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 324. Methylglutaconic aciduria
 [ 2 clinical trials,    2 drugs(DrugBank: 2 drugs),    0 target gene / 0 target pathway

Searched query = "Methylglutaconic aciduria", "3 methylglutaconyl CoA hydratase deficiency", "Barth syndrome", "Costeff syndrome", "Mitochondrial respiratory chain disorder", "Dilated cardiomyopathy with ataxia syndrome", "DCMA syndrome"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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agemax
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PhaseCountries
1NCT03098797May 1, 201725 February 2019A 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.Not recruiting12 YearsN/AMale12Phase 2/Phase 3United States
2NCT01461304October 20119 September 2019Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy MetabolismDietary Therapy for Inherited Disorders of Energy MetabolismVery Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency;Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2);Mitochondrial Trifunctional Protein Deficiency;Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency;Glycogen Storage Disorders;Pyruvate Carboxylase Deficiency Disease;ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of;Barth SyndromeDrug: triheptanoinJerry Vockley, MD, PhDUltragenyx Pharmaceutical IncNot recruiting1 MonthN/AAllPhase 2United States