324. メチルグルタコン酸尿症 ［臨床試験数：2，薬物数：2（DrugBank：2），標的遺伝子数：0，標的パスウェイ数：0］
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.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03098797 | May 1, 2017 | 25 February 2019 | 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. | Not recruiting | 12 Years | N/A | Male | 12 | Phase 2/Phase 3 | United States | |
| 2 | NCT01461304 | October 2011 | 9 September 2019 | Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism | Dietary Therapy for Inherited Disorders of Energy Metabolism | Very 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 Syndrome | Drug: triheptanoin | Jerry Vockley, MD, PhD | Ultragenyx Pharmaceutical Inc | Not recruiting | 1 Month | N/A | All | Phase 2 | United States |