246. Methylmalonic acidemia Clinical trials / Disease details
Clinical trials : 19 / Drugs : 26 - (DrugBank : 3) / Drug target genes : 17 - Drug target pathways : 23
| 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 Acidemia - ND | Nonsense Mutation Methylmalonic Acidemia MedDRA version: 12.1;Level: LLT;Classification code 10064180;Term: Blood methylmalonic acid increased | 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 | 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 | 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] | 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 | 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 | 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 Acidemia | Nonsense Mutation Methylmalonic Acidemia MedDRA version: 12.1;Level: LLT;Classification code 10064180;Term: Blood methylmalonic acid increased | 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 | 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 | 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 Acidemia | Nonsense Mutation Methylmalonic Acidemia MedDRA version: 12.1;Level: LLT;Classification code 10064180;Term: Blood methylmalonic acid increased | 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 | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 18 | Phase 2 | United Kingdom;France;Italy |