ATALUREN ( DrugBank: Ataluren )
6 diseases
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 19 | ライソゾーム病 | 2 |
| 113 | 筋ジストロフィー | 54 |
| 140 | ドラベ症候群 | 1 |
| 246 | メチルマロン酸血症 | 5 |
| 299 | 嚢胞性線維症 | 43 |
| 329 | 無虹彩症 | 2 |
19. ライソゾーム病
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
Showing 1 to 2 of 2 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 | EUCTR2015-003105-41-GB (EUCTR) | 13/10/2015 | 08/09/2015 | Treatment for Nonsense Mutation Mucopolysaccharidosis Type I | CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren (COMPASS) - COMPASS CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Atal ... | Nonsense mutation Mucopolysaccharidosis Type I MedDRA version: 18.1;Level: PT;Classification code 10056886;Term: Mucopolysaccharidosis I;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Nonsense mutation Mucopolysaccharidosis Type I MedDRA version: 18.1;Level: PT;Classification code 10 ... | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product ... | PTC Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 5 | 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 Kingdom | ||
| 2 | EUCTR2014-002596-28-GB (EUCTR) | 28/01/2015 | 12/06/2015 | Treatment for Nonsense Mutation Mucopolysaccharidosis Type I | A Phase 2 Study of Ataluren (PTC124) as Treatment for Nonsense Mutation Mucopolysaccharidosis Type I A Phase 2 Study of Ataluren(PTC124) as Treatment for Nonsense Mutation Mucopolysaccharidosis Type I ... | Nonsense mutation Mucopolysaccharidosis Type I MedDRA version: 20.1;Level: PT;Classification code 10056886;Term: Mucopolysaccharidosis I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] Nonsense mutation Mucopolysaccharidosis Type I MedDRA version: 20.1;Level: PT;Classification code 10 ... | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: ataluren Product ... | PTC Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | Germany;United Kingdom |
113. 筋ジストロフィー
臨床試験数 : 646 / 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170
Showing 1 to 10 of 54 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 | NCT04336826 (ClinicalTrials.gov) | December 29, 2021 | 3/4/2020 | A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From =6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) A Study to Evaluate the Safety and Pharmacokinetics of Atalurenin Participants From =6 Months to <2 ... | An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From =6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy An Open-Label Study Evaluating the Safety and Pharmacokinetics of Atalurenin Children From =6 Months ... | Nonsene Mutation Duchenne Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | NULL | Recruiting | 6 Months | 2 Years | Male | 10 | Phase 2 | United States |
| 2 | JPRN-jRCT2041200087 | 07/01/2021 | 25/12/2020 | An Open-label, Long-Term study of Ataluren in Nonsense Mutation Duchenne Muscular Dystrophy | An Open-Label, Long-Term Safety, Efficacy, and Tolerability Study for Ataluren (PTC124) in Patients with Nonsense Mutation Duchenne Muscular Dystrophy - PTC124-GD-049-DMD An Open-Label, Long-Term Safety, Efficacy, and Tolerability Study for Ataluren(PTC124) in Patients w ... | Nonsense Mutation Duchenne Muscular Dystrophy | Subjects will receive ataluren tid as an oral suspension at respective morning, mid-day, and evening doses of 10 mg/kg, 10 mg/kg, and 20 mg/kg. Subjects will receive atalurentid as an oral suspension at respective morning, mid-day, and evening ... | Penematsa Vinay | NULL | Recruiting | >= 5age old | Not applicable | Male | 25 | Phase 3 | China;Japan |
| 3 | NCT03796637 (ClinicalTrials.gov) | April 11, 2019 | 4/1/2019 | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrop ... | Phase 2, Non-Interventional, Clinical Study to Assess Dystrophin Levels in Subjects With Nonsense Mutation Duchenne Muscular Dystrophy Who Have Been Treated With Ataluren for =9 Months Phase 2, Non-Interventional, Clinical Study to Assess Dystrophin Levels in Subjects With Nonsense Mu ... | Duchenne Muscular Dystrophy | Other: Dystrophin levels;Drug: Ataluren | PTC Therapeutics | NULL | Completed | N/A | N/A | Male | 6 | Phase 2 | United States |
| 4 | NCT03648827 (ClinicalTrials.gov) | December 21, 2018 | 24/8/2018 | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrop ... | Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and After Treatment With Ataluren Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and Af ... | Duchenne Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | NULL | Completed | 2 Years | 7 Years | Male | 20 | Phase 2 | United States |
| 5 | EUCTR2017-001223-49-BG (EUCTR) | 16/01/2018 | 11/10/2017 | A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular Dystrophy A clinical trial to evaluate the safety and efficacy of Atalurenversus placebo in people who have sp ... | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Atalurenin Pati ... | Nonsense Mutation Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] Nonsense Mutation Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 100 ... | Trade Name: Translarna 125 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Trade Name: Translarna 250 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Trade Name: Translarna 1000 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Trade Name: Translarna 125 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 ... | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 340 | Phase 3 | United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argentina;Brazil;Malaysia;Poland;Australia;Bulgaria;China;Japan;Korea, Republic of United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argenti ... | ||
| 6 | NCT03179631 (ClinicalTrials.gov) | July 6, 2017 | 1/6/2017 | Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy | A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Atalurenin Pati ... | Muscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Disease;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, Inborn Muscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Mus ... | Drug: Ataluren;Drug: PLACEBO | PTC Therapeutics | NULL | Active, not recruiting | 5 Years | N/A | Male | 360 | Phase 3 | United States;Australia;Brazil;Bulgaria;Canada;China;Hong Kong;India;Japan;Korea, Republic of;Malaysia;Mexico;Poland;Puerto Rico;Russian Federation;Taiwan;Thailand;Turkey;Argentina;Chile;Jordan;Sri Lanka United States;Australia;Brazil;Bulgaria;Canada;China;Hong Kong;India;Japan;Korea, Republic of;Malays ... |
| 7 | EUCTR2013-005489-20-FR (EUCTR) | 18/10/2016 | 18/06/2015 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy Extension study of atalurenin patients with nonsense mutation Duchenne and Becker muscular dystrophy ... | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] Nonsense mutation dystrophinopathy MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: ... | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ... | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canad ... | ||
| 8 | NCT02819557 (ClinicalTrials.gov) | June 9, 2016 | 16/6/2016 | Study of Ataluren in =2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy | A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged =2 to <5 Years Old With Nonsense Mutation Dystrophinopathy A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren(PTC124®) in Patien ... | Duchenne Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | NULL | Completed | 2 Years | 5 Years | Male | 14 | Phase 2 | United States |
| 9 | EUCTR2013-005489-20-BG (EUCTR) | 25/08/2015 | 26/06/2015 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy Extension study of atalurenin patients with nonsense mutation Duchenne and Becker muscular dystrophy ... | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] Nonsense mutation dystrophinopathy MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: ... | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ... | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canad ... | ||
| 10 | EUCTR2013-005489-20-CZ (EUCTR) | 08/01/2015 | 24/10/2014 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy Extension study of atalurenin patients with nonsense mutation Duchenne and Becker muscular dystrophy ... | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] Nonsense mutation dystrophinopathy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: ... | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ... | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canad ... |
140. ドラベ症候群
臨床試験数 : 116 / 薬物数 : 65 - (DrugBank : 17) / 標的遺伝子数 : 50 - 標的パスウェイ数 : 64
Showing 1 to 1 of 1 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 | NCT02758626 (ClinicalTrials.gov) | November 2016 | 4/4/2016 | Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome | A Phase 2 Randomized, Double-Masked Placebo-Controlled Crossover Safety and Tolerability Study of Ataluren for Drug Resistant Epilepsy in Patients With Nonsense Mutation CDKL5 or Dravet Syndrome A Phase 2 Randomized, Double-Masked Placebo-Controlled Crossover Safety and Tolerability Study of At ... | Epilepsy | Drug: ataluren;Drug: Placebo | NYU Langone Health | PTC Therapeutics | Completed | 2 Years | 12 Years | All | 16 | Phase 2 | United States |
246. メチルマロン酸血症
臨床試験数 : 23 / 薬物数 : 31 - (DrugBank : 8) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 23
Showing 1 to 5 of 5 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 | EUCTR2009-016654-41-IT (EUCTR) | 11/11/2010 | 05/07/2010 | A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia - ND A Phase 2 Study of Ataluren(PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemi ... | A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia - ND A Phase 2 Study of Ataluren(PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemi ... | Nonsense Mutation Methylmalonic Acidemia MedDRA version: 12.1;Level: LLT;Classification code 10064180;Term: Blood methylmalonic acid increased Nonsense Mutation Methylmalonic Acidemia MedDRA version: 12.1;Level: LLT;Classification code 1006418 ... | Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ATALUREN Product ... | PTC THERAPEUTICS, INC. | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | France;United Kingdom;Italy | ||
| 2 | EUCTR2009-016654-41-BE (EUCTR) | 04/11/2010 | 22/12/2010 | Study of ataluren in patients with Methylmalonic Acidemia | A Phase 2 Study of Ataluren (PTC124®) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia A Phase 2 Study of Ataluren(PTC124®) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidem ... | Nonsense Mutation Methylmalonic Acidemia (MMA) MedDRA version: 14.0;Level: LLT;Classification code 10064180;Term: Blood methylmalonic acid increased;System Organ Class: 10022891 - Investigations;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Nonsense Mutation Methylmalonic Acidemia (MMA) MedDRA version: 14.0;Level: LLT;Classification code 1 ... | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ... | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | France;Belgium;Italy;United Kingdom;Switzerland | ||
| 3 | NCT01141075 (ClinicalTrials.gov) | July 19, 2010 | 7/6/2010 | Ataluren for Nonsense Mutation Methylmalonic Acidemia | A Phase 2 Study of Ataluren (PTC124®) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia A Phase 2 Study of Ataluren(PTC124®) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidem ... | Amino Acid Metabolism, Inborn Errors | Drug: Ataluren | PTC Therapeutics | Genzyme, a Sanofi Company | Terminated | 2 Years | N/A | All | 11 | Phase 2 | Belgium;France;Germany;Italy;Switzerland;United Kingdom |
| 4 | EUCTR2009-016654-41-GB (EUCTR) | 01/06/2010 | 17/03/2010 | A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia A Phase 2 Study of Ataluren(PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemi ... | A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia A Phase 2 Study of Ataluren(PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemi ... | Nonsense Mutation Methylmalonic Acidemia MedDRA version: 12.1;Level: LLT;Classification code 10064180;Term: Blood methylmalonic acid increased Nonsense Mutation Methylmalonic Acidemia MedDRA version: 12.1;Level: LLT;Classification code 1006418 ... | Product Name: Ataluren 125mg Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 Product Name: Ataluren 250mg Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 Product Name: Ataluren 1000mg Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 Product Name: Ataluren125mg Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: ... | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 18 | 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;Belgium;Italy;United Kingdom | ||
| 5 | EUCTR2009-016654-41-FR (EUCTR) | 02/03/2010 | 04/01/2010 | A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia A Phase 2 Study of Ataluren(PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemi ... | A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia A Phase 2 Study of Ataluren(PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemi ... | Nonsense Mutation Methylmalonic Acidemia MedDRA version: 12.1;Level: LLT;Classification code 10064180;Term: Blood methylmalonic acid increased Nonsense Mutation Methylmalonic Acidemia MedDRA version: 12.1;Level: LLT;Classification code 1006418 ... | Product Name: Ataluren 125mg Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 Product Name: Ataluren 250mg Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 Product Name: Ataluren 1000mg Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC-0161480; PTC-124; Compound 1a; RPS 2505; PTC-C124; 291844 Product Name: Ataluren125mg Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: ... | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 18 | Phase 2 | France;Belgium;Italy;United Kingdom |
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
Showing 1 to 10 of 43 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 | NCT03256799 (ClinicalTrials.gov) | March 17, 2017 | 18/8/2017 | Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations | An Open Label Study to Investigate the Role of Ivacaftor for the Treatment of Cystic Fibrosis in Combination With Ataluren (PTC124) in Cystic Fibrosis Patients Using Ataluren for Nonsense Mutations An Open Label Study to Investigate the Role of Ivacaftor for the Treatment of Cystic Fibrosis in Com ... | Cystic Fibrosis | Drug: Ivacaftor/Ataluren | University of Alabama at Birmingham | NULL | Completed | 19 Years | N/A | All | 1 | Phase 4 | United States |
| 2 | NCT03256968 (ClinicalTrials.gov) | January 27, 2017 | 18/8/2017 | PTC Study to Evaluate Ataluren in Combination With Ivacaftor | An Open Label N of 1 Study to Evaluate the Study and Efficacy of Long-Term Treatment With Ivacaftor in Combination With Ataluren (PTC124) in Subjects With Nonsense Mutation Cystic Fibrosis An Open Label N of 1 Study to Evaluate the Study and Efficacy of Long-Term Treatment With Ivacaftor ... | Cystic Fibrosis | Drug: Ataluren | University of Alabama at Birmingham | NULL | Completed | 6 Years | N/A | All | 1 | Phase 4 | United States |
| 3 | EUCTR2014-005355-83-GR (EUCTR) | 05/04/2016 | 08/02/2016 | Confirmatory extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;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] Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;Level: PT;Classification code 10011762;Term: ... | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ... | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ... | ||
| 4 | EUCTR2014-005355-83-GB (EUCTR) | 22/03/2016 | 15/02/2016 | Confirmatory extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 extension Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;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] Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;Level: PT;Classification code 10011762;Term: ... | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ... | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ... | ||
| 5 | EUCTR2014-005355-83-BG (EUCTR) | 19/02/2016 | 16/12/2015 | Confirmatory extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;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] Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;Level: PT;Classification code 10011762;Term: ... | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ... | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ... | ||
| 6 | EUCTR2014-005355-83-NL (EUCTR) | 22/01/2016 | 25/11/2015 | Confirmatory extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis | Nonsense Mutation 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] Nonsense Mutation Cystic Fibrosis MedDRA version: 19.0;Level: PT;Classification code 10011762;Term: ... | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ... | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ... | ||
| 7 | EUCTR2014-005355-83-FR (EUCTR) | 08/12/2015 | 04/12/2015 | Confirmatory extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;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] Nonsense Mutation Cystic Fibrosis MedDRA version: 18.1;Level: PT;Classification code 10011762;Term: ... | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ... | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ... | ||
| 8 | EUCTR2014-005355-83-IT (EUCTR) | 16/10/2015 | 26/08/2015 | Confirmatory extension study of ataluren (PTC124) in patients with Cystic Fibrosis | A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis - NA A Phase 3 extension Study of Ataluren(PTC124®) in Patients withNonsense Mutation Cystic Fibrosis - N ... | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.0;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] Nonsense Mutation Cystic Fibrosis MedDRA version: 18.0;Level: PT;Classification code 10011762;Term: ... | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ... | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ... | ||
| 9 | EUCTR2014-005355-83-ES (EUCTR) | 08/10/2015 | 02/09/2015 | Confirmatory extension study of ataluren in patients with Cystic Fibrosis | A Phase 3 extension Study of Ataluren (PTC124®) in Patients withNonsense Mutation Cystic Fibrosis | Nonsense Mutation Cystic Fibrosis MedDRA version: 18.0;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] Nonsense Mutation Cystic Fibrosis MedDRA version: 18.0;Level: PT;Classification code 10011762;Term: ... | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ... | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 200 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ... | ||
| 10 | EUCTR2013-004581-34-BE (EUCTR) | 01/10/2015 | 31/07/2014 | Confirmatory study of ataluren in patients with Cystic Fibrosis | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients with Nonsense Mutation Cystic Fibrosis A Phase 3 Efficacy and Safety Study of Ataluren(PTC124®) in Patients with Nonsense Mutation Cystic F ... | Nonsense Mutation Cystic Fibrosis 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] Nonsense Mutation Cystic Fibrosis MedDRA version: 17.1;Level: PT;Classification code 10011762;Term: ... | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product ... | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 208 | Phase 3 | United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil;Australia;Denmark;Bulgaria;Netherlands;Germany United States;Greece;Spain;Israel;Italy;United Kingdom;France;Canada;Argentina;Poland;Belgium;Brazil ... |
329. 無虹彩症
臨床試験数 : 3 / 薬物数 : 2 - (DrugBank : 2) / 標的遺伝子数 : 0 - 標的パスウェイ数 : 0
Showing 1 to 2 of 2 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 | NCT04117880 (ClinicalTrials.gov) | December 31, 2018 | 19/9/2019 | A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia | Nonsense Mutation Aniridia: An Ataluren (PTC124) Phase 2 Extension Study | Aniridia | Drug: Ataluren | PTC Therapeutics | NULL | Withdrawn | 2 Years | N/A | All | 0 | Phase 2 | NULL |
| 2 | NCT02647359 (ClinicalTrials.gov) | January 31, 2016 | 29/12/2015 | Study of Ataluren in Participants With Nonsense Mutation Aniridia | STAR: A Phase 2, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study of the Safety and Efficacy of Ataluren (PTC124) for the Treatment of Nonsense Mutation Aniridia STAR: A Phase 2, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study of the Safety and ... | Aniridia | Drug: Ataluren;Drug: Placebo | PTC Therapeutics | NULL | Completed | 2 Years | N/A | All | 39 | Phase 2 | United States;Canada |