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
Showing 1 to 10 of 11 diseases
| 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 | 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 Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homoz ... | 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 A Phase 2a, Randomized, Double-blind, Placebo-controlled, Incomplete Block, Crossover Study to Evalu ... | 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] Cystic Fibrosis MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;Sy ... | 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 Product Code: VX-371in hypertonic saline INN or Proposed INN: Not yet assigned Other descriptive name: ... | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 2 | France;United States;Czech Republic;Ireland;United Kingdom | ||
| 2 | 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 Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homoz ... | 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 A Phase 2a, Randomized, Double-blind, Placebo-controlled, Incomplete Block, Crossover Study to Evalu ... | 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] Cystic Fibrosis MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;Sy ... | 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 Product Code: VX-371in hypertonic saline INN or Proposed INN: not yet assigned Other descriptive name: ... | Vertex Pharmaceuticals Inc. | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 2 | France;United States;Czech Republic;Ireland;United Kingdom | ||
| 3 | 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 Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homoz ... | 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 A Phase 2a, Randomized, Double-blind, Placebo-controlled, Incomplete Block, Crossover Study to Evalu ... | 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] Cystic Fibrosis MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;Sy ... | 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 Product Code: VX-371in hypertonic saline INN or Proposed INN: Not yet assigned Other descriptive name: ... | 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 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ... | France;United States;Czech Republic;Ireland;United Kingdom | ||
| 4 | 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 Study of the Effects of Lumacaftor/Ivacaftor on Exercise Tolerance in Subjects With Cystic Fibrosi ... | 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. A Phase 4, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design Study of the Effect of Luma ... | 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] Cystic fibrosis in patients homozygous for the F508del-CFTR Mutation MedDRA version: 20.0;Level: PT; ... | 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 Trade Name: Orkambi Product Name: lumacaftor/ivacaftor Product Code: VX-809/VX-770 INN or Proposed INN: ... | 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 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | Australia;United Kingdom | ||
| 5 | EUCTR2010-020413-90-IE (EUCTR) | 08/01/2014 | 13/12/2013 | Study of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation Study of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Cystic Fibrosis ... | A Phase 2, Multicenter, Double-Blind, Placebo-Controlled, Multiple-Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation A Phase 2, Multicenter, Double-Blind, Placebo-Controlled, Multiple-Dose Study to Evaluate the Safety ... | Cystic Fibrosis MedDRA version: 17.0;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] Cystic Fibrosis MedDRA version: 17.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;Sy ... | Product Name: VX-809 Product Code: VX-809, VRT-826809 INN or Proposed INN: lumacaftor Product Name: Kalydeco Product Code: VX-770, VRT-813077 INN or Proposed INN: ivacaftor Product Name: Kalydeco Product Code: VX-770, VRT-813077 INN or Proposed INN: ivacaftor Product Name: Lumacaftor/Ivacaftor Product Code: VX-809/VX-770 INN or Proposed INN: Lumacaftor INN or Proposed INN: Ivacaftor Product Name: VX-809 Product Code: VX-809, VRT-826809 INN or Proposed INN: lumacaftor Product Name: Kal ... | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 120 | Phase 2 | France;United States;Belgium;Ireland;Australia;Germany;United Kingdom | ||
| 6 | EUCTR2010-020413-90-GB (EUCTR) | 06/01/2014 | 10/12/2013 | Study of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation Study of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Cystic Fibrosis ... | A Phase 2, Multicenter, Double-Blind, Placebo-Controlled, Multiple-Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation A Phase 2, Multicenter, Double-Blind, Placebo-Controlled, Multiple-Dose Study to Evaluate the Safety ... | 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] Cystic Fibrosis MedDRA version: 20.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;Sy ... | Product Name: VX-809 Product Code: VX-809, VRT-826809 INN or Proposed INN: lumacaftor Product Name: Kalydeco Product Code: VX-770, VRT-813077 INN or Proposed INN: ivacaftor Product Name: Kalydeco Product Code: VX-770, VRT-813077 INN or Proposed INN: ivacaftor Product Name: Lumacaftor/Ivacaftor Product Code: VX-809/VX-770 INN or Proposed INN: Lumacaftor Product Name: VX-809 Product Code: VX-809, VRT-826809 INN or Proposed INN: lumacaftor Product Name: Kal ... | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 120 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ... | United States;Belgium;Ireland;Australia;Germany;United Kingdom | ||
| 7 | EUCTR2010-020413-90-BE (EUCTR) | 08/02/2011 | 15/10/2010 | Study of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation Study of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Cystic Fibrosis ... | A Phase 2, Multicenter, Double-Blind, Placebo-Controlled, Multiple-Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation A Phase 2, Multicenter, Double-Blind, Placebo-Controlled, Multiple-Dose Study to Evaluate the Safety ... | Cystic Fibrosis MedDRA version: 16.1;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] Cystic Fibrosis MedDRA version: 16.1;Level: PT;Classification code 10011762;Term: Cystic fibrosis;Sy ... | Product Name: VX-809 Product Code: VX-809, VRT-826809 INN or Proposed INN: lumacaftor Product Name: Kalydeco Product Code: VX-770, VRT-813077 INN or Proposed INN: ivacaftor Product Name: Kalydeco Product Code: VX-770, VRT-813077 INN or Proposed INN: ivacaftor Product Name: Lumacaftor/Ivacaftor Product Code: VX-809/VX-770 INN or Proposed INN: Lumacaftor INN or Proposed INN: Ivacaftor Product Name: VX-809 Product Code: VX-809, VRT-826809 INN or Proposed INN: lumacaftor Product Name: Kal ... | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 293 | Phase 2 | United States;Belgium;Ireland;Australia;Germany;United Kingdom;New Zealand | ||
| 8 | EUCTR2010-020413-90-DE (EUCTR) | 06/01/2011 | 15/10/2010 | Study of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation Study of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Cystic Fibrosis ... | A Phase 2, Multicenter, Double-Blind, Placebo-Controlled, Multiple-Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation A Phase 2, Multicenter, Double-Blind, Placebo-Controlled, Multiple-Dose Study to Evaluate the Safety ... | Cystic Fibrosis MedDRA version: 17.0;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] Cystic Fibrosis MedDRA version: 17.0;Level: PT;Classification code 10011762;Term: Cystic fibrosis;Sy ... | Product Name: VX-809 Product Code: VX-809, VRT-826809 INN or Proposed INN: lumacaftor Product Name: Kalydeco Product Code: VX-770, VRT-813077 INN or Proposed INN: ivacaftor Product Name: Kalydeco Product Code: VX-770, VRT-813077 INN or Proposed INN: ivacaftor Product Name: Lumacaftor/Ivacaftor Product Code: VX-809/VX-770 INN or Proposed INN: Lumacaftor INN or Proposed INN: Ivacaftor Product Name: VX-809 Product Code: VX-809, VRT-826809 INN or Proposed INN: lumacaftor Product Name: Kal ... | Vertex Pharmaceuticals Incorporated | NULL | Not Recruiting | Female: yes Male: yes | 293 | Phase 2 | United States;Belgium;Australia;Germany;United Kingdom;New Zealand | ||
| 9 | EUCTR2017-001078-41-Outside-EU/EEA (EUCTR) | 27/03/2017 | A study in people with cystic fibrosis (a rare pulmonary disease) to assess the pharmacokinetics and safety of a combination of two experimental drugs A study in people with cystic fibrosis (a rare pulmonary disease) to assess the pharmacokinetics and ... | A Phase 3, Open-label Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation A Phase 3, Open-label Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Lumacaftor ... | 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Cystic Fibrosis MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, famil ... | Trade Name: lumacaftor/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: lumacaftor/ivacaftor Product Name: lumacaftor/ivacaftor Product Code: VX-809/VX-770 INN or ... | Vertex Pharmaceuticals Incorporated | NULL | NA | Female: yes Male: yes | 58 | Phase 3 | United States;Canada | |||
| 10 | EUCTR2017-001309-34-Outside-EU/EEA (EUCTR) | 29/03/2017 | Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With A ... | A Phase 3b, Open-Label Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Cystic Fibrosis and Advanced Lung Disease, Homozygous for the F508del-CFTR Mutation A Phase 3b, Open-Label Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 ... | Cystic Fibrosis 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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Cystic Fibrosis MedDRA version: 19.1;Level: SOC;Classification code 10010331;Term: Congenital, famil ... | Trade Name: lumacaftor/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: lumacaftor/ivacaftor Product Name: lumacaftor/ivacaftor Product Code: VX-809/VX-770 INN or ... | Vertex Pharmaceuticals Incorporated | NULL | NA | Female: yes Male: yes | 46 | Phase 3 | United States |