324. Methylglutaconic aciduria Clinical trials / Disease details
Clinical trials : 4 / Drugs : 4 - (DrugBank : 3) / Drug target gene : 1 - Drug target pathways : 10
| 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 |