324. メチルグルタコン酸尿症
[臨床試験数:3,薬物数:4(DrugBank:3),標的遺伝子数:1,標的パスウェイ数:8

Searched query = "Methylglutaconic aciduria", "3-methylglutaconyl-CoA hydratase deficiency", "3-MGA", "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. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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PhaseCountries
1EUCTR2015-001382-10-GB
(EUCTR)
21/01/201924/08/2015A study comparing the drug 'bezafibrate' against a placebo in the treatment of Barth SyndromeTreatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN): A randomised placebo controlled pilot trial conducted by the nationally commissioned Barth Syndrome Service - CARDIOMAN Barth Syndrome is a rare, life threatening, genetic disease which affects young males. It is caused by abnormal fats (lipids) in the powerhouses of cells (mitochondria) and those who suffer with it often develop heart failure, heart rhythm abnormalities, bacterial infections, poor growth or feeding, weak muscles, developmental delay, severe exercise intolerance, lethargy and fatigue.
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
University Hospitals Bristol NHS Foundation TrustNULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
15Phase 2United Kingdom
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.NULLActive, not recruiting12 YearsN/AMale12Phase 2;Phase 3United States
3NCT01461304
(ClinicalTrials.gov)
November 30, 201125/10/2011Compassionate 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 IncAvailable1 MonthN/AAllUnited States