Orkambi ( DrugBank: - )
1 disease
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 299 | 嚢胞性線維症 | 21 |
299. 嚢胞性線維症
臨床試験数 : 1,695 / 薬物数 : 1,527 - (DrugBank : 268) / 標的遺伝子数 : 111 - 標的パスウェイ数 : 174
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2019-001314-41-NL (EUCTR) | 06/05/2019 | 06/05/2019 | This 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 | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 4 | Netherlands | |||
| 2 | NCT03795363 (ClinicalTrials.gov) | April 10, 2019 | 28/12/2018 | Orkambi Treatment in 2 to 5 Year Old Children With CF | Nutritional Impact of Orkambi Treatment in 2 to 5 Year Old Children Homozygous for F508del Mutations | Cystic Fibrosis | Drug: Orkambi | Children's Hospital of Philadelphia | Vertex Pharmaceuticals Incorporated | Completed | 2 Years | 5 Years | All | 28 | United States | |
| 3 | NCT03956589 (ClinicalTrials.gov) | October 1, 2018 | 2/5/2019 | Functional Respiratory Imaging and Orkambi in CF | Functional 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 Phe508del | Cystic Fibrosis | Drug: Orkambi | University Hospital, Antwerp | NULL | Terminated | 12 Years | N/A | All | 12 | Phase 4 | Belgium |
| 4 | EUCTR2017-003761-99-DE (EUCTR) | 27/06/2018 | 19/02/2018 | A Study of the Effects of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del | An 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 Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 2 | Germany | ||
| 5 | EUCTR2018-001573-24-BE (EUCTR) | 11/06/2018 | 08/05/2018 | Evaluation 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 imaging | Functional 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 Hospital | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 4 | Belgium | ||
| 6 | NCT03474042 (ClinicalTrials.gov) | November 29, 2017 | 15/3/2018 | GLPG2737 on Top of Orkambi in Subjects With Cystic Fibrosis | A 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 | Drug: GLPG2737;Drug: Placebo | Galapagos NV | NULL | Completed | 18 Years | N/A | All | 22 | Phase 2 | Germany |
| 7 | EUCTR2017-000540-18-DE (EUCTR) | 08/11/2017 | 13/09/2017 | Study 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] | 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 Other descriptive name: VX-770 Trade Name: Kalydeco 150 mg film-coated tablets Product Name: Ivacaftor Product Code: VX-770 INN or Proposed INN: IVACAFTOR Other descriptive name: VX-770 | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 90 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Germany | ||
| 8 | EUCTR2017-002181-42-DE (EUCTR) | 27/10/2017 | 28/07/2017 | GLPG2737 on top of Orkambi in subjects with cystic fibrosis | A 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 NV | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | Germany | ||
| 9 | NCT03150719 (ClinicalTrials.gov) | May 24, 2017 | 3/5/2017 | A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF) | 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 | Drug: Tezacaftor/Ivacaftor;Drug: Ivacaftor;Drug: Placebo | Vertex Pharmaceuticals Incorporated | NULL | Completed | 12 Years | N/A | All | 98 | Phase 3 | United States;France;Germany |
| 10 | EUCTR2016-004996-33-ES (EUCTR) | 03/05/2017 | 10/03/2017 | A Pilot Study to Evaluate the Use of Smart Adherence Technology to Measure Lumacaftor/Ivacaftor Adherence in CF Subjects Homozygous for the F508del-CFTR Mutation | A Phase 4, Open-label Treatment, Randomized, Multicenter, 2-arm, Parallelgroup, Pilot Study of Adherence to Lumacaftor/Ivacaftor in CF Subjects Homozygous for the F508del-CFTR Mutation | Cystic fibrosis subjects Homozygous for the F508del-CFTR Mutation MedDRA version: 19.1;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] | Trade Name: Orkambi Product Name: Orkambi INN or Proposed INN: Lumacaftor Other descriptive name: LUMACAFTOR INN or Proposed INN: IVACAFTOR Other descriptive name: IVACAFTOR | Vertex Pharmaceuticals Incoporated | NULL | Not Recruiting | Female: yes Male: yes | 75 | Phase 4 | United States;Canada;Spain;Australia;United Kingdom | ||
| 11 | EUCTR2015-004841-13-CZ (EUCTR) | 04/01/2017 | 10/08/2016 | A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation | A Phase 2a, Randomized, Double-blind, Placebo-controlled, Incomplete Block, Crossover Study to Evaluate the Safety and Efficacy of VX-371 in Subjects Aged 12 Years or Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation, and Being Treated With Orkambi | 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 Code: VX-371 in hypertonic saline INN or Proposed INN: Not yet assigned Other descriptive name: VX-371 Product Code: VX-371 in saline INN or Proposed INN: Not yet assigned Other descriptive name: VX-371 Trade Name: Orkambi Product Name: lumacaftor/ivacaftor 200mg/125mg Product Code: VX-809/VX-770 INN or Proposed INN: IVACAFTOR Other descriptive name: Ivacaftor INN or Proposed INN: Lumacaftor Other descriptive name: LUMACAFTOR Product Name: Hypertonic saline INN or Proposed INN: 4.2% NaCl/inhalation solution | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 2 | France;United States;Czech Republic;Ireland;United Kingdom | ||
| 12 | EUCTR2016-001585-29-NL (EUCTR) | 21/12/2016 | 18/07/2016 | Lumacaftor/Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis Who Have an A455E-CFTR Mutation | A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy of Lumacaftor/Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis Who Have an A455E-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] | Trade Name: Orkambi 200 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 | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Netherlands | ||
| 13 | EUCTR2015-004841-13-IE (EUCTR) | 08/08/2016 | 07/06/2016 | A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation | A Phase 2a, Randomized, Double-blind, Placebo-controlled, Incomplete Block, Crossover Study to Evaluate the Safety and Efficacy of VX-371 in Subjects Aged 12 Years or Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation, and Being Treated With Orkambi | 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 Code: VX-371 in hypertonic saline INN or Proposed INN: not yet assigned Other descriptive name: VX-371 Product Code: VX-371 in saline INN or Proposed INN: Not yet assigned Other descriptive name: VX-371 Trade Name: Orkambi Product Name: lumacaftor/ivacaftor 200mg/125mg Product Code: VX-809/VX-770 INN or Proposed INN: IVACAFTOR INN or Proposed INN: Lumacaftor Other descriptive name: LUMACAFTOR Product Name: Hypertonic Saline INN or Proposed INN: 4.2% NaCl/Inhalation solution | Vertex Pharmaceuticals Inc. | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 2 | France;United States;Czech Republic;Ireland;United Kingdom | ||
| 14 | EUCTR2015-004841-13-GB (EUCTR) | 20/07/2016 | 09/06/2016 | A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation | A Phase 2a, Randomized, Double-blind, Placebo-controlled, Incomplete Block, Crossover Study to Evaluate the Safety and Efficacy of VX-371 in Subjects Aged 12 Years or Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation, and Being Treated With Orkambi | 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 Code: VX-371 in hypertonic saline INN or Proposed INN: Not yet assigned Other descriptive name: VX-371 Product Code: VX-371 in saline INN or Proposed INN: Not yet assigned Other descriptive name: VX-371 Trade Name: Orkambi Product Name: lumacaftor/ivacaftor 200mg/125mg Product Code: VX-809/VX-770 INN or Proposed INN: IVACAFTOR INN or Proposed INN: Lumacaftor Other descriptive name: LUMACAFTOR Product Name: Hypertonic saline INN or Proposed INN: 4.2% NaCl/Inhalation solution | Vertex Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 150 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Czech Republic;Ireland;United Kingdom | ||
| 15 | EUCTR2016-000066-34-GB (EUCTR) | 24/06/2016 | 25/07/2017 | A Study of the Effects of Lumacaftor/Ivacaftor on Exercise Tolerance in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation | A Phase 4, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design Study of the Effect of Lumacaftor/Ivacaftor Combination Therapy on Exercise Tolerance in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation. | Cystic fibrosis in patients homozygous for the F508del-CFTR 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] - Respiratory Tract Diseases [C08] | Trade Name: Orkambi Product Name: lumacaftor/ivacaftor Product Code: VX-809/VX-770 INN or Proposed INN: lumacaftor Other descriptive name: LUMACAFTOR INN or Proposed INN: IVACAFTOR | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 66 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yes | Australia;United Kingdom | ||
| 16 | NCT02848560 (ClinicalTrials.gov) | March 2016 | 26/7/2016 | Monitoring Response to Orkambi in Cystic Fibrosis Lung Disease by Inhaled Xenon MRI | Validation of MRI as a Sensitive Tool to Longitudinally Monitor CF Lung Disease Progression and Response to CFTR Modulator Therapy in Young Children With CF | Cystic Fibrosis | Drug: Hyperpolarized Xenon | Children's Hospital Medical Center, Cincinnati | National Heart, Lung, and Blood Institute (NHLBI) | Active, not recruiting | 6 Years | 12 Years | All | 38 | United States | |
| 17 | NCT02709109 (ClinicalTrials.gov) | February 2016 | 29/2/2016 | A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation | A Phase 2a, Randomized, Double-blind, Placebo-controlled, Incomplete Block, Crossover Study to Evaluate the Safety and Efficacy of VX-371 in Subjects Aged 12 Years or Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation, and Being Treated With Orkambi | Cystic Fibrosis | Drug: VX-371;Drug: Saline;Drug: Placebo;Drug: Orkambi | Parion Sciences | Vertex Pharmaceuticals Incorporated | Completed | 12 Years | N/A | All | 142 | Phase 2 | United States;France;Ireland;United Kingdom |
| 18 | NCT03475381 (ClinicalTrials.gov) | January 22, 2016 | 4/1/2018 | Real-life Follow-up of Cystic Fibrosis Patients Treated With Ivacaftor+Lumacaftor (Orkambi*) | Real-life Follow-up of Cystic Fibrosis Patients Treated With Ivacaftor+Lumacaftor (Orkambi*) | Cystic Fibrosis | Drug: Ivacaftor+lumacaftor | Assistance Publique - Hôpitaux de Paris | Effi-Stat;Societe Francaise de la Mucoviscidose | Completed | 12 Years | N/A | All | 852 | France | |
| 19 | EUCTR2018-002624-16-FR (EUCTR) | 21/11/2018 | NA | Validation of respiratory epithelial functional assessment to predict clinical efficacy of Orkambi®. Pathway to personalized therapy in Cystic Fibrosis PREDICT-CF - PREDICT-CF | Homozygous F508del patient aged 12 years or older 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: Orkambi® Product Name: Orkambi® | ASSISTANC-PUBLIQUE-HOPITAUX DE PARIS (AP-HP) | NULL | NA | Female: yes Male: yes | 104 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): yes | France | |||
| 20 | EUCTR2017-000540-18-FR (EUCTR) | 01/08/2017 | Study 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] | 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 Other descriptive name: VX-770 Trade Name: Kalydeco 150 mg film-coated tablets Product Name: Ivacaftor Product Code: VX-770 INN or Proposed INN: IVACAFTOR Other descriptive name: VX-770 | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 90 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;France;Germany | |||
| 21 | EUCTR2015-004841-13-FR (EUCTR) | 23/06/2016 | A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation | A Phase 2a, Randomized, Double-blind, Placebo-controlled, Incomplete Block, Crossover Study to Evaluate the Safety and Efficacy of VX-371 in Subjects Aged 12 Years or Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation, and Being Treated With Orkambi | Cystic Fibrosis MedDRA version: 19.0;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 Code: VX-371 in hypertonic saline INN or Proposed INN: Not yet assigned Other descriptive name: VX-371 Product Code: VX-371 in saline INN or Proposed INN: Not yet assigned Other descriptive name: VX-371 Trade Name: Orkambi Product Name: lumacaftor/ivacaftor 200mg/125mg tablets Product Code: VX-809/VX-770 INN or Proposed INN: IVACAFTOR Other descriptive name: Ivacaftor INN or Proposed INN: Lumacaftor Other descriptive name: LUMACAFTOR Product Name: Hypertonic saline INN or Proposed INN: 4.2% NaCl/inhalation solution | Vertex Pharmaceuticals Inc. | NULL | Not Recruiting | Female: yes Male: yes | 150 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;France;Czech Republic;Ireland;United Kingdom |