299. 嚢胞性線維症 Cystic fibrosis Clinical trials / Disease details
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 | NCT05344508 (ClinicalTrials.gov) | June 30, 2023 | 6/4/2022 | An Expanded Access Study to Assess Brensocatib for Participants With Non-Cystic Fibrosis Bronchiectasis | Brensocatib in Patients With Non-Cystic Fibrosis Bronchiectasis | Non-Cystic Fibrosis Bronchiectasis | Drug: Brensocatib | Insmed Incorporated | NULL | Available | 18 Years | 85 Years | All | NULL | ||
| 2 | NCT05663255 (ClinicalTrials.gov) | March 1, 2023 | 15/12/2022 | Effectiveness of CFTR Modulators According to Co-therapy | Is Effectiveness of CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) Modulators Impacted by Co-therapy? A Population-based Comparative Effectiveness Study Using Data From French Cystic Fibrosis Registry Linked to French National Healthcare Insurance Database | Cystic Fibrosis | Drug: Discontinuation of respiratory co-therapy (azithromycin, RhDNase, or inhaled antibiotics) | Hospices Civils de Lyon | NULL | Not yet recruiting | 6 Years | N/A | All | 1990 | NULL | |
| 3 | NCT05696912 (ClinicalTrials.gov) | January 30, 2023 | 7/12/2022 | Functional Tests to Resolve Unsolved Rare Diseases. Rares. | Resolving Unsolved Rare Diseases : Functional Tests and New Diagnosis Strategy to Study Genetic Variants From High-throughput Sequencing (RID) | Intellectual Disability;Rubinstein-Taybi Syndrome;Cystic Fibrosis;Congenital Heart Defect;Periventricular Nodular Heterotopia;Neurodegeneration With Brain Iron Accumulation (NBIA);Albinism | Genetic: Ex-vivo approach concerning 25 patients;Genetic: In-vitro approach concerning 25 patients | University Hospital, Bordeaux | NULL | Recruiting | N/A | N/A | All | 50 | N/A | France |
| 4 | NCT04702360 (ClinicalTrials.gov) | June 23, 2021 | 6/1/2021 | ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation | ELX/TEZ/IVA Expanded Access Program for Patients 6 Through 11 Years of Age With Cystic Fibrosis Who Have At Least One F508del Mutation | Cystic Fibrosis | Drug: ELX/TEZ/IVA;Drug: IVA | Vertex Pharmaceuticals Incorporated | NULL | Approved for marketing | 6 Years | 11 Years | All | NULL | ||
| 5 | NCT04058210 (ClinicalTrials.gov) | July 21, 2020 | 13/8/2019 | VX-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 Mutation | Cystic Fibrosis | Drug: ELX/TEZ/IVA;Drug: IVA | Vertex Pharmaceuticals Incorporated | NULL | Approved for marketing | 12 Years | N/A | All | NULL | ||
| 6 | NCT04038710 (ClinicalTrials.gov) | September 5, 2019 | 26/7/2019 | Clinical 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 Fibrosis | Drug: Triple combination therapy | National Jewish Health | NULL | Completed | 12 Years | N/A | All | 8 | United States | |
| 7 | NCT03278314 (ClinicalTrials.gov) | August 27, 2017 | 6/9/2017 | Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis | Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis | Cystic Fibrosis | Drug: tezacaftor/ivacaftor | Vertex Pharmaceuticals Incorporated | NULL | Approved for marketing | 12 Years | N/A | All | NULL | ||
| 8 | EUCTR2016-002851-92-CZ (EUCTR) | 05/10/2016 | 02/08/2016 | NA | An Open-Label Study Evaluating the Efficacy and Safety of Liprotamase in Subjects with Exocrine Pancreatic Insufficiency due to Cystic Fibrosis - EASY: Extended Access to Sollpura over Years | Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency;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 | ANTHERA Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 4 | United States;Hungary;Czech Republic;Canada;Spain;Poland;Israel | ||
| 9 | EUCTR2016-002851-92-ES (EUCTR) | 19/09/2016 | 08/08/2016 | NA | An Open-Label Study Evaluating the Efficacy and Safety of Liprotamase in Subjects with Exocrine Pancreatic Insufficiency due to Cystic Fibrosis - EASY: Extended Access to Sollpura over Years | Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Liprotamasa INN or Proposed INN: SIN ASIGNAR Other descriptive name: LIPASA INN or Proposed INN: SIN ASIGNAR Other descriptive name: PROTEASA INN or Proposed INN: SIN ASIGNAR Other descriptive name: AMILASA | ANTHERA Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | United States;Czech Republic;Hungary;Canada;Poland;Spain;Israel | ||
| 10 | EUCTR2016-002851-92-HU (EUCTR) | 08/09/2016 | 25/07/2016 | NA | An Open-Label Study Evaluating the Efficacy and Safety of Liprotamase in Subjects with Exocrine Pancreatic Insufficiency due to Cystic Fibrosis - EASY: Extended Access to Sollpura over Years | Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency;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 | ANTHERA Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 70 | Phase 3 | United States;Hungary;Canada;Spain;Israel | ||
| 11 | NCT02823964 (ClinicalTrials.gov) | September 2016 | 29/6/2016 | EASY: Extended Access to Sollpura Over Years | An Open-Label Study Evaluating the Efficacy and Safety of Liprotamase in Subjects With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis | Exocrine Pancreatic Insufficiency;Cystic Fibrosis | Drug: Liprotamase | Anthera Pharmaceuticals | NULL | Completed | 7 Years | N/A | All | 25 | Phase 4 | United States;Czechia;Hungary;Israel;Poland;Spain;Czech Republic |
| 12 | NCT02310789 (ClinicalTrials.gov) | July 31, 2015 | 3/9/2014 | (Study: Vertex IIS) Does Ivacaftor Alter Wild Type CFTR-Open Probability In The Sweat Gland Secretory Coil? | (Study: Vertex IIS) A Study To Access the Effects of Ivacaftor on Wild Type CFTR-Open Probability (PO) In The Sweat Gland Secretory Coil | Cystic Fibrosis | Drug: Ivacaftor;Drug: ß-Adrenergic cocktail;Drug: Pilocarpine Nitrate 5%;Device: Macroduct sweat stimulator | Richard Barry Moss | NULL | Completed | 18 Years | N/A | All | 8 | N/A | United States |
| 13 | EUCTR2013-005481-19-DK (EUCTR) | 27/05/2014 | 27/05/2014 | High Dose Antioxidant Treatment for Patients with Cystic Fibrosis | High Dose Antioxidant Treatment for Patients with Cystic Fibrosis | Cystic Fibrosis MedDRA version: 17.0;Level: LLT;Classification code 10068288;Term: Cystic fibrosis pulmonary exacerbation;System Organ Class: 100000004862;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Trade Name: Mucolysin INN or Proposed INN: ACETYLCYSTEINE Other descriptive name: ACETYLCYSTEINE Trade Name: Mucomyst INN or Proposed INN: ACETYLCYSTEINE Other descriptive name: ACETYLCYSTEINE Trade Name: ACC INN or Proposed INN: ACETYLCYSTEINE Other descriptive name: ACETYLCYSTEINE Trade Name: Tad INN or Proposed INN: GLUTATHIONE Other descriptive name: GLUTATHIONE SODIUM | Aarhus University Hospital | NULL | Not Recruiting | Female: yes Male: yes | Phase 2 | Denmark | |||
| 14 | NCT01381289 (ClinicalTrials.gov) | June 2011 | 22/6/2011 | VX-770 Expanded Access Program | VX-770 Expanded Access Program (EAP) | Cystic Fibrosis | Drug: VX-770 | Vertex Pharmaceuticals Incorporated | NULL | Approved for marketing | 6 Years | N/A | Both | N/A | NULL | |
| 15 | EUCTR2007-001401-15-DK (EUCTR) | 21/05/2010 | 14/04/2010 | Treatment of patients with cystic fibrosis with N-acetylcysteine | Treatment of patients with cystic fibrosis with N-acetylcysteine | Patients with cystic fibrosis and chronic Pseudomonas lung infection MedDRA version: 12.1;Level: LLT;Classification code 10011763;Term: Cystic fibrosis lung | Trade Name: ACC600Tabs Product Name: N-acetylcystein Product Code: R05C B01 INN or Proposed INN: ACETYLCYSTEINE | Rigshospitalet | NULL | Not Recruiting | Female: yes Male: yes | Phase 4 | Denmark | |||
| 16 | NCT00989807 (ClinicalTrials.gov) | September 2009 | 2/10/2009 | Expanded Access Program for Aztreonam Lysine for Inhalation in Canadian Patients With Cystic Fibrosis | Expanded Access for Aztreonam Lysine for Inhalation in Canadian Patients With Cystic Fibrosis and Pseudomonas Aeruginosa Airway Infection Who Have Limited Treatment Options and Are at Risk for Disease Progression | Cystic Fibrosis;Pseudomonas Aeruginosa | Drug: Aztreonam lysine | Gilead Sciences | NULL | Approved for marketing | 6 Years | N/A | Both | N/A | Canada | |
| 17 | EUCTR2006-006693-24-FR (EUCTR) | 23/08/2007 | 16/05/2007 | A Randomized, Double-Blind, Placebo-Controlled, Dose-finding Study to Evaluate the Efficacy and Safety of Aerosolized Moli1901 in Adolescents (12 Years of Age or Older) and Adults with Cystic Fibrosis | A Randomized, Double-Blind, Placebo-Controlled, Dose-finding Study to Evaluate the Efficacy and Safety of Aerosolized Moli1901 in Adolescents (12 Years of Age or Older) and Adults with Cystic Fibrosis | Cystic fibrosis is the most common fatal inherited disease in the Caucasian population, affecting about 4 in 10.000 children. In Cystic Fibrosis chloride transport accross the respiratory epithelium is deficient, so the mucus contains less water and its viscosity is abnormally increased. Moli 1901 corrects the abnormal transport of chloride thereby reducing the formation of mucus plugs and improving clearance. | Product Name: Moli 1901 (2622U90, duramycin) Product Code: Moli 1901 | AOP Orphan Pharmaceuticals AG | NULL | Not Recruiting | Female: yes Male: yes | 160 | Phase 2;Phase 3 | France;Hungary;Czech Republic;Poland;Spain;Austria;Germany;Italy;Sweden | ||
| 18 | NCT00499720 (ClinicalTrials.gov) | October 2003 | 9/7/2007 | Aztreonam Lysine for Inhalation in Patients With Cystic Fibrosis and Pseudomonas Aeruginosa Airway Infection | Expanded Access Program for Aztreonam Lysine for Inhalation in Patients With Cystic Fibrosis and Pseudomonas Aeruginosa Airway Infection Who Have Limited Treatment Options and Are at Risk for Disease Progression | Cystic Fibrosis;Pseudomonas Aeruginosa Airway Infection | Drug: Aztreonam Lysine for Inhalation | Gilead Sciences | NULL | Approved for marketing | 6 Years | N/A | Both | N/A | United States;Puerto Rico | |
| 19 | EUCTR2016-002851-92-PL (EUCTR) | 02/08/2016 | NA | An Open-Label Study Evaluating the Efficacy and Safety of Liprotamase in Subjects with Exocrine Pancreatic Insufficiency due to Cystic Fibrosis - EASY: Extended Access to Sollpura over Years | Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency;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 | ANTHERA Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 3 | United States;Czech Republic;Hungary;Canada;Spain;Poland;Israel | |||
| 20 | EUCTR2015-000397-36-Outside-EU/EEA (EUCTR) | 03/02/2015 | Program to Give the Drug Aztreonamn Lysine for Inhalation to Canadian Patients with Cystic Fibrosis and infection by the Bacteria Pseudomonas aeruginosa Who Have Limited Treatment Options and are at Risk for Disease Progression | Expanded Access Program for Aztreonam Lysine for Inhalation in Canadian Patients with Cystic Fibrosis and Pseudomonas aeruginosa Airway Infection Who Have Limited Treatment Options and are at Risk for Disease Progression | Adult and paediatric cystic fibrosis (CF) patients with pulmonary Pseudomonas aeruginosa (PA) infection. MedDRA version: 17.1;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: Cayston INN or Proposed INN: AZTREONAM | Gilead Sciences, Inc. | NULL | NA | Female: yes Male: yes | 45 | Canada | ||||
| 21 | EUCTR2008-002673-13-DE (EUCTR) | 27/08/2008 | Anti-inflammatory pulmonal therapy of CF-patients with Amitriptyline and Placebo | Anti-inflammatory pulmonal therapy of CF-patients with Amitriptyline and Placebo | Deficiency of the Cftr-molecule results in accumulation of ceramide in respiratory epithelial cells. Ceramide triggers a pro-inflammatory and -apoptotic status in cystic fibrosis patients. Amitriptyline reduces ceramide levels in the lung tissue, normalises the activity of cytokines and prevents constitutive cell death of epithelial cells. Amitriptyline prevents pulmonary infections of CF-mice with P. aeruginosa. Amitriptyline may result in an improved lung function of cystic fibrosis patients. | Trade Name: Amitriptylin-CT Product Name: Amitriptyline INN or Proposed INN: Amitriptyline | PSKS | NULL | Not Recruiting | Female: yes Male: yes | Phase 2 | Germany | ||||
| 22 | EUCTR2015-000396-26-Outside-EU/EEA (EUCTR) | 02/02/2015 | Program to Give the Drug Aztreonam Lysine for Inhalation to Patients with Cystic Fibrosis and infection by the Bacteria Pseudomonas aeruginosa Who Have Limited Treatment Options and are at Risk for Disease Progression | Expanded Access Program for Aztreonam Lysine for Inhalation in Patients with Cystic Fibrosis and Pseudomonas aeruginosa Airway Infection Who Have Limited Treatment Options and are at Risk for Disease Progression | Adult and paediatric cystic fibrosis (CF) patients with pulmonary Pseudomonas aeruginosa (PA) infection.;Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08] | Trade Name: Cayston INN or Proposed INN: AZTREONAM | Gilead Sciences, Inc. | NULL | NA | Female: yes Male: yes | 603 | United States |