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 | EUCTR2017-003319-21-BE (EUCTR) | 08/11/2018 | 21/08/2018 | Study Assessing the Safety, Tolerability and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults with Cystic Fibrosis | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 240 | Phase 1;Phase 2 | United States;France;Canada;Belgium;Denmark;Germany;United Kingdom | ||
| 2 | EUCTR2017-003319-21-DE (EUCTR) | 20/07/2018 | 09/04/2018 | Study Assessing the Safety, Tolerability and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults with Cystic Fibrosis | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 240 | Phase 1;Phase 2 | United States;France;Canada;Belgium;Denmark;Germany;United Kingdom | ||
| 3 | EUCTR2017-003319-21-GB (EUCTR) | 04/06/2018 | 03/07/2018 | Study Assessing the Safety, Tolerability and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults with Cystic Fibrosis | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 240 | Phase 1;Phase 2 | France;United States;Canada;Belgium;Denmark;Germany;United Kingdom | ||
| 4 | NCT03500263 (ClinicalTrials.gov) | January 30, 2018 | 9/4/2018 | Safety, Tolerability, and Pharmacokinetics of PTI-808, PTI-801, and PTI-428 Combination Therapy in Subjects With Cystic Fibrosis | A 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 Fibrosis | Cystic Fibrosis | Drug: PTI-808;Drug: Placebo;Drug: PTI-801;Drug: PTI-428 | Proteostasis Therapeutics, Inc. | NULL | Completed | 18 Years | N/A | All | 12 | Phase 1/Phase 2 | United Kingdom |
| 5 | NCT03251092 (ClinicalTrials.gov) | July 17, 2017 | 7/8/2017 | Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic Fibrosis | A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults With Cystic Fibrosis | Healthy Volunteer - Complete;Cystic Fibrosis - Complete | Drug: PTI-808;Drug: Placebo;Drug: PTI-428;Drug: PTI-801 | Proteostasis Therapeutics, Inc. | NULL | Completed | 18 Years | 99 Years | All | 179 | Phase 1/Phase 2 | United States;Australia;Belgium;Canada;Denmark;France;Germany;New Zealand;United Kingdom |
| 6 | NCT03140527 (ClinicalTrials.gov) | April 10, 2017 | 27/4/2017 | Study Assessing the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis | A Multi-Center, Randomized, Placebo-Controlled, Phase 1, Two-Part Study Designed to Assess the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects With Cystic Fibrosis | Healthy Volunteer;Cystic Fibrosis | Drug: PTI-801;Drug: Placebo;Drug: PTI-808 | Proteostasis Therapeutics, Inc. | NULL | Completed | 18 Years | N/A | All | 171 | Phase 1 | United States;Canada;Denmark;Germany;Sweden;Portugal |