrials.gov)
August 28, 201914/8/2019A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)Cystic FibrosisDrug: ELX/TEZ/IVA;Drug: IVA;Drug: TEZ/IVAVertex Pharmaceuticals IncorporatedNULLCompleted12 YearsN/AAll271Phase 3United States;Australia;Belgium;Canada;Denmark;France;Germany;Ireland;Israel;Italy;Netherlands;Spain;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
121EUCTR2018-003970-28-DE
(EUCTR)
12/08/201921/05/2019A Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic FibrosisA Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Deutivacaftor
Product Code: VX-561
INN or Proposed INN: DEUTIVACAFTOR
Product Name: Deutivacaftor
Product Code: VX-561
INN or Proposed INN: DEUTIVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
88Phase 2United States;Belgium;Ireland;Australia;Netherlands;Germany;United Kingdom
122NCT04043806
(ClinicalTrials.gov)
August 9, 201931/7/2019A Study Evaluating the Long-term Safety of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety of VX-445 Combination Therapy in Subjects With Cystic FibrosisCystic FibrosisDrug: ELX/TEZ/IVA;Drug: IVAVertex Pharmaceuticals IncorporatedNULLActive, not recruiting12 YearsN/AAll458Phase 3United States;Australia;Canada;Denmark;Germany;Ireland;Israel;Poland;Spain;Switzerland;United Kingdom
123NCT03587961
(ClinicalTrials.gov)
August 1, 20193/7/2018Personalized Theratyping TrialPersonalized Theratyping TrialCystic FibrosisDrug: SymdekoUniversity of Alabama at BirminghamNULLRecruiting6 YearsN/AAll20Early Phase 1United States
124NCT04058340
(ClinicalTrials.gov)
July 30, 201912/8/2019Taste Receptors Regulation in CF PatientsThe Effects of Taste Receptors Regulation in Upper Airway Innate Immunity of CF PatientsCystic FibrosisDietary Supplement: lactizole nebulization;Other: PlaceboMedical Universtity of LodzNULLRecruiting6 YearsN/AAll30N/APoland
125EUCTR2019-000261-21-IE
(EUCTR)
26/07/201930/05/2019A 4-week study to test different doses of BI 1265162 in adolescents and adults with cystic fibrosis using the Respimat® inhaler - BALANCE - CF(TM)1A randomised, double-blind, placebo-controlled and parallel group trial to evaluate efficacy and safety of twice daily inhaled doses of BI 1265162 delivered by Respimat® inhaler as add-on therapy to standard of care over 4 weeks in patients with cystic fibrosis - BALANCE - CF(TM)1 Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Boehringer Ingelheim LimitedNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
98Phase 2France;United States;Canada;Belgium;Spain;Ireland;Germany;United Kingdom;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
126EUCTR2019-000261-21-FR
(EUCTR)
19/07/201921/05/2019A 4-week study to test different doses of BI 1265162 in adolescents and adults with cystic fibrosis using the Respimat®inhalerA randomised, double-blind, placebo-controlled and parallel group trial to evaluate efficacy and safety of twice daily inhaled doses of BI 1265162 delivered by Respimat® inhaler as add-on therapy to standard of care over 4 weeks in patients with cystic fibrosis Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: BI 1265162 10 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Product Name: BI 1265162 25 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Product Name: BI 1265162 50 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Product Name: BI 1265162 100 µg
INN or Proposed INN: not yet available
Other descriptive name: BI 1265162
Boehringer Ingelheim FranceNULLNot RecruitingFemale: yes
Male: yes
98Phase 2United States;France;Canada;Spain;Belgium;Ireland;Germany;United Kingdom;Sweden
127EUCTR2017-004134-29-DE
(EUCTR)
12/07/201905/03/2018A Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic FibrosisA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-659/Tezacaftor/Ivacaftor
Product Code: VX-659/ TEZ/IVA
INN or Proposed INN: VX-659
Other descriptive name: VX-659
INN or Proposed INN: TEZACAFTOR
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
460Phase 3United States;Canada;Spain;Poland;Ireland;Denmark;Australia;Israel;Norway;Germany;United Kingdom;Switzerland
128EUCTR2018-002496-18-NL
(EUCTR)
08/07/201913/02/2019A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic FibrosisA Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects Aged 18 Years and Older With Cystic Fibrosis Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-121
INN or Proposed INN: N/A
Other descriptive name: VX-121
Product Name: Tezacaftor
Product Code: VX-661
INN or Proposed INN: TEZACAFTOR
Product Name: VX-561
INN or Proposed INN: N/A
Other descriptive name: VX-561
Trade Name: Symkevi 100mg/150mg Film-coated tablets
Product Name: tezacaftor/ivacaftor 100mg/150mg
Product Code: VX-661/VX-770
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
108Phase 2United States;Portugal;Germany;Netherlands;United Kingdom
129EUCTR2018-003970-28-BE
(EUCTR)
04/07/201923/04/2019A Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic FibrosisA Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Deutivacaftor
Product Code: VX-561
INN or Proposed INN: DEUTIVACAFTOR
Product Name: Deutivacaftor
Product Code: VX-561
INN or Proposed INN: DEUTIVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
88Phase 2United States;Belgium;Ireland;Australia;Netherlands;Germany;United Kingdom
130NCT03650712
(ClinicalTrials.gov)
July 1, 201918/7/2018EnVision CF Multicenter Study of Glucose Tolerance in Cystic FibrosisEnVision CF Multicenter Study of Glucose Tolerance in Cystic FibrosisCystic Fibrosis-related DiabetesDiagnostic Test: Oral glucose tolerance test;Diagnostic Test: Continuous glucose monitoring;Diagnostic Test: Dexa scanKatie Larson OdeNULLRecruiting6 YearsN/AAll144N/AUnited States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
131NCT04302662
(ClinicalTrials.gov)
June 27, 20194/3/2020Phase 2 Combination Study With Escalating Doses of MS1819-SD on Top of a Stable Dose of PPEsA Multicenter, Open-label Phase 2 Study With Escalating Doses of MS1819-SD on Top of a Stable Dose of PPEs, to Investigate the Efficacy and Safety of This Combination for the Compensation of Severe Exocrine Pancreatic Insufficiency in CF Patients Not Fully Compensated With Only PPEsCystic Fibrosis;Cystic Fibrosis Gastrointestinal Disease;Cystic Fibrosis of PancreasDrug: MS1819-SDAzurRx SASNULLRecruiting12 YearsN/AAll24Phase 2Hungary
132NCT03597347
(ClinicalTrials.gov)
June 20, 201912/7/2018Trial of Inhaled Molgramostim in CF Subjects With NTM InfectionAn Open-label, Non-controlled, Multicenter, Pilot Trial, Using Inhaled Molgramostim in Cystic Fibrosis Subjects With Nontuberculous Mycobacterial (NTM) InfectionMycobacterium Infections, Nontuberculous;Cystic Fibrosis (CF)Drug: Molgramostim nebulizer solution;Device: PARI eFlow nebulizer systemSavara Inc.NULLActive, not recruiting18 YearsN/AAll30Phase 2United States
133EUCTR2018-003970-28-GB
(EUCTR)
18/06/201917/04/2019A Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic FibrosisA Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Deutivacaftor
Product Code: VX-561
INN or Proposed INN: DEUTIVACAFTOR
INN or Proposed INN: DEUTIVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: Ivacaftor
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
88Phase 2United States;Belgium;Ireland;Australia;Netherlands;Germany;United Kingdom
134NCT03734744
(ClinicalTrials.gov)
June 17, 20196/11/2018PK/PD of Vitamin D3 in Adults With CFA Pilot Study Evaluating Single, High-dose Pharmacokinetics/Pharmacodynamics of Vitamin D3 in CFVitamin D Deficiency;Cystic FibrosisDietary Supplement: Vitamin D3University of Southern CaliforniaNULLRecruiting18 YearsN/AAll12N/AUnited States
135NCT03485456
(ClinicalTrials.gov)
May 29, 201926/3/2018DPI-Tobra-Kind Cyclops® in Children With Cystic FibrosisPharmacokinetic Evaluation and Tolerability of Dry Powder Tobramycin Via the Cyclops® in Children With Cystic FibrosisCystic FibrosisDrug: TobramycinUniversity Medical Center GroningenNULLRecruiting6 Years18 YearsAll10Phase 1;Phase 2Netherlands
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
136NCT04010799
(ClinicalTrials.gov)
May 27, 201910/6/2019A Clinical Study to Investigate Safety, Tolerability and Distribution of CHF 6333 After One or After Repeated Inhalation in Patients With Cystic Fibrosis (CF) and in Patients With Non Cystic Fibrosis (NCFB) BronchiectasisA Phase Ib, Randomised, Double-blind, Placebo-controlled Study to Investigate the Safety, Tolerability and Pharmacokinetics of Inhaled CHF 6333 After Single and Repeated Ascending Doses in Patients Affected by Cystic Fibrosis and Non Cystic Fibrosis BronchiectasisCystic Fibrosis;Non-Cystic Fibrosis BronchiectasisDrug: CHF 6333;Drug: PlaceboChiesi Farmaceutici S.p.A.NULLRecruiting18 YearsN/AAll48Phase 1Germany
137NCT03965832
(ClinicalTrials.gov)
May 24, 201924/5/2019HFNT During Exercise in CFA Pilot Study to Evaluate the Feasibility of Using High-flow Nasal Therapy During Exercise in Patients With Cystic Fibrosis and Severe Lung DiseaseCystic FibrosisDevice: HFNT during exercise;Device: Standard oxygen therapy/RAThe Leeds Teaching Hospitals NHS TrustNULLRecruiting18 YearsN/AAll20N/AUnited Kingdom
138EUCTR2018-002496-18-PT
(EUCTR)
20/05/201905/02/2019A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic FibrosisA Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects Aged 18 Years and Older With Cystic Fibrosis Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-121
INN or Proposed INN: N/A
Other descriptive name: VX-121
Product Name: Tezacaftor
Product Code: VX-661
INN or Proposed INN: TEZACAFTOR
Product Name: VX-561
INN or Proposed INN: N/A
Other descriptive name: VX-561
Trade Name: Symkevi 100mg/150mg Film-coated tablets
Product Name: tezacaftor/ivacaftor 100mg/150mg
Product Code: VX-661/VX-770
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
108Phase 2United States;Portugal;Netherlands;Germany;United Kingdom
139NCT03822455
(ClinicalTrials.gov)
May 16, 201929/1/2019A Phase 2b Randomised, Placebo Controlled Study of OligoG in Patients With Cystic FibrosisA Phase 2b Randomised, Double-blind, Parallel-group Study of Alginate Oligosaccharide (OligoG) Dry Powder Inhalation in Addition to Standard of Care Compared to Placebo in Addition to Standard of Care in Patients With Cystic Fibrosis (CF)Cystic FibrosisDrug: OligoG DPIAlgiPharma ASAlgiPharma Australia Pty. Ltd.Active, not recruiting18 YearsN/AAll20Phase 2Australia
140EUCTR2017-003723-29-PL
(EUCTR)
09/05/201907/11/2018A study of safety and efficacy of lenabasum in Cystic FibrosisA Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - A Phase 2 safety and efficacy study of Lenabasum in Cystic Fibrosis Cystic Fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
415Phase 2Portugal;Serbia;United States;Slovakia;Greece;Spain;Austria;Russian Federation;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Romania;Bulgaria;Netherlands;Germany;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
141EUCTR2019-001314-41-NL
(EUCTR)
06/05/201906/05/2019This study investigates the effect of switching from Orkambi treatment to Symkevi. In particuar we want to investigate the effect of the switch on the uptake, concentration in the blood an degradation of ivacaftor.Kinetics of ivacaftor at Switch Orkambi Symkevi study - SOS Cystic fibrosis;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Trade Name: Orkambi: contains lumacaftor and ivacaftor
Product Name: Orkambi
Trade Name: Symkevi
Product Name: Symkevi
Trade Name: Kalydeco
Product Name: Kalydeco
NULLNot RecruitingFemale: yes
Male: yes
100Phase 4Netherlands
142EUCTR2018-002496-18-DE
(EUCTR)
02/05/201928/01/2019A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic FibrosisA Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects Aged 18 Years and Older With Cystic Fibrosis Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
108Phase 2Portugal;United States;Netherlands;Germany;United Kingdom
143NCT03961516
(ClinicalTrials.gov)
May 1, 201916/5/2019Glycemic Characterization and Pancreatic Imaging Correlates in Cystic FibrosisGlycemic Characterization and Pancreatic Imaging Correlates in Cystic FibrosisCystic Fibrosis;Cystic Fibrosis-related Diabetes;Cystic Fibrosis Liver Disease;Pancreatic Steatosis;Hepatic SteatosisDevice: Frequently Sampled Oral Glucose Tolerance Test and CGM;Radiation: MRI Pancreas and LiverUniversity Hospitals Cleveland Medical CenterNULLRecruiting6 Years40 YearsAll40United States
144NCT02748798
(ClinicalTrials.gov)
May 201919/4/2016Developing Optimal Parameters for Hyperpolarized Noble Gas and Inert Fluorinated Gas MRI of Lung DisordersDeveloping Optimal Parameters for Hyperpolarized Noble Gas (3He and 129Xe) and Inert Fluorinated Gas Magnetic Resonance Imaging of Lung DisordersLung Transplant;Lung Resection;Lung Cancer;Asthma;Cystic Fibrosis;Chronic Obstructive Pulmonary Disease;Emphysema;Mesothelioma;Asbestosis;Pulmonary Embolism;Interstitial Lung Disease;Pulmonary Fibrosis;Bronchiectasis;Seasonal Allergies;Cold Virus;Lung Infection;Pulmonary Hypertension;Pulmonary Dysplasia;Obstructive Sleep ApneaDrug: HP 3He;Drug: HP 129Xe;Drug: PFP;Drug: SF6;Device: 129Xe Small and Large Human Lung Coil;Device: 3He Human Lung Coil;Device: PFP and SF6 Human Lung CoilThunder Bay Regional Research InstituteThunder Bay Regional Health Sciences Centre;St. Joseph's Care Group;Lakehead UniversityUnknown status18 YearsN/AAll160Early Phase 1Canada
145NCT03903913
(ClinicalTrials.gov)
May 201915/2/2019Safety, Tolerability and Efficacy of S-1226 in Cystic FibrosisA Phase IIa Open Label Study to Evaluate the Safety, Tolerability and Efficacy of S-1226 Administered by Nebulization in Subjects With Mild to Moderate Cystic FibrosisCystic FibrosisDrug: S-1226SolAeroMed Inc.NULLRecruiting14 Years50 YearsAll12Phase 2Canada
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
146NCT03912233
(ClinicalTrials.gov)
April 30, 201910/4/2019A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic FibrosisA Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects Aged 18 Years and Older With Cystic FibrosisCystic FibrosisDrug: VX-121;Drug: TEZ;Drug: VX-561;Drug: TEZ/IVA;Drug: IVA;Drug: PlaceboVertex Pharmaceuticals IncorporatedNULLCompleted18 YearsN/AAll87Phase 2United States;Germany;Netherlands;Portugal;United Kingdom
147NCT03911713
(ClinicalTrials.gov)
April 17, 20199/4/2019A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic FibrosisA Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic FibrosisCystic FibrosisDrug: VX-561;Drug: IVA;Drug: PlaceboVertex Pharmaceuticals IncorporatedNULLCompleted18 YearsN/AAll77Phase 2United States;Australia;Belgium;Germany;Ireland;Netherlands;United Kingdom
148NCT03795363
(ClinicalTrials.gov)
April 10, 201928/12/2018Orkambi Treatment in 2 to 5 Year Old Children With CFNutritional Impact of Orkambi Treatment in 2 to 5 Year Old Children Homozygous for F508del MutationsCystic FibrosisDrug: OrkambiChildren's Hospital of PhiladelphiaVertex Pharmaceuticals IncorporatedRecruiting2 Years5 YearsAll32United States
149NCT03870841
(ClinicalTrials.gov)
April 3, 201924/10/2018The Effect of PC945 on Aspergillus Fumigatus Lung Infection in Patients With Cystic FibrosisAn Open-label Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of Inhaled PC945 in Adult Cystic Fibrosis (CF) Patients With Persistent Pulmonary Aspergillus Fumigatus InfectionAspergillosis;Cystic FibrosisDrug: PC945Pulmocide LtdNULLTerminated18 YearsN/AAll4Phase 2United Kingdom
150NCT03710538
(ClinicalTrials.gov)
April 1, 201920/6/2018The Effect of a Pre-meal Snack and/or Exercise on Post-prandial Glycemic Excursions in Adults With Cystic FibrosisThe Effect of a Pre-meal Snack and/or Exercise on Post-prandial Glycemic Excursions in Adults With Cystic FibrosisCystic FibrosisDietary Supplement: Snack beverage;Behavioral: ExerciseInstitut de Recherches Cliniques de MontrealNULLRecruiting18 YearsN/AAll14N/ACanada
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
151NCT03724955
(ClinicalTrials.gov)
April 201929/10/2018Pilot E2 for Hypogonadal Women With CFBDPilot Study Evaluating the Effects of Estradiol for Hypogonadal Women With Cystic Fibrosis and OsteopeniaCystic Fibrosis Related Bone DiseaseDrug: Estradiol 2 mg;Other: PlaceboEmory UniversityNULLWithdrawn18 YearsN/AFemale0Phase 4United States
152EUCTR2018-003831-31-HU
(EUCTR)
28/03/201909/10/2018MS1819-SD phase II clinical trial for Exocrine Pancreatic Insufficiency caused by Cystic Fibrosis.A multicenter, open-label Phase 2 study with escalating doses of MS1819-SD on top of a stable dose of PPEs, to investigate the efficacy and safety of this combination for the compensation of severe exocrine pancreatic insufficiency in CF patients not fully compensated with only PPEs - MS1819/18/02 Exocrine Pancreatic Insufficiency
MedDRA version: 20.0;Level: LLT;Classification code 10033628;Term: Pancreatic insufficiency;System Organ Class: 100000004856 ;Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
AzurRxNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
24Phase 2Hungary
153EUCTR2017-001259-29-GB
(EUCTR)
12/03/201921/12/2018A study to establish the tolerabilty and adverse effects in people with a severe form of Cystic Fibrosi (delta F 508 homozygous) when using a combination of two products - cyteamine (a licensed medicine being used in a new disease) and EGCG (a food supplement).A PHASE 1B/2A STUDY TO ASSESS THE TOLERABILITY AND ADVERSE EFFECT PROFILE OF CYSTEAMINE (CYSTAGON) IN ADULTS AND CHILDREN WITH HOMOZYGOUS ?F508 CYSTIC FIBROSIS, IN PATIENTS TAKING EPIGALLOCATECHIN-3-GALLATE (EGCG, EPINERVE) FOOD SUPPLEMENTATION - Delta Dose Study DeltaF508 Homozygous Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Cystagon
Product Name: Cystagon
INN or Proposed INN: cysteamine bitartrate
Other descriptive name: 2-aminoethanethiol;2,3-dihydroxybutanedioic acid
University of LIverpoolNULLNot RecruitingFemale: yes
Male: yes
44Phase 1;Phase 2United Kingdom
154EUCTR2018-002496-18-GB
(EUCTR)
08/03/201930/01/2019A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic FibrosisA Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects Aged 18 Years and Older With Cystic Fibrosis Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-121
INN or Proposed INN: N/A
Other descriptive name: VX-121
Product Name: Tezacaftor
Product Code: VX-661
INN or Proposed INN: TEZACAFTOR
Product Name: VX-561
INN or Proposed INN: N/A
Other descriptive name: VX-561
Trade Name: Symkevi 100mg/150mg Film-coated tablets
Product Name: tezacaftor/ivacaftor 100mg/150mg
Product Code: VX-661/VX-770
INN or Proposed INN: TEZACAFTOR
Other descriptive name: TEZ
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVA
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
108Phase 2Portugal;United States;Netherlands;Germany;United Kingdom
155NCT03902236
(ClinicalTrials.gov)
March 1, 20191/4/2019Reaction Time and Postural Control in Individuals With Cystic Fibrosis and BronchiectasisEvaluation of Reaction Time and Postural Control in Individuals With Cystic Fibrosis and BronchiectasisCystic Fibrosis;BronchiectasisOther: Reaction Time and Postural Control;Other: Muscle oxygenation;Other: Exercise capacity;Other: BalanceHacettepe UniversityNULLRecruiting6 Years18 YearsAll60Turkey
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
156EUCTR2018-000185-11-AT
(EUCTR)
27/02/201919/10/2018A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Trikafta
Product Name: 100 mg ELX/50 mg TEZ /75 mg IVA FDC
Product Code: ELX/TEZ/IVA
INN or Proposed INN: Elexacaftor
Other descriptive name: VX-445
INN or Proposed INN: Tezacaftor
Other descriptive name: VX-661
INN or Proposed INN: Ivacaftor
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
460Phase 3United States;Greece;Austria;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
157EUCTR2017-003723-29-NL
(EUCTR)
25/02/201920/06/2018A study of safety and efficacy of lenabasum in Cystic FibrosisA Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - A Phase 2 safety and efficacy study of Lenabasum in Cystic Fibrosis Cystic Fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: LENABASUM
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: LENABASUM
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
415Phase 2United States;Serbia;Portugal;Slovakia;Greece;Spain;Réunion;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland;Romania;Bulgaria;Netherlands;Germany;Sweden
158NCT03632525
(ClinicalTrials.gov)
February 22, 201921/6/2018Intravenous Iron in Adults With Cystic FibrosisA Pilot Trial of Intravenous Iron for the Treatment of Iron Deficiency in Adult Patients With Cystic FibrosisCystic Fibrosis;Iron-deficiencyDrug: Ferric carboxymaltoseUniversity of OxfordNULLRecruiting18 YearsN/AAll20Phase 4United Kingdom
159NCT03783286
(ClinicalTrials.gov)
February 6, 201918/12/2018Ivacaftor Treatment in 6 Month to 2 Year Old CF SubjectsNutritional Impact of Ivacaftor Treatment in 6 Month to 2 Year Old Children With CF Gating MutationsCystic FibrosisDrug: IvacaftorChildren's Hospital of PhiladelphiaVertex Pharmaceuticals IncorporatedRecruiting6 Months2 YearsAll18United States
160EUCTR2017-002533-32-BE
(EUCTR)
17/01/201928/09/2018A Study to Assess the Efficacy and Safety of Study Drug INS1007 Administered Once Daily in Patients with Non-Cystic Fibrosis Bronchiectasis.A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Center Study to Assess the Efficacy, Safety and Tolerability, and Pharmacokinetics of INS1007 Administered Once Daily for 24 Weeks in Subjects with Non-Cystic Fibrosis Bronchiectasis - The Willow Study. Non-Cystic Fibrosis Bronchiectasis
MedDRA version: 20.0;Level: SOC;Classification code 10038738;Term: Respiratory, thoracic and mediastinal disorders;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
MedDRA version: 20.1;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Insmed IncorporatedNULLNot Recruiting Female: yes
Male: yes
240Phase 2United States;Spain;United Kingdom;Italy;Belgium;Poland;Singapore;Australia;Denmark;Bulgaria;Germany;Netherlands;New Zealand;Sweden;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
161NCT03746483
(ClinicalTrials.gov)
January 10, 201915/11/2018OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic FibrosisA Phase 2, Open-Label, Multicenter, 2x2 Crossover Trial to Assess the Safety and Efficacy of MS1819-SD in Patients With Exocrine Pancreatic Insufficiency Due to Cystic FibrosisExocrine Pancreatic Insufficiency (EPI);Cystic Fibrosis (CF)Drug: MS1819-SD;Drug: Porcine PERTAzurRx BioPharma, Inc.NULLCompleted18 YearsN/AAll32Phase 2United States;Poland
162NCT04589897
(ClinicalTrials.gov)
January 7, 201924/9/2020Manuka Honey Sinus Rinse StudyA Pilot Study Investigating the Effects of a Manuka Honey Sinus Rinse on Sino-nasal Outcome Test Scores in Cystic Fibrosis PatientsCystic Fibrosis;Infection, BacterialDevice: Manuka honey sinus rinse;Device: Standard sinus rinseSwansea UniversityUniversity Hospital of WalesSuspended18 YearsN/AAll40N/AUnited Kingdom
163EUCTR2018-000185-11-GR
(EUCTR)
20/12/201828/11/2018A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Trikafta
Product Name: 100 mg ELX/50 mg TEZ /75 mg IVA FDC
Product Code: ELX/TEZ/IVA
INN or Proposed INN: Elexacaftor
Other descriptive name: VX-445
INN or Proposed INN: Tezacaftor
Other descriptive name: VX-661
INN or Proposed INN: Ivacaftor
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
460Phase 3United States;Greece;Austria;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
164EUCTR2015-004143-39-IT
(EUCTR)
19/12/201820/09/2018Saline hypertonic in preschoolers and lung structure as measured by computed tomography.A Phase 3 randomised, double-blind, controlled trial of inhaled 7% hypertonic saline versus 0.9% isotonic saline for 48 weeks in patients with Cystic Fibrosis at 3-6 years of age in parallel with the North American SHIP clinical trial - Ship-CT study Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011763;Term: Cystic fibrosis lung;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Erasmus MCNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
120Phase 3France;United States;Spain;Belgium;Denmark;Australia;Netherlands;Italy
165EUCTR2015-002743-33-NL
(EUCTR)
14/12/201818/07/2018A clinical trial to investigate the efficacy and safety of inhaled colistimethate sodium in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa)A double-blind, placebo-controlled, multi-centre, clinical trial to investigate the efficacy and safety of 12 months of therapy with inhaled colistimethate sodium in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa) - PROMIS I Non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa
MedDRA version: 21.0;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders ;Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]
Zambon S.p.A.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
420Phase 3Portugal;Greece;Belgium;Spain;Australia;Israel;Germany;Netherlands;New Zealand;Italy;United Kingdom;Switzerland
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
166NCT03647228
(ClinicalTrials.gov)
December 13, 201823/8/2018A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients With Cystic FibrosisA Double-Blind, Placebo-Controlled, Dose-Escalation, Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION-827359, an Antisense Oligonucleotide Inhibitor of ENaC, Administered to Healthy Volunteers and Patients With Cystic FibrosisHealthy Subjects;Cystic FibrosisDrug: IONIS-ENaCRx;Drug: PlaceboIonis Pharmaceuticals, Inc.NULLActive, not recruiting18 Years50 YearsAll98Phase 1Germany;United Kingdom
167NCT03669614
(ClinicalTrials.gov)
December 7, 201830/8/2018SAD and MAD of Inhaled AR-501 in Health Adults and P. Aeruginosa Infected Cystic Fibrosis SubjectsA P1/2a Randomized, Double-Blind, Two-Part, Dose-Ascending, Multicenter Study of the Safety and PK of AR-501 (Gallium Citrate), Administered Via Inhalation, in Healthy Adult and P. Aeruginosa Infected Cystic Fibrosis SubjectsCystic FibrosisDrug: inhaled AR-501;Drug: inhaled AR-501 placeboAridis Pharmaceuticals, Inc.NULLActive, not recruiting18 Years49 YearsAll96Phase 1;Phase 2United States
168NCT02323100
(ClinicalTrials.gov)
December 2, 201818/12/2014Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis)A Double Blind, Placebo Controlled, Dose Escalation Trial of Glycerol Phenylbutyrate Corrector Therapy for Cystic FibrosisCystic FibrosisDrug: Ravicti low dose;Drug: Ravicti high dose;Drug: PlaceboNational Jewish HealthUniversity of Alabama at Birmingham;Children's Hospital of Philadelphia;Johns Hopkins University;Horizon Pharma Ireland, Ltd., Dublin IrelandSuspended18 YearsN/AAll36Phase 1;Phase 2United States
169NCT03624101
(ClinicalTrials.gov)
December 1, 20187/8/2018Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon MutationsNovel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon MutationsCystic FibrosisDrug: Tezacaftor/IvacaftorUniversity of Alabama at BirminghamNULLRecruiting18 YearsN/AAll5Phase 4United States
170EUCTR2017-003723-29-CZ
(EUCTR)
27/11/201809/07/2018A study of safety and efficacy of lenabasum in Cystic FibrosisA Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - A Phase 2 safety and efficacy study of Lenabasum in Cystic Fibrosis Cystic Fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
415Phase 2Portugal;United States;Serbia;Slovakia;Greece;Spain;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland;Romania;Bulgaria;Germany;Netherlands;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
171NCT03756922
(ClinicalTrials.gov)
November 27, 201827/11/2018A DDI Study of FDL169 and FDL176 in Healthy SubjectsA Phase 1/2, Drug-Drug Interaction Study of FDL169 and FDL176 in Healthy Subjects and in Cystic Fibrosis Subjects Homozygous for the F508del-CFTR MutationCystic FibrosisDrug: FDL169;Drug: FDL176Flatley Discovery Lab LLCNULLSuspended18 Years55 YearsAll78Phase 1;Phase 2United Kingdom
172EUCTR2015-002743-33-GR
(EUCTR)
27/11/201814/11/2018A clinical trial to investigate the efficacy and safety of inhaled colistimethate sodium in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa)A double-blind, placebo-controlled, multi-centre, clinical trial to investigate the efficacy and safety of 12 months of therapy with inhaled colistimethate sodium in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa) - PROMIS I Non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa
MedDRA version: 21.0;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]
Trade Name: Promixin/Tadim
Product Name: Colistimethate sodium
INN or Proposed INN: COLISTIMETHATE SODIUM
Zambon S.p.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
420Phase 3Portugal;Greece;Spain;Israel;Switzerland;United Kingdom;Italy;France;Belgium;Australia;Germany;Netherlands;New Zealand
173EUCTR2017-003723-29-PT
(EUCTR)
19/11/201821/03/2018A study of safety and efficacy of lenabasum in Cystic FibrosisA Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - A Phase 2 safety and efficacy study of Lenabasum in Cystic Fibrosis Cystic Fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: LENABASUM
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: LENABASUM
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
415Phase 2United States;Serbia;Portugal;Slovakia;Greece;Spain;Réunion;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Poland;Belgium;Romania;Australia;Bulgaria;Germany;Netherlands;Sweden
174EUCTR2018-000243-87-GB
(EUCTR)
19/11/201821/08/2018A study in people with cystic fibrosis who have a fungal lung infection, looking at the safety of inhaled PC945, it's effect on the body and how the body affects the drug.An open-label study to assess the safety, pharmacokinetics and pharmacodynamics of inhaled PC945 in adult Cystic Fibrosis (CF) patients with persistent pulmonary Aspergillus fumigatus infection. Pulmonary aspergillosis
MedDRA version: 20.0;Level: LLT;Classification code 10059259;Term: Pulmonary aspergillosis;System Organ Class: 100000004862;Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]
Product Name: PC945Pulmocide LtdNULLNot RecruitingFemale: yes
Male: yes
18Phase 2United Kingdom
175EUCTR2017-003723-29-DE
(EUCTR)
19/11/201814/02/2018A study of safety and efficacy of lenabasum in Cystic FibrosisA Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - A Phase 2 safety and efficacy study of Lenabasum in Cystic Fibrosis Cystic Fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: LENABASUM
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: LENABASUM
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
415Phase 2Romania;Bulgaria;Germany;Netherlands;Sweden;United States;Serbia;Portugal;Slovakia;Greece;Spain;Réunion;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
176EUCTR2018-000185-11-NL
(EUCTR)
14/11/201819/09/2018A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Trifakta
Product Name: 100 mg ELX/50 mg TEZ /75 mg IVA FDC
Product Code: ELX/TEZ/IVA
INN or Proposed INN: Elexacaftor
Other descriptive name: VX-445
INN or Proposed INN: Tezacaftor
Other descriptive name: VX-661
INN or Proposed INN: Ivacaftor
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
460Phase 3United States;Czechia;Greece;Austria;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Netherlands;Germany;Sweden
177EUCTR2017-003319-21-BE
(EUCTR)
08/11/201821/08/2018Study Assessing the Safety, Tolerability and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults with Cystic FibrosisA Phase 1 / 2 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults with Cystic Fibrosis Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: N-(5-hydroxy-2,4-bis(trimethylsilyl)phenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide
Product Code: PTI-808
INN or Proposed INN: None
Other descriptive name: PTI-808
Product Name: Sodium (R)-8-methyl-2-(3-methylbenzofuran-2-yl)-5-(1-(tetrahydro-2H-pyran-4-yl)ethoxy)quinoline-4-ca
Product Code: PTI-801
INN or Proposed INN: None
Other descriptive name: PTI-801
Product Name: N-(trans-3-(5-((R)-1-hydroxyethyl)-1,3,4-oxadiazol-2-yl)cyclobutyl)-3-phenylisoxazole-5-carboxamide
Product Code: PTI-428
INN or Proposed INN: None
Other descriptive name: PTI-428
Proteostasis Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
240Phase 1;Phase 2United States;France;Canada;Belgium;Denmark;Germany;United Kingdom
178NCT03748199
(ClinicalTrials.gov)
November 8, 20185/11/2018Clinical Study to Investigate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of POL6014 in Patients With CFPhase-Ib/IIa Study to Investigate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Orally Inhaled Multiple Doses of POL6014 in Patients With Cystic FibrosisCystic FibrosisDrug: POL6014;Drug: PlaceboSanthera PharmaceuticalsNULLUnknown status18 Years55 YearsAll40Phase 1;Phase 2Germany
179NCT03617718
(ClinicalTrials.gov)
November 1, 201819/7/2018Project 2 Airway Potential Hydrogen (pH) in AsthmaMethods to Identify and Treat Severe Asthma Patients Project 2: Airway pH PhenotypingCystic Fibrosis;Asthma;Severe Persistent Asthma;HealthyDrug: Glycine BufferUniversity Hospitals Cleveland Medical CenterNational Institutes of Health (NIH)Recruiting18 Years50 YearsAll75Phase 1;Phase 2United States
180EUCTR2017-002968-40-PL
(EUCTR)
31/10/201829/06/2018A Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in subjects with cistic fibrosisA Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation - Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Subjects Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-661/ivacaftor 50 mg/ 75 mg
INN or Proposed INN: TEZACAFTOR
Other descriptive name: VRT-893661
INN or Proposed INN: IVACAFTOR
Other descriptive name: VX-770
Product Name: VX-661/ivacaftor 100 mg/ 150 mg
INN or Proposed INN: TEZACAFTOR
Other descriptive name: VRT-893661
INN or Proposed INN: IVACAFTOR
Other descriptive name: VX-770
Product Name: Ivacaftor
Product Code: VX-770, VRT-813077
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150mg
Product Name: Ivacaftor
Product Code: VX-770, VRT-813077
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
121Phase 3France;United States;Canada;Belgium;Poland;Ireland;Denmark;Australia;Germany;United Kingdom;Switzerland
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
181EUCTR2018-000185-11-GB
(EUCTR)
19/10/201804/07/2018A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Trikafta
Product Name: 100 mg ELX/50 mg TEZ /75 mg IVA FDC
Product Code: ELX/TEZ/IVA
INN or Proposed INN: Elexacaftor
Other descriptive name: VX-445
INN or Proposed INN: Tezacaftor
Other descriptive name: VX-661
INN or Proposed INN: Ivacaftor
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
460Phase 3United States;Greece;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
182EUCTR2018-000185-11-CZ
(EUCTR)
15/10/201809/07/2018A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
460Phase 3United States;Greece;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
183EUCTR2017-003723-29-GR
(EUCTR)
15/10/201808/08/2018A study of safety and efficacy of lenabasum in Cystic FibrosisA Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - A Phase 2 safety and efficacy study of Lenabasum in Cystic Fibrosis Cystic Fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
415Phase 2Portugal;United States;Serbia;Slovakia;Greece;Spain;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland;Romania;Bulgaria;Germany;Netherlands;Sweden
184NCT03265288
(ClinicalTrials.gov)
October 10, 201821/2/2017Study of LAU-7b in the Treatment of Cystic Fibrosis in AdultsAPPLAUD: A Double-Blind, Randomized, Placebo-Controlled, Phase II Study of the Efficacy and Safety of LAU-7b in the Treatment of Cystic Fibrosis in AdultsCystic FibrosisDrug: LAU-7b;Drug: Placebo oral capsuleLaurent Pharmaceuticals Inc.Cystic Fibrosis FoundationRecruiting18 YearsN/AAll136Phase 2United States;Australia;Canada
185NCT03525574
(ClinicalTrials.gov)
October 9, 20182/5/2018A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del MutationCystic FibrosisDrug: VX-445;Drug: TEZ;Drug: IVAVertex Pharmaceuticals IncorporatedNULLActive, not recruiting12 YearsN/AAll507Phase 3United States;Australia;Austria;Belgium;Canada;Czechia;France;Germany;Greece;Italy;Netherlands;Sweden;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
186NCT03691779
(ClinicalTrials.gov)
October 2, 201828/9/2018Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of AgeA Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-445/TEZ/IVA Triple Combination Therapy in Cystic Fibrosis Subjects 6 Through 11 Years of AgeCystic FibrosisDrug: VX-445;Drug: TEZ;Drug: IVAVertex Pharmaceuticals IncorporatedNULLCompleted6 Years11 YearsAll66Phase 3United States;Australia;Ireland;United Kingdom
187EUCTR2018-000183-28-CZ
(EUCTR)
01/10/201809/07/2018A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445/50-mg TEZ/75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: Tezacaftor
Other descriptive name: VX-661
INN or Proposed INN: Ivacaftor
INN or Proposed INN: VX-445
Other descriptive name: VX-445
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: Ivacaftor
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
360Phase 3United States;Greece;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
188NCT03956589
(ClinicalTrials.gov)
October 1, 20182/5/2019Functional Respiratory Imaging and Orkambi in CFFunctional Respiratory Imaging (FRI) to Assess the Short-term Effect of the Product ORKAMBI (Lumacaftor/ Ivacaftor) on Lung Function in ORKAMBInaive Patients With Cystic Fibrosis Homozygous for Phe508delCystic FibrosisDrug: OrkambiUniversity Hospital, AntwerpNULLTerminated12 YearsN/AAll12Phase 4Belgium
189EUCTR2018-000183-28-GR
(EUCTR)
01/10/201825/07/2018A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
360Phase 3United States;Greece;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
190EUCTR2018-000184-89-GB
(EUCTR)
27/09/201804/07/2018A Phase 3 Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F).A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100mg VX-445/50mg TEZ /75mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: VX-445
Other descriptive name: VX-445
INN or Proposed INN: tezacaftor
Other descriptive name: VX-661
INN or Proposed INN: ivacaftor
Product Name: 100 mg TEZ/150 mg IVA FDC
Product Code: TEZ/IVA
INN or Proposed INN: tezacaftor
Other descriptive name: VX-661
INN or Proposed INN: ivacaftor
Trade Name: Kalydeco 150 mg film-coated tablets
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
100Phase 3United States;Belgium;Netherlands;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
191EUCTR2018-000183-28-GB
(EUCTR)
27/09/201804/07/2018A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445/50-mg TEZ/75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: Tezacaftor
Other descriptive name: VX-661
INN or Proposed INN: Ivacaftor
INN or Proposed INN: VX-445
Other descriptive name: VX-445
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: Ivacaftor
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
360Phase 3United States;Greece;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
192EUCTR2016-004479-35-PL
(EUCTR)
26/09/201817/05/2018A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 through 11 Years With Cystic FibrosisA Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation - A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 through 11 Years With Cystic F Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
65Phase 3France;Belgium;Poland;Ireland;Denmark;Australia;Germany;United Kingdom;Switzerland
193EUCTR2017-004134-29-PL
(EUCTR)
24/09/201813/07/2018A Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic FibrosisA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
460Phase 3United States;Canada;Spain;Poland;Ireland;Denmark;Australia;Israel;Norway;Germany;United Kingdom;Switzerland
194EUCTR2017-003723-29-BG
(EUCTR)
21/09/201808/05/2018A study of safety and efficacy of lenabasum in Cystic FibrosisA Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - A Phase 2 safety and efficacy study of Lenabasum in Cystic Fibrosis Cystic Fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
415Phase 2Serbia;Portugal;United States;Slovakia;Greece;Spain;Austria;Russian Federation;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Romania;Bulgaria;Netherlands;Germany;Sweden
195EUCTR2018-000183-28-DE
(EUCTR)
20/09/201803/07/2018A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
360Phase 3United States;Greece;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
196EUCTR2018-002550-71-DE
(EUCTR)
12/09/201818/07/2018A clinical study to investigate safety, tolerability and dose of orally inhaled multiple doses of POL6014 in patients with Cystic FibrosisPhase-Ib/IIa study to investigate safety, tolerability, pharmacokinetics and pharmacodynamics of orally inhaled multiple doses of POL6014 in patients with Cystic Fibrosis Cystic Fibrosis;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]Product Name: POL6014
INN or Proposed INN: lonodelestat
Other descriptive name: CYCLO[L-ALANYL-L-SERYL-L-ISOLEUCYL-L-PROLYL-L-PROLYL-L-GLUTAMINYL-L-LYSYL-L-TYROSYL-D-PROLYL-L-PROLYL-(2S)-2-AMINODECANOYL-L-ALPHA-GLUTAMYL-L-THREONYL]
Santhera Pharmaceuticals (Switzerland) LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
32Phase 1;Phase 2Germany
197EUCTR2018-000184-89-NL
(EUCTR)
12/09/201816/08/2018A Phase 3 Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F).A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
100Phase 3United States;Belgium;Netherlands;United Kingdom
198EUCTR2018-000184-89-BE
(EUCTR)
07/09/201809/07/2018A Phase 3 Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F).A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
100Phase 3United States;Belgium;Netherlands;United Kingdom
199EUCTR2018-000183-28-BE
(EUCTR)
07/09/201809/07/2018A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
360Phase 3United States;Greece;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
200EUCTR2018-000185-11-BE
(EUCTR)
07/09/201817/07/2018A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Trikafta
Product Name: 100 mg ELX /50 mg TEZ /75 mg IVA FDC
Product Code: ELX/TEZ/IVA
INN or Proposed INN: Elexacaftor
Other descriptive name: VX-445
INN or Proposed INN: Tezacaftor
Other descriptive name: VX-661
INN or Proposed INN: Ivacaftor
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
460Phase 3United States;Greece;Austria;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
201EUCTR2018-000183-28-NL
(EUCTR)
03/09/201818/07/2018A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445/50-mg TEZ/75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: Tezacaftor
Other descriptive name: VX-661
INN or Proposed INN: Ivacaftor
INN or Proposed INN: VX-445
Other descriptive name: VX-445
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: Ivacaftor
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
360Phase 3United States;Greece;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Australia;Netherlands;Germany;Sweden
202EUCTR2017-004134-29-DK
(EUCTR)
29/08/201803/07/2018A Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic FibrosisA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-659/Tezacaftor/Ivacaftor
Product Code: VX-659/ TEZ/IVA
INN or Proposed INN: VX-659
Other descriptive name: VX-659
INN or Proposed INN: TEZACAFTOR
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
460Phase 3United States;Canada;Spain;Poland;Ireland;Australia;Denmark;Israel;Norway;Germany;United Kingdom;Switzerland
203EUCTR2017-002533-32-DK
(EUCTR)
28/08/201809/11/2017A Study to Assess the Efficacy and Safety of Study Drug INS1007 Administered Once Daily in Patients with Non-Cystic Fibrosis Bronchiectasis.A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Center Study to Assess the Efficacy, Safety and Tolerability, and Pharmacokinetics of INS1007 Administered Once Daily for 24 Weeks in Subjects with Non-Cystic Fibrosis Bronchiectasis - The Willow Study. Non-Cystic Fibrosis Bronchiectasis
MedDRA version: 20.0;Level: SOC;Classification code 10038738;Term: Respiratory, thoracic and mediastinal disorders;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
MedDRA version: 21.0;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Insmed IncorporatedNULLNot Recruiting Female: yes
Male: yes
240Phase 2United States;Spain;United Kingdom;Italy;Belgium;Poland;Singapore;Denmark;Australia;Bulgaria;Germany;Netherlands;New Zealand;Sweden;Korea, Republic of
204EUCTR2018-000183-28-SE
(EUCTR)
27/08/201829/06/2018A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445/50-mg TEZ/75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: Tezacaftor
Other descriptive name: VX-661
INN or Proposed INN: Ivacaftor
INN or Proposed INN: VX-445
Other descriptive name: VX-445
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: Ivacaftor
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
360Phase 3United States;Greece;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
205EUCTR2018-002579-16-SE
(EUCTR)
26/08/201816/07/2018Measurement of the posaconazole concentration in exhaled breath in CF patients after a single dose posaconazole to correlate to the concentration in blood and saliva.Evaluation of the possible use of analysis of posaconazole in exhaled breath as a surrogate marker for the lung to monitor adequate dosages of posaconazole in CF patients treated for Aspergillus spp. related lung-disease. Part 1. Pharmacokinetic single center study. Cystic Fibrosis (CF) is the most common life–limiting autosomal recessive disease among people of European heritage. The condition is a result of a mutation in the cystic fibrosis transmembrane conductance regulator (cftr) gene on chromosome seven, which encodes a chloride channel. In the lung defective channel activity leads to thick, viscous secretion and impaired mucociliary clearance. This causes trapping of mucus, colonization with bacteria and fungi, and a persistent inflammatory response.
MedDRA version: 20.0;Level: PT;Classification code 10074549;Term: Cystic fibrosis respiratory infection suppression;System Organ Class: 10042613 - Surgical and medical procedures;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Noxafil® Karolinska University Hospital, Stockholm CF centerNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Phase 2Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
206EUCTR2018-000183-28-AT
(EUCTR)
24/08/201820/07/2018A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: 100-mg VX-445/50-mg TEZ/75-mg IVA FDC
Product Code: VX-445/TEZ/IVA
INN or Proposed INN: Tezacaftor
Other descriptive name: VX-661
INN or Proposed INN: Ivacaftor
INN or Proposed INN: VX-445
Other descriptive name: VX-445
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: Ivacaftor
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
360Phase 3United States;Greece;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
207EUCTR2018-002366-39-GB
(EUCTR)
23/08/201820/06/2019A pilot trial of intravenous iron for the treatment of low iron levels in adult patients with cystic fibrosis.A pilot trial of intravenous iron for the treatment of iron deficiency in adult patients with cystic fibrosis - Intravenous iron in adults with cystic fibrosis Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
University of Oxford / Clinical Trials and Research GovernanceNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
20Phase 4United Kingdom
208NCT03601637
(ClinicalTrials.gov)
August 23, 201818/7/2018Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508delA Phase 3, 2-part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508delCystic FibrosisDrug: LUM;Drug: IVAVertex Pharmaceuticals IncorporatedNULLRecruiting12 Months23 MonthsAll40Phase 3United States;Canada
209EUCTR2017-004134-29-GB
(EUCTR)
22/08/201823/02/2018A Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic FibrosisA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-659/Tezacaftor/Ivacaftor
Product Code: VX-659/ TEZ/IVA
INN or Proposed INN: VX-659
Other descriptive name: VX-659
INN or Proposed INN: TEZACAFTOR
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
460Phase 3United States;Canada;Spain;Poland;Ireland;Denmark;Australia;Israel;Norway;Germany;Switzerland;United Kingdom
210NCT03591094
(ClinicalTrials.gov)
August 21, 20186/7/2018Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic FibrosisA Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect of PTI-428 in Subjects With Cystic FibrosisCystic FibrosisDrug: PTI-428;Drug: PlaceboProteostasis Therapeutics, Inc.NULLCompleted18 YearsN/AAll40Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
211NCT03625466
(ClinicalTrials.gov)
August 10, 20187/8/2018A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508delAn Exploratory Phase 2, 2-part, Randomized, Double-blind, Placebo-controlled Study With a Long-term, Open-label Period to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508delCystic FibrosisDrug: LUM;Drug: IVA;Drug: Matched PlacebosVertex Pharmaceuticals IncorporatedNULLActive, not recruiting2 Years5 YearsAll51Phase 2Germany
212EUCTR2016-004479-35-FR
(EUCTR)
08/08/201804/06/2018A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 through 11 Years With Cystic FibrosisA Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
65Phase 3France;Poland;Belgium;Ireland;Denmark;Australia;Germany;United Kingdom;Switzerland
213NCT03551691
(ClinicalTrials.gov)
August 7, 201829/5/2018PPIs and Fat Absorption in CF and EPIProton Pump Inhibitors and Fat Absorption in Subjects With Cystic Fibrosis and Pancreatic InsufficiencyPancreatic Insufficiency;Cystic FibrosisDrug: Omeprazole 40mg Capsule;Drug: Placebo oral capsuleChildren's Hospital of PhiladelphiaChiesi Farmaceutici S.p.A.Recruiting12 YearsN/AAll24Phase 2United States
214NCT03633526
(ClinicalTrials.gov)
August 3, 201827/7/2018Evaluation of VX-659/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of AgeA Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-659/TEZ/IVA Triple Combination Therapy in Cystic Fibrosis Subjects 6 Through 11 Years of AgeCystic FibrosisDrug: VX-659/TEZ/IVA;Drug: IVAVertex Pharmaceuticals IncorporatedNULLTerminated6 Years11 YearsAll18Phase 3United States
215NCT03525548
(ClinicalTrials.gov)
August 3, 20182/5/2018A Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F)Cystic FibrosisDrug: VX-445/TEZ/IVA;Drug: TEZ/IVA;Drug: IVA;Drug: PlaceboVertex Pharmaceuticals IncorporatedNULLCompleted12 YearsN/AAll113Phase 3United States;Belgium;Netherlands;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
216NCT03655249
(ClinicalTrials.gov)
August 1, 201828/8/2018Effects of AD on VI in Patients With CFEffects of Autogenic Drainage on Ventilation Inhomogeneity in Patients With Cystic FibrosisCystic FibrosisOther: Autogenic drainage;Drug: AerosoltherapyCliniques universitaires Saint-Luc- Université Catholique de LouvainNULLRecruiting6 YearsN/AAll30N/ABelgium
217NCT03593434
(ClinicalTrials.gov)
July 30, 201828/6/2018Airway Clearance Therapy on Hyperpolarized 129Xenon and MRIThe Effect of Airway Clearance Therapy on Hyperpolarized 129Xenon MRI Compared With Lung Clearance Index and Spirometry in Cystic FibrosisCystic FibrosisDrug: hyperpolarized Xenon gasChildren's Hospital Medical Center, CincinnatiNational Heart, Lung, and Blood Institute (NHLBI)Recruiting6 Years21 YearsAll20United States
218EUCTR2017-003319-21-DE
(EUCTR)
20/07/201809/04/2018Study Assessing the Safety, Tolerability and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults with Cystic FibrosisA Phase 1 / 2 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults with Cystic Fibrosis Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Sodium (R)-8-methyl-2-(3-methylbenzofuran-2-yl)-5-(1-(tetrahydro-2H-pyran-4-yl)ethoxy)quinoline-4-ca
Product Code: PTI-801
INN or Proposed INN: None
Other descriptive name: PTI-801
Product Name: N-(5-hydroxy-2,4- bis(trimethylsilyl)phenyl)-4-oxo- 1,4-dihydroquinoline-3-carboxamide
Product Code: PTI-808
INN or Proposed INN: None
Other descriptive name: PTI-808
Product Name: N-(5-hydroxy-2,4- bis(trimethylsilyl)phenyl)-4-oxo- 1,4-dihydroquinoline-3-carboxamide
Product Code: PTI-808
INN or Proposed INN: None
Other descriptive name: PTI-808
Product Name: N-(trans-3-(5-((R)-1-hydroxyethyl)-1,3,4-oxadiazol-2-yl)cyclobutyl)-3-phenylisoxazole-5-carboxamide
Product Code: PTI-428
INN or Proposed INN: None
Other descriptive name: PTI-428
Proteostasis Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
240Phase 1;Phase 2United States;France;Canada;Belgium;Denmark;Germany;United Kingdom
219NCT03447262
(ClinicalTrials.gov)
July 13, 201821/2/2018A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination TherapyA Phase 3, Open-label Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del MutationCystic FibrosisDrug: VX-659;Drug: TEZ;Drug: IVAVertex Pharmaceuticals IncorporatedNULLCompleted12 YearsN/AAll484Phase 3United States;Australia;Canada;Denmark;Germany;Ireland;Israel;Poland;Spain;Switzerland;United Kingdom
220NCT03565692
(ClinicalTrials.gov)
July 1, 201831/5/2018Lum-Iva-biota: Exploring the Respiratory Mycobiota and Microbiota Profile in French CF Patients Taking Lumacaftor-IvacaftorLum-Iva-biota: Exploring the Respiratory Mycobiota and Microbiota Profile in French CF Patients Taking Lumacaftor-IvacaftorCystic FibrosisBiological: the lung mycobiota and microbiota profile;Biological: the gut mycobiota and microbiota profileUniversity Hospital, BordeauxSociete Francaise de la MucoviscidoseRecruiting2 YearsN/AAll250France
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
221EUCTR2017-002968-40-DK
(EUCTR)
28/06/201824/06/2018 A study lasting 96 weeks to assess a combined treatment called Tezacaftor and Ivacaftor in children aged 6 to 11 years who have Cystic FibrosisA Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
121Phase 3France;United States;Canada;Poland;Belgium;Ireland;Australia;Denmark;Germany;United Kingdom;Switzerland
222EUCTR2017-002968-40-GB
(EUCTR)
28/06/201807/03/2018A study lasting 96 weeks to assess a combined treatment called Tezacaftor and Ivacaftor in children aged 6to 11 years who have Cystic FibrosisA Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-661/ivacaftor 50 mg/ 75 mg
INN or Proposed INN: TEZACAFTOR
Other descriptive name: VRT-893661
INN or Proposed INN: IVACAFTOR
Other descriptive name: VX-770
Trade Name: Symkevi
Product Name: VX-661/ivacaftor 100 mg/ 150 mg
INN or Proposed INN: TEZACAFTOR
Other descriptive name: VRT-893661
INN or Proposed INN: IVACAFTOR
Other descriptive name: VX-770
Product Name: Ivacaftor
Product Code: VX-770, VRT-813077
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150mg
Product Name: Ivacaftor
Product Code: VX-770, VRT-813077
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
121Phase 3France;United States;Canada;Poland;Belgium;Ireland;Denmark;Australia;Germany;Switzerland;United Kingdom
223EUCTR2017-003761-99-DE
(EUCTR)
27/06/201819/02/2018A Study of the Effects of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508delAn Exploratory Phase 2, 2-part, Randomized, Double blind, Placebo controlled Study With a Long term, Open label Period to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: lumacaftor / ivacaftor
Product Code: VX-809 / VX-770
INN or Proposed INN: LUMACAFTOR
Other descriptive name: LUMACAFTOR
INN or Proposed INN: IVACAFTOR
Product Name: lumacaftor / ivacaftor
Product Code: VX-809 / VX-770
INN or Proposed INN: LUMACAFTOR
Other descriptive name: LUMACAFTOR
INN or Proposed INN: IVACAFTOR
Trade Name: Orkambi 100 mg/125 mg film-coated tablets
Product Name: LUM/IVA fixed-dose combination
INN or Proposed INN: LUMACAFTOR
Other descriptive name: LUMACAFTOR
INN or Proposed INN: IVACAFTOR
Other descriptive name: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
50Phase 2Germany
224EUCTR2017-003723-29-BE
(EUCTR)
27/06/201829/03/2018A study of safety and efficacy of lenabasum in Cystic FibrosisA Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - A Phase 2 safety and efficacy study of Lenabasum in Cystic Fibrosis Cystic Fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: LENABASUM
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: LENABASUM
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
415Phase 2Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland;United States;Serbia;Portugal;Slovakia;Greece;Spain;Réunion;Austria;Russian Federation;United Kingdom;Romania;Bulgaria;Germany;Netherlands;Sweden
225EUCTR2017-002968-40-BE
(EUCTR)
26/06/201830/03/2018A study lasting 96 weeks to assess a combined treatment called Tezacaftor and Ivacaftor in children aged 6to 11 years who have Cystic FibrosisA Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-661/ivacaftor 50 mg/ 75 mg
INN or Proposed INN: TEZACAFTOR
Other descriptive name: VRT-893661
INN or Proposed INN: IVACAFTOR
Other descriptive name: VX-770
Product Name: VX-661/ivacaftor 100 mg/ 150 mg
INN or Proposed INN: TEZACAFTOR
Other descriptive name: VRT-893661
INN or Proposed INN: IVACAFTOR
Other descriptive name: VX-770
Product Name: Ivacaftor
Product Code: VX-770, VRT-813077
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150mg
Product Name: Ivacaftor
Product Code: VX-770, VRT-813077
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
121Phase 3France;United States;Canada;Poland;Belgium;Ireland;Denmark;Australia;Germany;United Kingdom;Switzerland
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
226EUCTR2016-004479-35-BE
(EUCTR)
26/06/201830/03/2018A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 through 11 Years With Cystic FibrosisA Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
65Phase 3France;Poland;Belgium;Ireland;Denmark;Australia;Germany;United Kingdom;Switzerland
227EUCTR2016-004479-35-DE
(EUCTR)
26/06/201806/04/2018A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 through 11 Years With Cystic FibrosisA Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
65Phase 3France;Poland;Belgium;Ireland;Denmark;Australia;Germany;United Kingdom;Switzerland
228NCT03502070
(ClinicalTrials.gov)
June 26, 201829/3/2018Human Factors Study to Validate the User Interface of TOBI Podhaler Using Placebo CapsulesA Multicenter, Human Factors Validation Study in Cystic Fibrosis Patients Aged 6 Years and Older to Evaluate the User Interface of TOBI® Podhaler™ (Tobramycin Inhalation Powder) Using Placebo CapsulesCystic FibrosisDrug: Placebo;Device: Tobi PodhalerMylan Inc.NULLCompleted6 YearsN/AAll47Phase 4United States
229EUCTR2018-000098-61-GB
(EUCTR)
22/06/201801/06/2018A study to evaluate the efficacy, safety, tolerability and pharmacokinetics of the combination of GLPG3067, GLPG2222 and GLPG2737 in adult patients with cystic fibrosisA Phase II, randomized, double-blind, placebo-controlled, multi-center study to evaluate the efficacy, safety, tolerability and pharmacokinetics of orally administered combination of GLPG3067, GLPG2222 and GLPG2737, in adult subjects with cystic fibrosis homozygous or heterozygous for F508del CFTR Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Galapagos NVNULLNot Recruiting Female: yes
Male: yes
144Phase 2Serbia;France;United States;Belgium;Ireland;Denmark;Australia;Netherlands;Germany;New Zealand;United Kingdom
230NCT04278040
(ClinicalTrials.gov)
June 20, 20187/2/2020Inhalations of Ultra-low Doses of Melphalan for the Treatment of Non-cystic Fibrosis BronchiectasisSafety, Tolerability and Efficacy of Ultra-low Doses of Alkylating Drug Melphalan Inhalations for the Treatment of Non-cystic Fibrosis BronchiectasisNon-cystic Fibrosis BronchiectasisDrug: MelphalanFederal State Budgetary Institution, Pulmonology Scientific Research InstituteMoscow State University of Medicine and DentistryRecruiting18 Years70 YearsAll7Phase 2Russian Federation
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
231NCT03525444
(ClinicalTrials.gov)
June 15, 20182/5/2018A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)Cystic FibrosisDrug: VX-445/TEZ/IVA;Drug: IVA;Drug: PlaceboVertex Pharmaceuticals IncorporatedNULLCompleted12 YearsN/AAll405Phase 3United States;Australia;Austria;Belgium;Canada;Czechia;France;Germany;Greece;Italy;Netherlands;Sweden;United Kingdom
232EUCTR2018-001573-24-BE
(EUCTR)
11/06/201808/05/2018Evaluation of the short-term effect of ORKAMBI on lung function in patients with cystic fibrosis who have two copies of the F508del mutation using functional respiratory imagingFunctional Respiratory Imaging (FRI) to assess the short-term effect of the product ORKAMBI (lumacaftor/ ivacaftor) on lung function in ORKAMBI-naive patients with Cystic Fibrosis Homozygous for Phe508del. Cystic Fibrosis Homozygous (homozygous for the F508del mutation)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Orkambi
INN or Proposed INN: LUMACAFTOR
Other descriptive name: LUMACAFTOR
INN or Proposed INN: IVACAFTOR
Antwerp University HospitalNULLNot RecruitingFemale: yes
Male: yes
20Phase 4Belgium
233EUCTR2017-003723-29-SE
(EUCTR)
07/06/201801/03/2018A study of safety and efficacy of lenabasum in Cystic FibrosisA Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - A Phase 2 safety and efficacy study of Lenabasum in Cystic Fibrosis Cystic Fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: LENABASUM
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: LENABASUM
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
415Phase 2United States;Serbia;Portugal;Slovakia;Greece;Spain;Réunion;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland;Romania;Bulgaria;Germany;Netherlands;Sweden
234EUCTR2017-003319-21-GB
(EUCTR)
04/06/201803/07/2018Study Assessing the Safety, Tolerability and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults with Cystic FibrosisA Phase 1 / 2 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults with Cystic Fibrosis Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: N-(5-hydroxy-2,4-bis(trimethylsilyl)phenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide
Product Code: PTI-808
INN or Proposed INN: None
Other descriptive name: PTI-808
Product Name: Sodium (R)-8-methyl-2-(3-methylbenzofuran-2-yl)-5-(1-(tetrahydro-2H-pyran-4-yl)ethoxy)quinoline-4-ca
Product Code: PTI-801
INN or Proposed INN: None
Other descriptive name: PTI-801
Product Name: N-(trans-3-(5-((R)-1-hydroxyethyl)-1,3,4-oxadiazol-2-yl)cyclobutyl)-3-phenylisoxazole-5-carboxamide
Product Code: PTI-428
INN or Proposed INN: None
Other descriptive name: PTI-428
Proteostasis Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
240Phase 1;Phase 2France;United States;Canada;Belgium;Denmark;Germany;United Kingdom
235NCT03540524
(ClinicalTrials.gov)
May 31, 201827/4/2018A Study Looking at the Safety, Tolerability and Efficacy of the Combination of the Study Drugs GLPG2451 and GLPG2222 With or Without GLPG2737 in Patients With Cystic Fibrosis.Assessment of Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Oral Doses of the Combination of GLPG2451 and GLPG2222, With or Without GLPG2737, in Adult Subjects With Cystic FibrosisCystic FibrosisDrug: GLPG2451 dose regimen A;Drug: GLPG2451 dose regimen B;Drug: GLPG2222;Drug: GLPG2737Galapagos NVNULLCompleted18 YearsN/AAll10Phase 1Belgium;Bulgaria;Germany;Greece;Netherlands;Serbia;Sweden;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
236EUCTR2016-001440-18-NL
(EUCTR)
30/05/201816/01/2018The influence of cytochrome P450 3A4 inhibitors on serum levels of Ivacaftor in cystic fibrosis patients and healthy subjects.Pharmacokinetic interactions between ivacaftor and cytochrome P450 3A4 inhibitors in cystic fibrosis patients and healthy controls - IACI cystic fibrosis and healthy subjects;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Trade Name: Ivacaftor
Trade Name: claritromycin 500mg film coated tablets
Product Name: Claritromycin
Trade Name: Azithromycin 500 mg film-coated tablet
Product Name: azithromycin
Trade Name: Norvir 100 mg film-coated tablets
Product Name: Norvir
university medical center utrechtNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Phase 4Netherlands
237NCT03482960
(ClinicalTrials.gov)
May 29, 201823/3/2018Comparison of 129Xe MRI With 19F MRI in CF Lung DiseaseComparison of 129Xe MRI With 19F MRI in CF Lung DiseaseCystic FibrosisDrug: Hyperpolarized Xenon gas;Drug: PFPUniversity of North Carolina, Chapel HillCystic Fibrosis FoundationCompleted18 YearsN/AAll15Early Phase 1United States
238EUCTR2017-003723-29-ES
(EUCTR)
21/05/201809/04/2018A study of safety and efficacy of lenabasum in Cystic FibrosisA Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - A Phase 2 safety and efficacy study of Lenabasum in Cystic Fibrosis Cystic Fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
415Phase 2Portugal;Serbia;United States;Slovakia;Greece;Spain;Réunion;Austria;Russian Federation;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Romania;Australia;Bulgaria;Netherlands;Germany;Sweden
239EUCTR2017-004134-29-ES
(EUCTR)
18/05/201821/03/2018A Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic FibrosisA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-659/Tezacaftor/Ivacaftor
Product Code: VX-659/ TEZ/IVA
INN or Proposed INN: VX-659
Other descriptive name: VX-659
INN or Proposed INN: TEZACAFTOR
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
460Phase 3United States;Canada;Poland;Spain;Ireland;Denmark;Australia;Israel;Norway;Germany;United Kingdom;Switzerland
240EUCTR2017-004133-82-ES
(EUCTR)
18/05/201815/03/2018A Study Evaluating Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic FibrosisA Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-659/Tezacaftor/Ivacaftor
Product Code: VX-659/TEZ/IVA
INN or Proposed INN: VX-659
Other descriptive name: VX-659
INN or Proposed INN: TEZACAFTOR
INN or Proposed INN: IVACAFTOR
Product Name: Tezacaftor/Ivacaftor/100mg/150mg
Product Code: VX-661/VX-770 (TEZ/IVA)
INN or Proposed INN: TEZACAFTOR
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
100Phase 3United States;Spain;Ireland;Australia;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
241EUCTR2017-004132-11-ES
(EUCTR)
18/05/201805/03/2018A Study to Evaluate the Efficacy and Safety of VX-659 Drug in Combination With Other Drugs in Subjects With Cystic FibrosisA Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
360Phase 3United States;Canada;Poland;Spain;Ireland;Denmark;Australia;Israel;Norway;Germany;United Kingdom;Switzerland
242EUCTR2017-003723-29-AT
(EUCTR)
17/05/201828/03/2018A study of safety and efficacy of lenabasum in Cystic FibrosisA Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - A Phase 2 safety and efficacy study of Lenabasum in Cystic Fibrosis Cystic Fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
415Phase 2Portugal;United States;Serbia;Slovakia;Greece;Spain;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland;Romania;Bulgaria;Germany;Netherlands;Sweden
243NCT03462056
(ClinicalTrials.gov)
May 17, 20185/3/2018Ready to Use Therapeutic Food (RUTF) to Promote Growth in Cystic FibrosisPilot Study of Ready to Use Therapeutic Food to Promote Weight Gain in Cystic FibrosisCystic FibrosisDietary Supplement: Cystic Fibrosis Ready to Use Supplemental FoodWashington University School of MedicineCystic Fibrosis FoundationCompleted2 Years12 YearsAll16Phase 1;Phase 2United States
244NCT03559062
(ClinicalTrials.gov)
May 17, 20185/6/2018A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic FibrosisA Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR MutationCystic FibrosisDrug: TEZ/IVA;Drug: IVA;Drug: PlaceboVertex Pharmaceuticals IncorporatedNULLCompleted6 Years11 YearsAll67Phase 3Australia;Belgium;Denmark;France;Germany;Ireland;Poland;Switzerland;United Kingdom
245EUCTR2017-004134-29-IE
(EUCTR)
15/05/201805/03/2018A Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic FibrosisA Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-659/Tezacaftor/Ivacaftor
Product Code: VX-659/ TEZ/IVA
INN or Proposed INN: VX-659
Other descriptive name: VX-659
INN or Proposed INN: TEZACAFTOR
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
460Phase 3United States;Canada;Spain;Poland;Ireland;Denmark;Australia;Israel;Norway;Germany;United Kingdom;Switzerland
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
246EUCTR2017-003723-29-GB
(EUCTR)
11/05/201805/02/2018A study of safety and efficacy of lenabasum in Cystic FibrosisA Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - A Phase 2 safety and efficacy study of Lenabasum in Cystic Fibrosis Cystic Fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: LENABASUM
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: LENABASUM
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
415Phase 2United States;Serbia;Portugal;Slovakia;Greece;Spain;Réunion;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland;Romania;Bulgaria;Germany;Netherlands;Sweden
247NCT03375047
(ClinicalTrials.gov)
May 10, 20187/9/2017Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic FibrosisA Phase 1/2, Randomized, Double-Blinded, Placebo-Controlled, Combined Single and Multiple Ascending Dose Study Evaluating the Safety, Tolerability, and Biological Activity of MRT5005 Administered by Nebulization to Adult Subjects With Cystic FibrosisCystic FibrosisDrug: MRT5005;Drug: Normal salineTranslate Bio, Inc.NULLRecruiting18 YearsN/AAll40Phase 1;Phase 2United States
248NCT03428334
(ClinicalTrials.gov)
May 10, 201826/1/2018Roflumilast in Non-CF Bronchiectasis StudyA 4-week Single-arm Study of Roflumilast in Stable-state Non-cystic Fibrosis BronchiectasisNon-cystic Fibrosis BronchiectasisDrug: Oral roflumilastThe University of Hong KongNULLCompleted18 YearsN/AAll20Phase 2Hong Kong
249EUCTR2016-002749-42-NL
(EUCTR)
09/05/201829/08/2017Study of Aztreonam for Inhalation in Children with Cystic Fibrosis and New Infection of the Airways by Pseudomonas aeruginosa bacteriaRandomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonization - ALPINE2 (Aztreonam Lysine for Pseudomonas Infection Eradication 2) Cystic fibrosis and new onset lower respiratory tract culture positive for Pseudomonas aeruginosa
MedDRA version: 20.0;Level: LLT;Classification code 10068288;Term: Cystic fibrosis pulmonary exacerbation;System Organ Class: 100000113915;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Cayston
Product Code: AZLI
INN or Proposed INN: AZTREONAM
Gilead Sciences, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
140Phase 3United States;Greece;Spain;Ireland;Austria;Israel;United Kingdom;Italy;France;Belgium;Denmark;Netherlands;Germany
250EUCTR2017-004132-11-DK
(EUCTR)
08/05/201802/03/2018A Study to Evaluate the Efficacy and Safety of VX-659 Drug in Combination With Other Drugs in Subjects With Cystic FibrosisA Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
360Phase 3United States;Canada;Poland;Spain;Ireland;Australia;Denmark;Israel;Norway;Germany;United Kingdom;Switzerland
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
251NCT03579173
(ClinicalTrials.gov)
May 8, 201825/6/2018Determinants of Early Cystic Fibrosis Lung DiseaseDeterminants of Early Cystic Fibrosis Lung DiseaseCystic Fibrosis in ChildrenDiagnostic Test: Infant PFT;Diagnostic Test: Lung Clearance Index;Diagnostic Test: Passive tidal breathingIndiana UniversityNULLActive, not recruiting1 Month5 YearsAll55United States
252EUCTR2017-004133-82-GB
(EUCTR)
02/05/201814/06/2018A Study Evaluating Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic FibrosisA Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
100Phase 3United States;Spain;Ireland;Australia;Germany;United Kingdom
253EUCTR2017-004132-11-GB
(EUCTR)
02/05/201823/02/2018A Study to Evaluate the Efficacy and Safety of VX-659 Drug in Combination With Other Drugs in Subjects With Cystic FibrosisA Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-659/Tezacaftor/Ivacaftor
Product Code: VX-659/TEZ/IVA
INN or Proposed INN: VX-659
Other descriptive name: VX-659
INN or Proposed INN: TEZACAFTOR
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
360Phase 3United States;Canada;Spain;Poland;Ireland;Denmark;Australia;Israel;Norway;Germany;Switzerland;United Kingdom
254EUCTR2017-004132-11-IE
(EUCTR)
01/05/201805/03/2018A Study to Evaluate the Efficacy and Safety of VX-659 Drug in Combination With Other Drugs in Subjects With Cystic FibrosisA Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
360Phase 3United States;Canada;Poland;Spain;Ireland;Denmark;Australia;Israel;Norway;Germany;United Kingdom;Switzerland
255NCT03460990
(ClinicalTrials.gov)
May 1, 20181/3/2018A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F)Cystic FibrosisDrug: VX-659/TEZ/IVA;Drug: TEZ/IVA;Drug: IVA;Drug: PlaceboVertex Pharmaceuticals IncorporatedNULLCompleted12 YearsN/AAll116Phase 3United States;Australia;Germany;Ireland;Spain;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
256NCT03522831
(ClinicalTrials.gov)
May 1, 201819/3/2018CF BronchodilationA Double-blind Placebo-controlled Crossover Study to Assess the Effects of Bronchodilation on Dyspnea, Ventilatory Responses, and Exercise Tolerance in Adults With Cystic FibrosisLung Diseases;Cystic FibrosisDrug: Salbutamol;Drug: PlaceboUniversity of British ColumbiaNULLRecruiting19 YearsN/AAll20N/ACanada
257EUCTR2017-004133-82-IE
(EUCTR)
01/05/201805/03/2018A Study Evaluating Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic FibrosisA Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-659/Tezacaftor/Ivacaftor
Product Code: VX-659/TEZ/IVA
INN or Proposed INN: VX-659
Other descriptive name: VX-659
INN or Proposed INN: TEZACAFTOR
INN or Proposed INN: IVACAFTOR
Product Name: Tezacaftor/Ivacaftor/100mg/150mg
Product Code: VX-661/VX-770 (TEZ/IVA)
INN or Proposed INN: TEZACAFTOR
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
100Phase 3United States;Spain;Ireland;Australia;Germany;United Kingdom
258EUCTR2017-002968-40-IE
(EUCTR)
27/04/201808/03/2018 A study lasting 96 weeks to assess a combined treatment called Tezacaftor and Ivacaftor in children aged 6 to 11 years who have Cystic FibrosisA Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
121Phase 3France;United States;Canada;Poland;Belgium;Ireland;Denmark;Australia;Germany;United Kingdom;Switzerland
259EUCTR2017-002533-32-NL
(EUCTR)
26/04/201829/11/2017A Study to Assess the Efficacy and Safety of Study Drug INS1007 Administered Once Daily in Patients with Non-Cystic Fibrosis Bronchiectasis.A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Center Study to Assess the Efficacy, Safety and Tolerability, and Pharmacokinetics of INS1007 Administered Once Daily for 24 Weeks in Subjects with Non-Cystic Fibrosis Bronchiectasis - The Willow Study. Non-Cystic Fibrosis Bronchiectasis
MedDRA version: 20.0;Level: SOC;Classification code 10038738;Term: Respiratory, thoracic and mediastinal disorders;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
MedDRA version: 20.1;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: INS1007
INN or Proposed INN: not available
Other descriptive name: AZD7986
Product Name: INS1007
INN or Proposed INN: not available
Other descriptive name: AZD7986
Insmed IncorporatedNULLNot RecruitingFemale: yes
Male: yes
240Phase 2United States;Spain;Italy;United Kingdom;Belgium;Poland;Singapore;Australia;Denmark;Bulgaria;Netherlands;Germany;New Zealand;Sweden;Korea, Republic of
260EUCTR2017-003723-29-SK
(EUCTR)
26/04/201823/03/2018A study of safety and efficacy of lenabasum in Cystic FibrosisA Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - A Phase 2 safety and efficacy study of Lenabasum in Cystic Fibrosis Cystic Fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
415Phase 2United States;Serbia;Portugal;Slovakia;Greece;Spain;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Belgium;Poland;Romania;Bulgaria;Germany;Netherlands;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
261EUCTR2017-004132-11-DE
(EUCTR)
25/04/201827/02/2018A Study to Evaluate the Efficacy and Safety of VX-659 Drug in Combination With Other Drugs in Subjects With Cystic FibrosisA Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF) Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-659/Tezacaftor/Ivacaftor
Product Code: VX-659/TEZ/IVA
INN or Proposed INN: VX-659
Other descriptive name: VX-659
INN or Proposed INN: TEZACAFTOR
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
360Phase 3Germany;United States;Canada;Poland;Spain;Ireland;Denmark;Australia;Israel;Norway;United Kingdom;Switzerland
262NCT03537651
(ClinicalTrials.gov)
April 25, 201815/5/2018A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Subjects With an F508del CFTR MutationA Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR MutationCystic FibrosisDrug: TEZ;Drug: IVAVertex Pharmaceuticals IncorporatedNULLActive, not recruiting6 YearsN/AAll130Phase 3United States;Australia;Belgium;Canada;Denmark;France;Germany;Ireland;Poland;Switzerland;United Kingdom
263NCT03492567
(ClinicalTrials.gov)
April 25, 20183/4/2018Study of Circulating Blood Cell Monocytes as Predictive Biomarker of Bone Cystic Fibrosis Disease. Impact of CFTR Correctors.Study of Circulating Monocytes as Predictive Biomarker of Osteoclastogenesis in Cystic Fibrosis-related Bone Loss. Impact of CFTR Correctors.Cystic FibrosisBiological: Blood testAssistance Publique - Hôpitaux de ParisVaincre la MucoviscidoseCompleted18 YearsN/AAll25N/AFrance
264EUCTR2016-004479-35-GB
(EUCTR)
24/04/201826/04/2018A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 through 11 Years With Cystic FibrosisA Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-661/ivacaftor 50 mg/ 75 mg
INN or Proposed INN: TEZACAFTOR
Other descriptive name: VRT-893661
INN or Proposed INN: IVACAFTOR
Other descriptive name: VX-770
Product Name: VX-661/ivacaftor 100 mg/ 150 mg
INN or Proposed INN: TEZACAFTOR
Other descriptive name: VRT-893661
INN or Proposed INN: IVACAFTOR
Other descriptive name: VX-770
Product Name: Ivacaftor
Product Code: VX-770, VRT-813077
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150mg
Product Name: Ivacaftor
Product Code: VX-770, VRT-813077
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
65Phase 3France;Poland;Belgium;Ireland;Denmark;Australia;Germany;Switzerland;United Kingdom
265EUCTR2016-004479-35-DK
(EUCTR)
18/04/201822/02/2018A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 through 11 Years With Cystic FibrosisA Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
65Phase 3France;Poland;Belgium;Ireland;Australia;Denmark;Germany;United Kingdom;Switzerland
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
266EUCTR2017-003723-29-HU
(EUCTR)
16/04/201827/02/2018A study of safety and efficacy of lenabasum in Cystic FibrosisA Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis - A Phase 2 safety and efficacy study of Lenabasum in Cystic Fibrosis Cystic Fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Product Name: Lenabasum
Product Code: JBT-101
INN or Proposed INN: Lenabasum
Other descriptive name: resunab, ajulemic acid, anabasum
Corbus Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
415Phase 2Portugal;Serbia;United States;Slovakia;Greece;Spain;Austria;Russian Federation;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Romania;Bulgaria;Netherlands;Germany;Sweden
267NCT03527095
(ClinicalTrials.gov)
April 5, 20184/5/2018A Study to Compare the Pharmacokinetics of Different Oral Formulations of FDL169 in Healthy SubjectsA Phase 1, Open-label, Randomised, Cross Over Study to Compare the Pharmacokinetics of Different Oral Formulations of FDL169 in Healthy Subjects Following Single DosesCystic FibrosisDrug: FDL169Flatley Discovery Lab LLCNULLCompleted18 Years55 YearsAll11Phase 1United Kingdom
268NCT03489629
(ClinicalTrials.gov)
April 3, 201829/3/2018STaph Aureus Resistance-Treat Early and Repeat (STAR-TER)STaph Aureus Resistance-Treat Early and Repeat (STAR-TER)Cystic FibrosisDrug: Trimethoprim Sulfamethoxazole (TMP/SMX);Drug: Minocycline;Drug: Mupirocin;Drug: Chlorhexidine Gluconate;Behavioral: Environmental DecontaminationUniversity of North Carolina, Chapel HillUniversity of Washington;Cook Children's Medical Center;Indiana University;University of Michigan;University of Texas Southwestern Medical Center;St. Louis Children's HospitalRecruiting2 Years45 YearsAll42Phase 2United States
269EUCTR2017-002533-32-ES
(EUCTR)
29/03/201822/11/2017A Study to Assess the Efficacy and Safety of Study Drug INS1007 Administered Once Daily in Patients with Non-Cystic Fibrosis Bronchiectasis.A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Center Study to Assess the Efficacy, Safety and Tolerability, and Pharmacokinetics of INS1007 Administered Once Daily for 24 Weeks in Subjects with Non-Cystic Fibrosis Bronchiectasis - The Willow Study. Non-Cystic Fibrosis Bronchiectasis;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]Insmed IncorporatedNULLNot Recruiting Female: yes
Male: yes
240Phase 2United States;Spain;United Kingdom;Italy;France;Canada;Belgium;Poland;Singapore;Australia;Denmark;Bulgaria;Germany;Netherlands;New Zealand;Sweden;Korea, Republic of
270EUCTR2017-002533-32-PL
(EUCTR)
28/03/201818/01/2018A Study to Assess the Efficacy and Safety of Study Drug INS1007 Administered Once Daily in Patients with Non-Cystic Fibrosis Bronchiectasis.A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Center Study to Assess the Efficacy, Safety and Tolerability, and Pharmacokinetics of INS1007 Administered Once Daily for 24 Weeks in Subjects with Non-Cystic Fibrosis Bronchiectasis - The Willow Study. Non-Cystic Fibrosis Bronchiectasis
MedDRA version: 20.0;Level: SOC;Classification code 10038738;Term: Respiratory, thoracic and mediastinal disorders;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
MedDRA version: 21.0;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: INS1007
INN or Proposed INN: not available
Other descriptive name: AZD7986
Product Name: INS1007
INN or Proposed INN: not available
Other descriptive name: AZD7986
Insmed IncorporatedNULLNot RecruitingFemale: yes
Male: yes
240Phase 2United States;Spain;Italy;United Kingdom;Poland;Belgium;Singapore;Australia;Denmark;Bulgaria;Germany;Netherlands;New Zealand;Sweden;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
271EUCTR2017-002533-32-BG
(EUCTR)
23/03/201801/12/2017A Study to Assess the Efficacy and Safety of Study Drug INS1007 Administered Once Daily in Patients with Non-Cystic Fibrosis Bronchiectasis.A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Center Study to Assess the Efficacy, Safety and Tolerability, and Pharmacokinetics of INS1007 Administered Once Daily for 24 Weeks in Subjects with Non-Cystic Fibrosis Bronchiectasis - The Willow Study. Non-Cystic Fibrosis Bronchiectasis
MedDRA version: 20.0;Level: SOC;Classification code 10038738;Term: Respiratory, thoracic and mediastinal disorders;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
MedDRA version: 20.1;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: INS1007
INN or Proposed INN: not available
Other descriptive name: AZD7986
Product Name: INS1007
INN or Proposed INN: not available
Other descriptive name: AZD7986
Insmed IncorporatedNULLNot RecruitingFemale: yes
Male: yes
240Phase 2United States;Spain;Italy;United Kingdom;Belgium;Poland;Singapore;Australia;Denmark;Bulgaria;Germany;Netherlands;New Zealand;Sweden;Korea, Republic of
272EUCTR2017-002533-32-GB
(EUCTR)
22/03/201823/10/2017A Study to Assess the Efficacy and Safety of Study Drug INS1007 Administered Once Daily in Patients with Non-Cystic Fibrosis Bronchiectasis.A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Center Study to Assess the Efficacy, Safety and Tolerability, and Pharmacokinetics of INS1007 Administered Once Daily for 24 Weeks in Subjects with Non-Cystic Fibrosis Bronchiectasis - The Willow Study. Non-Cystic Fibrosis Bronchiectasis
MedDRA version: 20.0;Level: SOC;Classification code 10038738;Term: Respiratory, thoracic and mediastinal disorders;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
MedDRA version: 20.1;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: INS1007
INN or Proposed INN: not available
Other descriptive name: AZD7986
Product Name: INS1007
INN or Proposed INN: not available
Other descriptive name: AZD7986
Insmed IncorporatedNULLNot RecruitingFemale: yes
Male: yes
240Phase 2United Kingdom;United States;Spain;Italy;Belgium;Poland;Singapore;Australia;Denmark;Bulgaria;Germany;Netherlands;New Zealand;Sweden;Korea, Republic of
273NCT03768089
(ClinicalTrials.gov)
March 20, 20185/12/2018Study of VX-121 in Healthy Subjects and in Subjects With Cystic FibrosisA Phase 1/2 Study of VX-121 in Healthy Subjects and in Subjects With Cystic FibrosisCystic FibrosisDrug: VX-121;Drug: TEZ/IVA;Drug: IVA;Drug: Matched PlaceboVertex Pharmaceuticals IncorporatedNULLCompleted18 YearsN/AAll114Phase 1;Phase 2Netherlands;United Kingdom
274EUCTR2018-000126-55-NL
(EUCTR)
15/03/201815/05/2018A study to investigate the safety and the movement of the study drug VX-121 around the body in healthy people and patients with cystic fibrosisA Phase 1/2 Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-121
INN or Proposed INN: Not yet assigned
Product Name: tezacaftor/ivacaftor 100mg/150mg
Product Code: VX-661/VX-770
INN or Proposed INN: TEZACAFTOR
Other descriptive name: VX-661
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
120Phase 1;Phase 2Netherlands
275EUCTR2017-002533-32-DE
(EUCTR)
15/03/201803/11/2017A Study to Assess the Efficacy and Safety of Study Drug INS1007 Administered Once Daily in Patients with Non-Cystic Fibrosis Bronchiectasis.A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Center Study to Assess the Efficacy, Safety and Tolerability, and Pharmacokinetics of INS1007 Administered Once Daily for 24 Weeks in Subjects with Non-Cystic Fibrosis Bronchiectasis - The Willow Study. Non-Cystic Fibrosis Bronchiectasis
MedDRA version: 20.0;Level: SOC;Classification code 10038738;Term: Respiratory, thoracic and mediastinal disorders;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
MedDRA version: 20.1;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: INS1007
INN or Proposed INN: not available
Other descriptive name: AZD7986
Product Name: INS1007
INN or Proposed INN: not available
Other descriptive name: AZD7986
Insmed IncorporatedNULLNot RecruitingFemale: yes
Male: yes
240Phase 2United States;Spain;United Kingdom;Italy;Belgium;Poland;Singapore;Australia;Denmark;Bulgaria;Germany;Netherlands;New Zealand;Korea, Republic of;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
276NCT03447249
(ClinicalTrials.gov)
March 7, 201821/2/2018A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)Cystic FibrosisDrug: VX-659/TEZ/IVA;Drug: IVA;Drug: PlaceboVertex Pharmaceuticals IncorporatedNULLCompleted12 YearsN/AAll385Phase 3United States;Australia;Canada;Denmark;Germany;Ireland;Israel;Poland;Spain;Switzerland;United Kingdom
277NCT03516331
(ClinicalTrials.gov)
March 7, 201824/4/2018A Drug-Drug Interaction Study of FDL169 and FDL176 in Healthy SubjectsA Phase 1, Open Label, Drug-Drug Interaction Study of FDL169 and FDL176 in Healthy SubjectsCystic FibrosisDrug: FDL176 & FDL169 coadministrationFlatley Discovery Lab LLCNULLCompleted18 Years55 YearsAll16Phase 1United Kingdom
278NCT03421366
(ClinicalTrials.gov)
March 1, 201827/12/2017Modified Release Posaconazole in Patients With Cystic FibrosisModified Release Posaconazole in Patients With Cystic FibrosisCystic FibrosisDrug: PosaconazoleBayside HealthNULLRecruiting18 YearsN/AAll20Australia
279NCT04411901
(ClinicalTrials.gov)
March 1, 201822/5/2020The Role of Vitamin D3 in Pediatric Bronchiectasis SeverityThe Role of Vitamin D3 in Pediatric Bronchiectasis Severity( CF Versus Non CF Bronchioectasis)Cystic Fibrosis and Non CF BronchiectasisDrug: Cholecalciferol (vitaminD3)Heba OmaraNULLCompleted1 Year17 YearsAll40Phase 2;Phase 3Egypt
280NCT03367494
(ClinicalTrials.gov)
March 1, 201822/11/2017A New Optical Sweat Test Method Based on a Citric Acid-derived Multi-halide SensorA New Optical Sweat Test Method Based on a Citric Acid-derived Multi-halide SensorCystic FibrosisDiagnostic Test: Measurement of Sweat Chloride and Sweat BromideMilton S. Hershey Medical CenterNULLActive, not recruiting18 YearsN/AAll50N/AUnited States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
281NCT03589313
(ClinicalTrials.gov)
February 12, 20189/7/2018Pharmacokinetics of GLPG3067 in Male Subjects With Cystic Fibrosis.Evaluation of the Pharmacokinetics, Safety and Tolerability of a Single Dose of GLPG3067 Administered as Solid Formulation in Male Subjects With Cystic Fibrosis.Cystic FibrosisDrug: GLPG3067 single doseGalapagos NVNULLCompleted18 YearsN/AMale6Phase 1Belgium
282NCT03500263
(ClinicalTrials.gov)
January 30, 20189/4/2018Safety, Tolerability, and Pharmacokinetics of PTI-808, PTI-801, and PTI-428 Combination Therapy in Subjects With Cystic FibrosisA Phase 1 / 2, Randomized, Double-Blind, Placebo-Controlled Study Designed to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808, PTI-801, and PTI-428 Combination Therapy in Subjects With Cystic FibrosisCystic FibrosisDrug: PTI-808;Drug: Placebo;Drug: PTI-801;Drug: PTI-428Proteostasis Therapeutics, Inc.NULLCompleted18 YearsN/AAll12Phase 1;Phase 2United Kingdom
283EUCTR2017-002533-32-SE
(EUCTR)
30/01/201814/11/2017A Study to Assess the Efficacy and Safety of Study Drug INS1007 Administered Once Daily in Patients with Non-Cystic Fibrosis Bronchiectasis.A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Center Study to Assess the Efficacy, Safety and Tolerability, and Pharmacokinetics of INS1007 Administered Once Daily for 24 Weeks in Subjects with Non-Cystic Fibrosis Bronchiectasis - The Willow Study. Non-Cystic Fibrosis Bronchiectasis
MedDRA version: 20.0;Level: SOC;Classification code 10038738;Term: Respiratory, thoracic and mediastinal disorders;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
MedDRA version: 20.1;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Insmed IncorporatedNULLNot Recruiting Female: yes
Male: yes
240Phase 2United States;Spain;United Kingdom;Italy;France;Belgium;Poland;Singapore;Australia;Denmark;Bulgaria;Germany;Netherlands;New Zealand;Sweden;Korea, Republic of
284NCT03460704
(ClinicalTrials.gov)
January 29, 201814/2/2018Trial in Non-cystic Fibrosis Bronchiectasis Patients With Chronic Lung Infections Treated With Colistimethate Sodium (PROMIS II)A Double-blind, Placebo-controlled, Multi-centre, Clinical Trial to Investigate the Efficacy and Safety of 12 Months of Therapy With Inhaled Colistimethate Sodium in the Treatment of Subjects With Non-cystic Fibrosis Bronchiectasis Chronically Infected With Pseudomonas Aeruginosa (P. Aeruginosa)Non Cystic Fibrosis BronchiectasisDrug: Colistimethate sodium;Drug: Saline SolutionZambon SpANULLRecruiting18 Years90 YearsAll420Phase 3United States;Argentina;Australia;Canada;France;Germany;Greece;Israel;Italy;New Zealand;Poland;Portugal
285EUCTR2017-002533-32-IT
(EUCTR)
23/01/201805/11/2020A Study to Assess the Efficacy and Safety of Study Drug INS1007 Administered Once Daily in Patients with Non-Cystic FibrosisA Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Center Study to Assess the Efficacy, Safety and Tolerability, and Pharmacokinetics of INS1007 Administered Once Daily for 24 Weeks in Subjects with Non-Cystic Fibrosis Bronchiectasis - The Willow Study - N/A Non-Cystic Fibrosis Bronchiectasis
MedDRA version: 20.0;Level: SOC;Classification code 10038738;Term: Respiratory, thoracic and mediastinal disorders;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
MedDRA version: 21.0;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: INS1007
Product Code: [N/A]
Product Name: INS1007
Product Code: [N/A]
INSMED INCORPORATEDNULLNot RecruitingFemale: yes
Male: yes
240Phase 2United States;Spain;Italy;United Kingdom;Belgium;Poland;Singapore;Australia;Denmark;Bulgaria;Germany;Netherlands;New Zealand;Sweden;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
286EUCTR2016-002749-42-GR
(EUCTR)
12/01/201803/10/2017Study of Aztreonam for Inhalation in Children with Cystic Fibrosis and New Infection of the Airways by Pseudomonas aeruginosa bacteriaRandomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonization - ALPINE2 (Aztreonam Lysine for Pseudomonas Infection Eradication 2) Cystic fibrosis and new onset lower respiratory tract culture positive for Pseudomonas aeruginosa
MedDRA version: 20.0;Level: LLT;Classification code 10068288;Term: Cystic fibrosis pulmonary exacerbation;System Organ Class: 100000113915;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Cayston
Product Code: AZLI
INN or Proposed INN: AZTREONAM
Gilead Sciences, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
140Phase 3United States;Greece;Spain;Ireland;Austria;Israel;United Kingdom;Italy;France;Belgium;Denmark;Germany;Netherlands
287NCT02653027
(ClinicalTrials.gov)
January 1, 20187/1/2016Effect of Lumacaftor-ivacaftor on Glucose Handling and Tolerance in Cystic Fibrosis Phe508delA Study of the Effect of Combination Lumacaftor and Ivacaftor on Glucose Tolerance in Persons With Cystic Fibrosis Who Are Homozygous for the Phe508del Cystic Fibrosis Transmembrane Conductance Regulator Mutation.Diabetes;Cystic FibrosisDrug: Lumacaftor-ivacaftor;Other: OGTTMassachusetts General HospitalNULLWithdrawn18 Years65 YearsAll0N/ANULL
288NCT03451045
(ClinicalTrials.gov)
December 22, 201723/2/2018Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic FibrosisA Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic FibrosisCystic FibrosisDrug: Lenabasum 20 mg;Drug: Lenabasum 5 mg;Other: PlaceboCorbus Pharmaceuticals Inc.Cystic Fibrosis FoundationActive, not recruiting12 YearsN/AAll415Phase 2United States;Austria;Belgium;Bulgaria;Canada;Czechia;France;Germany;Greece;Hungary;Italy;Netherlands;Poland;Portugal;Romania;Russian Federation;Serbia;Slovakia;Spain;Sweden;United Kingdom
289NCT03424252
(ClinicalTrials.gov)
December 18, 201731/1/2018An Phase 1 Study to Evaluate the Pharmacokinetic (PK) Profile of FDL169 New Formulations in Healthy SubjectsA Phase 1, Open-label, Crossover, Randomised Study to Evaluate the Pharmacokinetic Profile of FDL169 Sublingual Formulations in the Fed State in Healthy SubjectsCystic FibrosisDrug: FDL169Flatley Discovery Lab LLCNULLCompleted18 Years55 YearsAll11Phase 1United Kingdom
290EUCTR2016-002749-42-DK
(EUCTR)
15/12/201711/10/2017Study of Aztreonam for Inhalation in Children with Cystic Fibrosis and New Infection of the Airways by Pseudomonas aeruginosa bacteriaRandomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonization - ALPINE2 (Aztreonam Lysine for Pseudomonas Infection Eradication 2) Cystic fibrosis and new onset lower respiratory tract culture positive for Pseudomonas aeruginosa
MedDRA version: 21.1;Level: LLT;Classification code 10068288;Term: Cystic fibrosis pulmonary exacerbation;System Organ Class: 100000004862;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Cayston
Product Code: AZLI
INN or Proposed INN: AZTREONAM
Gilead Sciences, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
140Phase 3United States;Greece;Spain;Ireland;Austria;Israel;Italy;United Kingdom;France;Belgium;Denmark;Germany;Netherlands
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
291NCT03218917
(ClinicalTrials.gov)
December 1, 201711/7/2017Assessment of INS1007 in Subjects With Non-Cystic Fibrosis BronchiectasisRandomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Center Study to Assess the Efficacy, Safety & Tolerability, and PK of INS1007 Administered Once Daily for 24 Weeks in Subjects With Non-CF Bronchiectasis - The Willow StudyNon-Cystic Fibrosis BronchiectasisDrug: INS1007 10 mg oral tablet;Drug: INS1007 25 mg oral tablet;Drug: Placebo Oral TabletInsmed IncorporatedNULLCompleted18 Years85 YearsAll256Phase 2United States;Australia;Belgium;Bulgaria;Denmark;Germany;Italy;Korea, Republic of;Netherlands;New Zealand;Poland;Singapore;Spain;United Kingdom;Sweden
292NCT03474042
(ClinicalTrials.gov)
November 29, 201715/3/2018GLPG2737 on Top of Orkambi in Subjects With Cystic FibrosisA Phase IIa, Randomized, Double-blind, Placebo-controlled Study to Evaluate GLPG2737 in Orkambi-treated Subjects With Cystic Fibrosis Homozygous for the F508del MutationCystic FibrosisDrug: GLPG2737;Drug: PlaceboGalapagos NVNULLCompleted18 YearsN/AAll22Phase 2Germany
293NCT03219164
(ClinicalTrials.gov)
November 28, 201713/7/2017Safety and Efficacy of 2 Treatment Regimens of Aztreonam for Inhalation Solution in Children With Cystic Fibrosis and New Onset Pseudomonas Aeruginosa InfectionRandomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects With Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas Aeruginosa (PA) Infection/ColonizationPseudomonas Aeruginosa Respiratory Tract Infection;Cystic FibrosisDrug: AZLI;Drug: PlaceboGilead SciencesNULLActive, not recruiting3 Months18 YearsAll149Phase 3United States;Austria;Belgium;Denmark;France;Germany;Greece;Israel;Italy;Netherlands;Spain;United Kingdom
294EUCTR2016-002749-42-IT
(EUCTR)
24/11/201714/09/2017Study of Aztreonam for Inhalation in Children with Cystic Fibrosis and New Infection of the Airways by Pseudomonas aeruginosa bacteriaRandomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonization - ALPINE2 (Aztreonam Lysine for Pseudomonas Infection Eradication 2) Cystic fibrosis and new onset lower respiratory tract culture positive for Pseudomonas aeruginosa
MedDRA version: 20.0;Level: LLT;Classification code 10068288;Term: Cystic fibrosis pulmonary exacerbation;System Organ Class: 100000113915;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Cayston
Product Code: AZLI
INN or Proposed INN: AZTREONAM
Gilead Sciences, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
140Phase 3United States;Greece;Spain;Ireland;Austria;Israel;Italy;United Kingdom;France;Belgium;Denmark;Germany;Netherlands
295EUCTR2016-005110-22-DE
(EUCTR)
20/11/201723/06/2017A clinical study to investigate safety, tolerability and dose of orally inhaled multiple doses of POL6014 in patients with Cystic FibrosisPhase-Ib/IIa study to investigate safety, tolerability, pharmacokinetics and pharmacodynamics of orally inhaled multiple doses of POL6014 in patients with Cystic Fibrosis Cystic fibrosis;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]Product Name: POL6014Santhera Pharmaceuticals (Switzerland) LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
40Phase 1;Phase 2Germany
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
296EUCTR2016-005230-30-IT
(EUCTR)
15/11/201705/11/2020A research study to find out if SPX-101 helps people with Cystic Fibrosis and to find out if it is safe.A Randomized, Double-Blind, Placebo-Controlled Phase II Study to Evaluate the Efficacy and Safety of SPX-101 Inhalation Solution in Subjects with Cystic Fibrosis (HOPE-1 STUDY: HYDRATION FOR OPTIMAL PULMONARY EFFECTIVENESS) - HOPE-1 Study: Hydration for Optimal Pulmonary Effectiveness Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: SPX-101
Product Code: [SPX-101]
INN or Proposed INN: SPX-101
SPYRYX BIOSCIENCES, INC.NULLNot RecruitingFemale: yes
Male: yes
90Phase 2Portugal;France;Canada;Australia;United Kingdom;Italy
297EUCTR2015-004263-36-DE
(EUCTR)
13/11/201706/09/2017Study to assess the effects of inhaled RPL554 in adults with cystic fibrosis.A Phase IIa, randomised, double blind, placebo controlled, three way crossover study to assess the pharmacokinetics of RPL554 administered to adult patients with Cystic Fibrosis. - Study to assess the effects of inhaled RPL554 in patients with cystic fibrosis. Cystic Fibrosis (CF)
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Name: RPL554
Product Code: RPL554
INN or Proposed INN: RPL554
Other descriptive name: RPL554
Verona Pharma plcNULLNot RecruitingFemale: yes
Male: yes
10Phase 2Germany;United Kingdom
298NCT03206788
(ClinicalTrials.gov)
November 11, 201729/6/2017Losartan and Inflammation in Cystic FibrosisLosartan as Anti-inflammatory Therapy to Augment F508del Cystic Fibrosis Transmembrane (CFTR) RecoveryCystic FibrosisDrug: Losartan;Drug: placeboUniversity of MiamiUniversity of Alabama at Birmingham;Children's Hospital Medical Center, Cincinnati;University of Kansas Medical Center;Cystic Fibrosis FoundationTerminated12 YearsN/AAll7Phase 2United States
299EUCTR2017-000540-18-DE
(EUCTR)
08/11/201713/09/2017Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Subjects With Cystic Fibrosis (CF) Who Have Previously Discontinued Orkambi Phase 3b, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety, Efficacy, and Tolerability of Tezacaftor/Ivacaftor (TEZ/IVA) in an Orkambi-experienced Population Who Are Homozygous for the F508del-CFTR Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Vertex Pharmaceuticals IncorporatedNULLNot Recruiting Female: yes
Male: yes
90Phase 3France;United States;Germany
300EUCTR2017-002181-42-DE
(EUCTR)
27/10/201728/07/2017GLPG2737 on top of Orkambi in subjects with cystic fibrosisA Phase IIa, randomized, double-blind, placebo-controlled study to evaluate GLPG2737 in Orkambi-treated subjects with cystic fibrosis homozygous for the F508del mutation Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Name: GLPG2737
Product Code: G1117337
INN or Proposed INN: Not applicable
Other descriptive name: GLPG2737
Galapagos NVNULLNot RecruitingFemale: yes
Male: yes
18Phase 2Germany
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
301EUCTR2016-002749-42-GB
(EUCTR)
09/10/201725/07/2017Study of Aztreonam for Inhalation in Children with Cystic Fibrosis and New Infection of the Airways by Pseudomonas aeruginosa bacteriaRandomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonization - ALPINE2 (Aztreonam Lysine for Pseudomonas Infection Eradication 2) Cystic fibrosis and new onset lower respiratory tract culture positive for Pseudomonas aeruginosa
MedDRA version: 21.1;Level: LLT;Classification code 10068288;Term: Cystic fibrosis pulmonary exacerbation;System Organ Class: 100000004862;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Cayston
Product Code: AZLI
INN or Proposed INN: AZTREONAM
Gilead Sciences, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
140Phase 3United States;Greece;Spain;Ireland;Austria;Israel;United Kingdom;Italy;France;Belgium;Denmark;Germany;Netherlands
302EUCTR2017-000797-11-NL
(EUCTR)
06/10/201730/05/2017A Study of VX-445 in Healthy Subjects and Subjects with Cystic FibrosisA Phase 1/2 Study of VX-445 in Healthy Subjects and Subjects with Cystic Fibrosis Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-445
INN or Proposed INN: VX-445
Other descriptive name: VX-445
INN or Proposed INN: VX-445
Other descriptive name: VX-445
Product Name: tezacaftor/ivacaftor 100mg/150mg
Product Code: VX-661/VX-770
INN or Proposed INN: tezacaftor
Other descriptive name: VX-661
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
80Phase 2United States;Belgium;Australia;Netherlands
303NCT03309358
(ClinicalTrials.gov)
September 28, 20175/10/2017A Study of the Safety and Tolerability of Inhaled SNSP113 in Healthy Subjects and Subjects With Stable Cystic FibrosisA Multiple-Site, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose (SAD) Study to Assess the Safety and Tolerability of Inhaled SNSP113 in Healthy Male Subjects (Part A) and Subjects With Stable Cystic Fibrosis (Part B)Lung Diseases;Pulmonary Disease;Cystic Fibrosis;Cystic Fibrosis Lung;Cystic Fibrosis Pulmonary Exacerbation;Cystic Fibrosis With Exacerbation;Respiratory Tract Disease;Pulmonary Inflammation;Multi-antibiotic Resistance;Antibiotic Resistant Infection;Lung Infection;Lung Infection Pseudomonal;Lung; Infection, Atypical Mycobacterium;Burkholderia Infections;Burkholderia Cepacia Infection;Lung InflammationDrug: Inhaled SNSP113;Drug: Inhaled PlaceboSynspira, Inc.NULLTerminated18 Years50 YearsAll32Phase 1United Kingdom
304NCT02566044
(ClinicalTrials.gov)
September 27, 201718/9/2015Safety, Pharmacokinetics and Pharmacodynamics Study of Inhaled QBW276 in Patients With Cystic FibrosisA Randomized, Double-blind, Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Doses of Inhaled QBW276 in Patients With Cystic FibrosisPulmonary Cystic FibrosisOther: Placebo;Drug: QBW276Novartis PharmaceuticalsNULLCompleted18 YearsN/AAll16Phase 1;Phase 2United States;Germany;United Kingdom
305NCT03181932
(ClinicalTrials.gov)
September 26, 20171/6/2017A Study of AeroVanc for the Treatment of MRSA Infection in CF PatientsA Phase III, Randomized, Double-blind, Placebo-controlled Study of AeroVanc for the Treatment of Persistent Methicillin-resistant Staphylococcus Aureus Lung Infection in Cystic Fibrosis PatientsMRSA;Cystic FibrosisDrug: Vancomycin inhalation powder;Drug: Placebo inhalation powderSavara Inc.NULLActive, not recruiting6 YearsN/AAll200Phase 3United States;Canada
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
306EUCTR2016-002749-42-DE
(EUCTR)
22/09/201702/08/2017Study of Aztreonam for Inhalation in Children with Cystic Fibrosis and New Infection of the Airways by Pseudomonas aeruginosa bacteriaRandomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonization - ALPINE2 (Aztreonam Lysine for Pseudomonas Infection Eradication 2) Cystic fibrosis and new onset lower respiratory tract culture positive for Pseudomonas aeruginosa
MedDRA version: 21.1;Level: LLT;Classification code 10068288;Term: Cystic fibrosis pulmonary exacerbation;System Organ Class: 100000004862;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Cayston
Product Code: AZLI
INN or Proposed INN: AZTREONAM
Gilead Sciences, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
140Phase 3United States;Greece;Spain;Ireland;Austria;Israel;Italy;United Kingdom;France;Belgium;Denmark;Germany;Netherlands
307EUCTR2017-000571-85-GB
(EUCTR)
19/09/201711/05/2017Study of Oral Liprotamase Therapy Of Non-Porcine OriginA Phase 3, Randomized, Open-Label, Assessor-Blind, Non-Inferiority, Active-Comparator Study Evaluating the Efficacy and Safety of Liprotamase in Subjects with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency - RESULT: Reliable, Emergent Solution Using Liprotamase Treatment Pancreatic Exocrine Insufficiency due to Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10073392;Term: Pancreatic exocrine insufficiency;System Organ Class: 100000173123;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: liprotamase
INN or Proposed INN: not assigned
Other descriptive name: LIPASE
INN or Proposed INN: not assigned
Other descriptive name: PROTEASE
INN or Proposed INN: not assigned
Other descriptive name: AMYLASE
Trade Name: PANCREAZE®
Product Name: PANCREAZE®
INN or Proposed INN: not assigned
Other descriptive name: PANCREATIN
ANTHERA Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
150Phase 3United States;Hungary;Poland;Spain;Lithuania;Israel;United Kingdom
308EUCTR2016-002749-42-BE
(EUCTR)
18/09/201724/07/2017Study of Aztreonam for Inhalation in Children with Cystic Fibrosis and New Infection of the Airways by Pseudomonas aeruginosa bacteriaRandomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonization - ALPINE2 (Aztreonam Lysine for Pseudomonas Infection Eradication 2) Cystic fibrosis and new onset lower respiratory tract culture positive for Pseudomonas aeruginosa
MedDRA version: 20.0;Level: LLT;Classification code 10068288;Term: Cystic fibrosis pulmonary exacerbation;System Organ Class: 100000004862;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Cayston
Product Code: AZLI
INN or Proposed INN: AZTREONAM
Gilead Sciences, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
140Phase 3United States;Greece;Spain;Ireland;Austria;Israel;United Kingdom;Italy;France;Belgium;Denmark;Germany;Netherlands
309EUCTR2017-000571-85-LT
(EUCTR)
15/09/201720/07/2017Study of Oral Liprotamase Therapy Of Non-Porcine OriginA Phase 3, Randomized, Open-Label, Assessor-Blind, Non-Inferiority, Active-Comparator Study Evaluating the Efficacy and Safety of Liprotamase in Subjects with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency - RESULT: Reliable, Emergent Solution Using Liprotamase Treatment Pancreatic Exocrine Insufficiency due to Cystic Fibrosis
MedDRA version: 20.0;Level: LLT;Classification code 10073392;Term: Pancreatic exocrine insufficiency;System Organ Class: 100000173123
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: liprotamase
INN or Proposed INN: not assigned
Other descriptive name: LIPASE
INN or Proposed INN: not assigned
Other descriptive name: PROTEASE
INN or Proposed INN: not assigned
Other descriptive name: AMYLASE
Trade Name: PANCREAZE®
Product Name: PANCREAZE®
INN or Proposed INN: not assigned
Other descriptive name: PANCREATIN
ANTHERA Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
150Phase 3United States;Hungary;Poland;Spain;Lithuania;Israel
310NCT02769637
(ClinicalTrials.gov)
September 7, 201710/5/2016Effect of Acid Blockade on Microbiota and Inflammation in Cystic Fibrosis (CF)Effect of Acid Blockade on Microbiota and Inflammation in Cystic Fibrosis (CF)Cystic FibrosisDrug: PPI treatmentUniversity of Colorado, DenverCystic Fibrosis FoundationTerminated10 Years21 YearsAll2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
311NCT03771313
(ClinicalTrials.gov)
September 1, 201711/7/2018Pharmacokinetic and Pharmacodynamic Analysis of Ceftaroline in Children and Adolescents With Cystic FibrosisPharmacokinetic and Pharmacodynamic Analysis of Ceftaroline in Children and Adolescents With Cystic FibrosisCystic FibrosisDrug: CeftarolineChildren's Hospital Medical Center, CincinnatiNULLRecruiting2 Years21 YearsAll24Phase 4United States
312NCT04468100
(ClinicalTrials.gov)
August 30, 20178/7/2020Efficacy and Safety of Tigerase® vs. Pulmozyme® in Patients With Cystic FibrosisA Multicenter Prospective Randomized Comparative Study of Pharmacokinetics, Clinical Efficacy and Safety of Tigerase® (JSC GENERIUM, Russia) vs. Pulmozyme® (Hoffmann-La Roche, Switzerland) as Part of Complex Therapy in Patients With Cystic FibrosisCystic Fibrosis;FibrosisBiological: Tigerase®;Biological: Pulmozyme®AO GENERIUMNULLCompleted18 YearsN/AAll100Phase 3Russian Federation
313EUCTR2015-003040-39-NL
(EUCTR)
28/08/201723/01/2017Dose-finding Study to Assess the Efficacy, Safety and Tolerability of Tobramycin Inhalation Powder in Patients With Non-Cystic Fibrosis Bronchiectasis and Pulmonary P. Aeruginosa InfectionA randomized, blinded, parallel group, multi-center dose-finding study, to assess the efficacy, safety and tolerability of different doses of tobramycin inhalation powder in patients with Non-Cystic Fibrosis Bronchiectasis and pulmonary P. aeruginosa infection Pseudomonas aeruginosa infection in patients with non-cystic fibrosis bronchiectasis
MedDRA version: 20.1;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
MedDRA version: 20.1;Classification code 10070295;Term: Infective exacerbation of bronchiectasis;System Organ Class: 10021881 - Infections and infestations ;Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]
Novartis Pharma Services AGNULLNot Recruiting Female: yes
Male: yes
180Phase 2France;Belgium;Spain;Ireland;Germany;Netherlands;Italy;United Kingdom;Switzerland
314EUCTR2016-005230-30-PT
(EUCTR)
28/08/201731/05/2017A research study to find out if SPX-101 helps people with Cystic Fibrosis and to find out if it is safe.A Randomized, Double-Blind, Placebo-Controlled Phase II Study to Evaluate the Efficacy and Safety of SPX-101 Inhalation Solution in Subjects with Cystic Fibrosis (HOPE-1 STUDY: HYDRATION FOR OPTIMAL PULMONARY EFFECTIVENESS) - HOPE-1 Study: Hydration for Optimal Pulmonary Effectiveness Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: SPX-101
INN or Proposed INN: SPX-101
Product Name: SPX-101
INN or Proposed INN: SPX-101
Spyryx Biosciences, Inc.NULLNot RecruitingFemale: yes
Male: yes
90Phase 2France;Portugal;Canada;Australia;Italy;United Kingdom
315EUCTR2017-000797-11-BE
(EUCTR)
28/08/201715/06/2017A Study of VX-445 in Healthy Subjects and Subjects with Cystic FibrosisA Phase 1/2 Study of VX-445 in Healthy Subjects and Subjects with Cystic Fibrosis Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-445
INN or Proposed INN: VX-445
Other descriptive name: VX-445
INN or Proposed INN: VX-445
Other descriptive name: VX-445
Product Name: tezacaftor/ivacaftor 100mg/150mg
Product Code: VX-661/VX-770
INN or Proposed INN: tezacaftor
Other descriptive name: VX-661
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Product Name: Tezacaftor
Product Code: VX-661
INN or Proposed INN: Tezacaftor (TEZ)
Other descriptive name: VX-661
Product Name: Deuterated Ivacaftor
Product Code: VX-561 (CTP-656)
INN or Proposed INN: VX-561
Other descriptive name: VX-561
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
104Phase 2United States;Belgium;Australia;Netherlands
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
316NCT03278314
(ClinicalTrials.gov)
August 27, 20176/9/2017Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic FibrosisTezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic FibrosisCystic FibrosisDrug: tezacaftor/ivacaftorVertex Pharmaceuticals IncorporatedNULLApproved for marketing12 YearsN/AAllNULL
317NCT03093714
(ClinicalTrials.gov)
August 23, 201716/3/2017A Study to Evaluate Safety, PK and PD of FDL169 in Cystic Fibrosis SubjectsA Randomized, Double-Blind, Placebo-Controlled, Parallel Study to Evaluate Safety, Pharmacokinetics (PK) and Pharmacodynamics(PD) of FDL169 in Cystic Fibrosis (CF) Subjects Homozygous for the F508del-CFTR MutationCystic FibrosisDrug: FDL169;Drug: PlaceboFlatley Discovery Lab LLCNULLCompleted18 Years85 YearsAll27Phase 1Australia;Czechia;Germany;United Kingdom
318NCT03277196
(ClinicalTrials.gov)
August 16, 20176/9/2017A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive MutationA Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive MutationCystic FibrosisDrug: IvacaftorVertex Pharmaceuticals IncorporatedNULLEnrolling by invitationN/A24 MonthsAll75Phase 3United States;Australia;Canada;Germany;Ireland;United Kingdom
319EUCTR2016-004558-13-PL
(EUCTR)
14/08/201730/06/2017A double-blind, placebo controlled, multicentre, clinical trial to investigate the efficacy and safety of 12 months of therapy with inhaled colistimethate sodium in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa)A double-blind, placebo controlled, multicentre, clinical trial to investigate the efficacy and safety of 12 months of therapy with inhaled colistimethate sodium in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa) - PROMIS II Non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa
MedDRA version: 21.0;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]
Trade Name: Promixin, 1 million International Units (IU) Powder for Nebuliser Solution
Product Name: Colistimethate sodium
INN or Proposed INN: COLISTIMETHATE SODIUM
Other descriptive name: Colistimethate Sodium
Zambon S.p.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
420Phase 3United States;France;Canada;Argentina;Poland
320EUCTR2016-003585-11-GB
(EUCTR)
10/08/201710/07/2017Study to Evaluate the Safety and Efficacy of VX-659 drug in combination with other drugs in Subjects Aged 18 Years and Older With Cystic FibrosisA Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-659 Combination Therapy in Subjects Aged 18 Years and Older With Cystic Fibrosis Cystic fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: VX-659
Product Code: VX-659
INN or Proposed INN: VX-659
Other descriptive name: VX-659
Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Product Name: Tezacaftor/Ivacaftor 100mg/150mg
Product Code: VX-661/VX-770
INN or Proposed INN: Tezacaftor
Other descriptive name: VX-661
INN or Proposed INN: IVACAFTOR
Product Name: Tezacaftor
Product Code: VX-661
INN or Proposed INN: TEZACAFTOR
Product Name: Deuterated Ivacaftor
Product Code: VX-561 (CTP-656)
INN or Proposed INN: Deuterated Ivacaftor
Other descriptive name: VX-561 (CTP-656)
Vertex Pharmaceuticals IncorporatedNULLNot RecruitingFemale: yes
Male: yes
105Phase 2United States;Ireland;Israel;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
321NCT03224351
(ClinicalTrials.gov)
August 8, 201718/7/2017A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic FibrosisA Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-659 Combination Therapy in Subjects Aged 18 Years and Older With Cystic FibrosisCystic FibrosisDrug: TEZ/IVA;Drug: VX-659;Drug: IVA;Drug: Matched Placebos;Drug: TEZ;Drug: VX-561Vertex Pharmaceuticals IncorporatedNULLCompleted18 YearsN/AAll124Phase 2United States;Ireland;Israel;United Kingdom
322NCT03172793
(ClinicalTrials.gov)
August 8, 20179/5/2017Telavancin Pharmacokinetics in Cystic Fibrosis PatientsPharmacokinetics and Tolerability of Telavancin at Differing Dosing Regimens in Cystic Fibrosis Adults Admitted With Acute Pulmonary ExacerbationsCystic FibrosisDrug: Telavancin InjectionJoseph L. Kuti, PharmDCumberland Pharmaceuticals, Inc.Completed18 YearsN/AAll18Phase 4United States
323EUCTR2016-002749-42-AT
(EUCTR)
07/08/201727/07/2017Study of Aztreonam for Inhalation in Children with Cystic Fibrosis and New Infection of the Airways by Pseudomonas aeruginosa bacteriaRandomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonization - ALPINE2 (Aztreonam Lysine for Pseudomonas Infection Eradication 2) Cystic fibrosis and new onset lower respiratory tract culture positive for Pseudomonas aeruginosa
MedDRA version: 21.1;Level: LLT;Classification code 10068288;Term: Cystic fibrosis pulmonary exacerbation;System Organ Class: 100000004862;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Cayston
Product Code: AZLI
INN or Proposed INN: AZTREONAM
Gilead Sciences, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
140Phase 3United States;Greece;Spain;Ireland;Austria;Israel;Italy;United Kingdom;France;Belgium;Denmark;Germany;Netherlands
324EUCTR2016-002749-42-ES
(EUCTR)
04/08/201708/08/2017Study of Aztreonam for Inhalation in Children with Cystic Fibrosis and New Infection of the Airways by Pseudomonas aeruginosa bacteriaRandomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonization - ALPINE2 (Aztreonam Lysine for Pseudomonas Infection Eradication 2) Cystic fibrosis and new onset lower respiratory tract culture positive for Pseudomonas aeruginosa
MedDRA version: 20.0;Level: LLT;Classification code 10068288;Term: Cystic fibrosis pulmonary exacerbation;System Organ Class: 100000113915;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Trade Name: Cayston
Product Code: AZLI
INN or Proposed INN: AZTREONAM
Gilead Sciences, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
140Phase 3France;United States;Greece;Belgium;Spain;Ireland;Austria;Israel;Netherlands;Germany;Italy;United Kingdom
325EUCTR2017-001379-21-GB
(EUCTR)
01/08/201720/07/2017A study to assess the safety and pharmacodynamics of long-term ivacaftor treatment in children less than 24 months of age with cystic fibrosis (a rare hereditary disease that affects the lungs, digestive system and other organs).A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Trade Name: Kalydeco
Product Name: Ivacaftor
Product Code: VX-770
INN or Proposed INN: IVACAFTOR
Vertex Pharmaceuticals IncorporatedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
75Phase 3United States;Canada;Ireland;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
326NCT03229252
(ClinicalTrials.gov)
August 1, 201714/7/2017An Efficacy and Safety Study of SPX-101 Inhalation Solution in Subjects With Cystic FibrosisA Randomized, Double-blind, Placebo-controlled Phase II Study to Evaluate the Efficacy and Safety of SPX-101 Inhalation Solution in Subjects With Cystic Fibrosis (HOPE-1 Study: Hydration for Optimal Pulmonary Effectiveness)Cystic FibrosisDrug: Placebo Inhalation Solution;Drug: SPX-101Spyryx Biosciences, Inc.NULLCompleted18 Years50 YearsAll91Phase 2Canada;France;Italy;Portugal;United Kingdom
327NCT03258424
(ClinicalTrials.gov)
July 28, 201718/8/2017Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO® as Background TherapyA Phase I, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects With Cystic Fibrosis.Cystic FibrosisDrug: PTI-428;Drug: PlaceboProteostasis Therapeutics, Inc.NULLCompleted18 YearsN/AAll15Phase 1United Kingdom
328EUCTR2016-001214-24-DE
(EUCTR)
27/07/201710/04/2017A Multi-center, Randomized, Placebo-Controlled Phase I/II Study Designed to Assess the Safety, Tolerability, and how the body breaks down the drug PTI-428 in Subjects with Cystic FibrosisA Phase I/II Multi-center, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects with Cystic Fibrosis - Proteostasis PTI428 Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: PTI-428
Product Code: PTI-428
Proteostasis TherapeuticsNULLNot RecruitingFemale: yes
Male: yes
132Phase 1;Phase 2France;United States;Czech Republic;Canada;Denmark;Germany;Italy;United Kingdom
329EUCTR2014-004915-35-DE
(EUCTR)
26/07/201714/10/2015Safety and effect of QBW276 in patients with cystic fibrosis A randomized, double blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple doses of inhaled QBW276 in patients with cystic fibrosis - Safety,pharmacokinetics and pharmacodynamics study of inhaledQBW276 in patients with cystic fibrosis Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Novartis Pharma AGNULLNot Recruiting Female: yes
Male: yes
56Phase 1;Phase 2United States;Germany;United Kingdom
330NCT02819856
(ClinicalTrials.gov)
July 21, 201716/6/2016SPI-1005 for Prevention and Treatment of Tobramycin Induced OtotoxicityA Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of SPI-1005 in Cystic Fibrosis (CF) Patients With Acute Pulmonary Exacerbation (APE) Receiving IV Tobramycin at Risk for OtotoxicityOtotoxicityDrug: Placebo;Drug: SPI-1005 Ebselen 200mg Capsule x1;Drug: SPI-1005 Ebselen 200mg Capsule x2;Drug: SPI-1005 Ebselen 200mg Capsule x3Sound Pharmaceuticals, IncorporatedMedical University of South Carolina;Cystic Fibrosis FoundationEnrolling by invitation18 YearsN/AAll80Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
331NCT03068312
(ClinicalTrials.gov)
July 18, 201724/2/2017A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C?T or D1152H CFTR MutationA Randomized, Double-blind, Placebo-controlled, Crossover Study to Evaluate the Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older and Have Either a 3849 + 10KB C?T or D1152H-CFTR MutationCystic FibrosisDrug: Ivacaftor;Drug: PlaceboVertex Pharmaceuticals IncorporatedNULLCompleted6 YearsN/AAll38Phase 3Israel
332NCT03251092
(ClinicalTrials.gov)
July 17, 20177/8/2017Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic FibrosisA Phase 1/2 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults With Cystic FibrosisHealthy Volunteer - Complete;Cystic Fibrosis - CompleteDrug: PTI-808;Drug: Placebo;Drug: PTI-428;Drug: PTI-801Proteostasis Therapeutics, Inc.NULLCompleted18 Years99 YearsAll179Phase 1;Phase 2United States;Australia;Belgium;Canada;Denmark;France;Germany;New Zealand;United Kingdom
333EUCTR2016-005230-30-GB
(EUCTR)
14/07/201703/05/2017A research study to find out if SPX-101 helps people with Cystic Fibrosis and to find out if it is safe.A Randomized, Double-Blind, Placebo-Controlled Phase II Study to Evaluate the Efficacy and Safety of SPX-101 Inhalation Solution in Subjects with Cystic Fibrosis (HOPE-1 STUDY: HYDRATION FOR OPTIMAL PULMONARY EFFECTIVENESS) - HOPE-1 Study: Hydration for Optimal Pulmonary Effectiveness Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: SPX-101
INN or Proposed INN: SPX-101
Product Name: SPX-101
INN or Proposed INN: SPX-101
Spyryx Biosciences, Inc.NULLNot RecruitingFemale: yes
Male: yes
90Phase 2Portugal;France;Canada;Australia;Italy;United Kingdom
334EUCTR2015-002743-33-PT
(EUCTR)
13/07/201710/07/2017A double-blind, placebo controlled, multicentre, clinical trial to investigate the efficacy and safety of 12 months of therapy with inhaled Promixin® (colistimethate sodium) in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa)A double-blind, placebo-controlled, multi-centre, clinical trial to investigate the efficacy and safety of 12 months of therapy with inhaled colistimethate sodium in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa) - PROMIS I Non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa
MedDRA version: 20.1;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]
Trade Name: Promixin/Tadim
Product Name: Colistimethate sodium
INN or Proposed INN: COLISTIMETHATE SODIUM
Other descriptive name: Colistimethate Sodium
Zambon S.p.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
420Phase 3Portugal;Greece;Spain;Israel;Switzerland;United Kingdom;Italy;France;Belgium;Australia;Germany;Netherlands;New Zealand
335EUCTR2016-001214-24-DK
(EUCTR)
10/07/201728/04/2017A Multi-center, Randomized, Placebo-Controlled Phase I/II Study Designed to Assess the Safety, Tolerability, and how the body breaks down the drug PTI-428 in Subjects with Cystic FibrosisA Phase I/II Multi-center, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects with Cystic Fibrosis - Proteostasis PTI428 Cystic Fibrosis
MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: PTI-428
Product Code: PTI-428
Product Code: PTI-428
Product Code: PTI-428
Proteostasis TherapeuticsNULLNot RecruitingFemale: yes
Male: yes
144Phase 1;Phase 2United States;France;Czech Republic;Canada;Denmark;Germany;United Kingdom;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
336NCT03450720
(ClinicalTrials.gov)
June 28, 201723/2/2018Pharmacokinetics of GLPG2737 in Male Subjects With Cystic FibrosisEvaluation of the Pharmacokinetics, Safety and Tolerability of a Single Dose of GLPG2737 Administered as Oral Suspension in Male Subjects With Cystic FibrosisCystic FibrosisDrug: GLPG2737 single doseGalapagos NVNULLCompleted18 YearsN/AMale6Phase 1Belgium
337NCT03173573
(ClinicalTrials.gov)
June 27, 201730/5/2017A Study to Assess the Safety, Tolerability and PK Profile of FDL176 in Healthy and CF ParticipantsA Five Part Phase 1 Study to Assess the Safety, Tolerability and Pharmacokinetic (PK) Profile of Single and Repeat Oral Doses of FDL176 in Healthy and Cystic Fibrosis (CF) ParticipantsCystic FibrosisDrug: FDL176;Drug: PlaceboFlatley Discovery Lab LLCNULLCompleted18 Years55 YearsAll109Phase 1Australia
338EUCTR2016-004033-25-ES
(EUCTR)
19/06/201731/03/2017Randomized clinical trial to assess the effect of nebulizad bicarbonate on bacterial infections in patients with cystic fibrosisEfect of nebulized bicarbonate on bacterial infections in patients with cystic fibrosis. Randomized clinical trial Cystic Fibrosis
MedDRA version: 20.0;Level: LLT;Classification code 10074550;Term: Preventive antimicrobial therapy in cystic fibrosis;System Organ Class: 100000004865
MedDRA version: 20.0;Classification code 10011764;Term: Cystic fibrosis NOS;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Product Name: Sodium bicarbonate
INN or Proposed INN: Sodium Bicarbonate
Other descriptive name: SODIUM BICARBONATE BP
Product Name: sodium chloride
INN or Proposed INN: Sodium chloride
Other descriptive name: SODIUM CHLORIDE
Product Name: sodium chloride
INN or Proposed INN: Sodium chloride
Other descriptive name: SODIUM CHLORIDE
Product Name: Water for injections
INN or Proposed INN: water for injections
Other descriptive name: WATER FOR INJECTIONS, EP
Fundació Parc TaulíNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 2Spain
339EUCTR2017-000571-85-HU
(EUCTR)
15/06/201713/04/2017Study of Oral Liprotamase Therapy Of Non-Porcine OriginA Phase 3, Randomized, Open-Label, Assessor-Blind, Non-Inferiority, Active-Comparator Study Evaluating the Efficacy and Safety of Liprotamase in Subjects with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency - RESULT: Reliable, Emergent Solution Using Liprotamase Treatment Pancreatic Exocrine Insufficiency due to Cystic Fibrosis;Therapeutic area: Diseases [C] - Immune System Diseases [C20]Product Name: liprotamase
INN or Proposed INN: not assigned
Other descriptive name: LIPASE
INN or Proposed INN: not assigned
Other descriptive name: PROTEASE
INN or Proposed INN: not assigned
Other descriptive name: AMYLASE
Trade Name: PANCREAZE®
Product Name: PANCREAZE®
INN or Proposed INN: not assigned
Other descriptive name: PANCREATIN
ANTHERA Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
192Phase 3United States;Hungary;Poland;Spain;Lithuania;Israel
340NCT03078088
(ClinicalTrials.gov)
June 15, 20171/3/2017Airway Alkalinization and Nasal ColonizationAirway Alkalinization and Nasal ColonizationHealthy Subjects;Cystic FibrosisDrug: Tham;Drug: SalineLakshmi DurairajNULLCompleted16 YearsN/AAll32Phase 1United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
341NCT03020719
(ClinicalTrials.gov)
June 14, 201711/1/2017The Role of Oral Glutathione on Growth Parameters in Children With Cystic FibrosisA Multi Center Placebo Controlled Double Blind Randomized Study Evaluating the Role of Oral Glutathione on Growth Parameters in Children With Cystic FibrosisCystic FibrosisDrug: Oral Glutathione;Drug: PlaceboUniversity of MinnesotaCystic Fibrosis FoundationCompleted2 Years11 YearsAll60Phase 2United States
342NCT03066453
(ClinicalTrials.gov)
June 13, 201723/2/2017Evaluation of Short Antibiotic Combination Courses Followed by Aerosols in Cystic FibrosisEvaluation of an Antibiotic Treatment With 14 Days of Intravenous Tobramycin Versus the Same Antibiotic Associated With 5 Days of Intravenous Tobramycin Followed by Tobramycin Aerosol for 9 Days in Cystic Fibrosis.Cystic FibrosisDrug: Tobi Inhalant Product;Drug: NebcinUniversity Hospital, LilleNULLRecruiting8 YearsN/AAll97Phase 3France
343NCT03154541
(ClinicalTrials.gov)
June 6, 20173/5/2017SynRinse Irrigation Pilot (SIP) TrialSynRinse Irrigation Pilot (SIP) TrialSinusitis, Chronic;Cystic Fibrosis With Other ManifestationsDrug: SynrinseUniversity of WashingtonNULLCompleted18 YearsN/AAll30Phase 1;Phase 2United States
344EUCTR2017-000571-85-ES
(EUCTR)
01/06/201721/04/2017Study of Oral Liprotamase Therapy Of Non-Porcine OriginA Phase 3, Randomized, Open-Label, Assessor-Blind, Non-Inferiority, Active-Comparator Study Evaluating the Efficacy and Safety of Liprotamase in Subjects with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency - RESULT: Reliable, Emergent Solution Using Liprotamase Treatment Pancreatic Exocrine Insufficiency due to Cystic Fibrosis;Therapeutic area: Diseases [C] - Immune System Diseases [C20]Product Name: liprotamase
INN or Proposed INN: not assigned
Other descriptive name: LIPASE
INN or Proposed INN: not assigned
Other descriptive name: PROTEASE
INN or Proposed INN: not assigned
Other descriptive name: AMYLASE
Trade Name: PANCREAZE®
Product Name: PANCREAZE®
INN or Proposed INN: not assigned
Other descriptive name: PANCREATIN
ANTHERA Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
150Phase 3United States;Hungary;Poland;Spain;Lithuania;Israel
345NCT03093974
(ClinicalTrials.gov)
June 1, 20179/3/2017Long Term Efficacy and Safety of Inhaled Colistimethate Sodium in Bronchiectasis Subjects With Chronic Pseudomonas Aeruginosa Infection.A Double-blind, Placebo-controlled, Multi-centre, Clinical Trial to Investigate the Efficacy and Safety of 12 Months of Therapy With Inhaled Colistimethate Sodium in the Treatment of Subjects With Non-cystic Fibrosis Bronchiectasis Chronically Infected With Pseudomonas Aeruginosa (P. Aeruginosa)Non Cystic Fibrosis BronchiectasisDrug: Colistimethate Sodium;Drug: Saline SolutionZambon SpANULLActive, not recruiting18 YearsN/AAll420Phase 3Australia;Belgium;Germany;Greece;Israel;Italy;Netherlands;New Zealand;Portugal;Spain;Switzerland;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
346NCT03056989
(ClinicalTrials.gov)
May 31, 201715/2/2017A Safety Study of SPX-101 Inhalation Solution in Subjects With Cystic FibrosisA Phase 1, Single-Center, Open-Label Safety Study of SPX-101 Inhalation Solution in Subjects With Cystic FibrosisCystic FibrosisDrug: SPX-101Spyryx Biosciences, Inc.NULLCompleted18 Years50 YearsAll5Phase 1Canada
347EUCTR2016-004477-40-ES
(EUCTR)
31/05/201710/03/2017A study looking at the safety, tolerability and efficacy of the study drug GLPG2222 in patients with cystic fibrosis who have the F508del CFTR mutation on both allelesA Phase IIa, randomized, double-blind, placebo-controlled study to evaluate multiple doses of GLPG2222 in subjects with Cystic Fibrosis who are homozygous for the F508del mutation Cystic Fibrosis
MedDRA version: 19.1;Level: PT;Classification code 10011762;Term: Cystic fibrosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Name: GLPG2222
Product Code: G957389
INN or Proposed INN: Not applicable
Other descriptive name: GLPG2222
Product Name: GLPG2222
Product Code: G957389
INN or Proposed INN: Not applicable
Other descriptive name: GLPG2222
Product Name: GLPG2222
Product Code: G957389
INN or Proposed INN: Not applicable
Other descriptive name: GLPG2222
Galapagos NVNULLNot RecruitingFemale: yes
Male: yes
50Phase 2Serbia;United States;Belgium;Spain;Netherlands;United Kingdom
348EUCTR2016-004558-13-FR
(EUCTR)
30/05/201720/03/2017A double-blind, placebo controlled, multicentre, clinical trial to investigate the efficacy and safety of 12 months of therapy with inhaled Promixin® (colistimethate sodium) in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa)A double-blind, placebo controlled, multicentre, clinical trial to investigate the efficacy and safety of 12 months of therapy with inhaled Promixin® (colistimethate sodium) in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa) - PROMIS II Non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa
MedDRA version: 19.1;Level: PT;Classification code 10006445;Term: Bronchiectasis;System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders;Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]
Trade Name: Promixin, 1 million International Units (IU) Powder for Nebuliser Solution
Product Name: Colistimethate sodium
INN or Proposed INN: COLISTIMETHATE SODIUM
Other descriptive name: Colistimethate Sodium
Zambon S.p.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
264Phase 3United States;France;Canada;Poland
349NCT02950805
(ClinicalTrials.gov)
May 30, 201723/8/2016A Study to Assess the Effect of AZD5634 on Mucociliary Clearance, Safety, Tolerability and Pharmacokinetic Parameters in Patients With Cystic FibrosisA Phase 1b Randomized Blinded Placebo-Controlled, Cross-Over Study to Assess the Effect of AZD5634 on Mucociliary Clearance as Well as Safety, Tolerability, and Pharmacokinetic Parameters Following Single Inhaled Dose Administration to Patients With Cystic Fibrosis.Pulmonary/Respiratory DiseasesDrug: Placebo;Drug: AZD5634AstraZenecaParexelCompleted18 Years60 YearsAll9Phase 1United States
350EUCTR2015-002743-33-DE
(EUCTR)
29/05/201701/03/2017A clinical trial to investigate the efficacy and safety of 12 months of therapy with inhaled colistimethate sodium in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa (P. aeruginosa)A double-blind, placebo-controlled, multi-centre, clinical trial to investigate the efficacy and safety of 12 months of therapy with inhaled colistimethate sodium in the treatment of subjects with non-<