246. メチルマロン酸血症 [臨床試験数:14,薬物数:22(DrugBank:2),標的遺伝子数:1,標的パスウェイ数:6

Searched query = "Methylmalonic acidemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
5 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2009-016654-41-IT11/11/201005/07/2010A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia - NDA Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia - ND Nonsense Mutation Methylmalonic Acidemia
MedDRA version: 12.1Level: LLTClassification code 10064180Term: 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.NULLNot RecruitingFemale: yes
Male: yes
18Phase 2France;United Kingdom;Italy
2EUCTR2009-016654-41-BE04/11/201022/12/2010Study of ataluren in patients with Methylmalonic AcidemiaA Phase 2 Study of Ataluren (PTC124®) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia Nonsense Mutation Methylmalonic Acidemia (MMA)
MedDRA version: 14.0Level: LLTClassification code 10064180Term: Blood methylmalonic acid increasedSystem 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.NULLNot RecruitingFemale: yes
Male: yes
18Phase 2France;Belgium;Italy;United Kingdom;Switzerland
3NCT01141075July 19, 20107/6/2010Ataluren for Nonsense Mutation Methylmalonic AcidemiaA Phase 2 Study of Ataluren (PTC124®) as an Oral Treatment for Nonsense Mutation Methylmalonic AcidemiaAmino Acid Metabolism, Inborn ErrorsDrug: AtalurenPTC TherapeuticsGenzyme, a Sanofi CompanyTerminated2 YearsN/AAll11Phase 2Belgium;France;Germany;Italy;Switzerland;United Kingdom
4EUCTR2009-016654-41-GB01/06/201017/03/2010A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic AcidemiaA 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
PTC Therapeutics, Inc.NULLNot Recruiting Female: yes
Male: yes
18 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): yes Therapeut1c conf1rmatory - (Phase 3): no Therapeut1c use (Phase 4): noFrance;Belgium;Italy;United Kingdom
5EUCTR2009-016654-41-FR02/03/201004/01/2010A Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic AcidemiaA Phase 2 Study of Ataluren (PTC124) as an Oral Treatment for Nonsense Mutation Methylmalonic Acidemia Nonsense Mutation Methylmalonic Acidemia
MedDRA version: 12.1Level: LLTClassification code 10064180Term: 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
18Phase 2United Kingdom;France;Italy