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.	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	NCT04689360 (ClinicalTrials.gov)	December 23, 2020	23/12/2020	An Intermediate Size Expanded Access Protocol of Elamipretide	An Intermediate Size Expanded Access Protocol of Elamipretide for Subcutaneous Injection in Patients With Genetically Confirmed Rare Diseases With Known Mitochondrial Dysfunction	Mitochondrial Diseases;Barth Syndrome	Drug: elamipretide	Stealth BioTherapeutics Inc.	NULL	Available	1 Year	80 Years	All			NULL
2	NCT03098797 (ClinicalTrials.gov)	May 1, 2017	21/3/2017	A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome	A 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 Extension	Barth Syndrome	Drug: Elamipretide	Stealth BioTherapeutics Inc.	NULL	Completed	12 Years	N/A	Male	12	Phase 2/Phase 3	United States