324. メチルグルタコン酸尿症 Methylglutaconic aciduria Clinical trials / Disease details


臨床試験数 : 4 薬物数 : 4 - (DrugBank : 3) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 10

  
2 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04689360
(ClinicalTrials.gov)
December 23, 202023/12/2020An Intermediate Size Expanded Access Protocol of ElamipretideAn Intermediate Size Expanded Access Protocol of Elamipretide for Subcutaneous Injection in Patients With Genetically Confirmed Rare Diseases With Known Mitochondrial DysfunctionMitochondrial Diseases;Barth SyndromeDrug: elamipretideStealth BioTherapeutics Inc.NULLAvailable1 Year80 YearsAllNULL
2NCT03098797
(ClinicalTrials.gov)
May 1, 201721/3/2017A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth SyndromeA Phase 2 Randomized, Double-Blind, Placebo-Controlled Crossover Trial to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Injections of Elamipretide (MTP-131) in Subjects With Genetically Confirmed Barth Syndrome Followed by an Open-Label Treatment ExtensionBarth SyndromeDrug: ElamipretideStealth BioTherapeutics Inc.NULLCompleted12 YearsN/AMale12Phase 2/Phase 3United States