Elamipretide ( DrugBank: Elamipretide )


2 diseases
IDDisease name (Link within this page)Number of trials
21Mitochondrial disease5
324Methylglutaconic aciduria2

21. Mitochondrial disease


Clinical trials : 33 Drugs : 42 - (DrugBank : 32) / Drug target genes : 47 - Drug target pathways : 67
No.TrialIDDate_
enrollment
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registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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agemin
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agemax
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PhaseCountries
1EUCTR2021-003907-16-HU
(EUCTR)
02/03/202204/01/2022Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD)A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD) Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD)
MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: HLGT;Classification code 10052635;Term: Cytoplasmic disorders congenital;Level: HLT;Classification code 10052637;Term: Genetic mitochondrial abnormalities NEC;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Elamipretide
Product Code: MTP-131
INN or Proposed INN: ELAMIPRETIDE
Other descriptive name: N/A
Stealth BioTherapeutics Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
130Phase 3United States;Hungary;Finland;Spain;Denmark;Australia;Norway;Netherlands;Germany;United Kingdom;Italy
2NCT05162768
(ClinicalTrials.gov)
March 20228/12/2021Study to Evaluate the Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease Resulting From Nuclear DNA Mutations (nPMD)A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Disease Resulting From Pathogenic Nuclear DNA Mutations (nPMD) - NuPower StudyMitochondrial Myopathies;Mitochondrial Pathology;Mitochondrial DNA Mutation;Mitochondrial Diseases;Mitochondrial DNA Deletion;Mitochondrial DNA Depletion;Mitochondrial Metabolism Defect;Mitochondrial Complex I DeficiencyDrug: Elamipretide;Drug: PlaceboStealth BioTherapeutics Inc.NULLRecruiting18 Years70 YearsAll130Phase 3United States
3NCT02976038
(ClinicalTrials.gov)
December 201618/11/2016Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)A Multicenter, Open-Label Phase 2 Extension Trial to Characterize the Long-term Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)Primary Mitochondrial DiseaseDrug: elamipretideStealth BioTherapeutics Inc.NULLTerminated16 YearsN/AAll36Phase 2United States
4NCT02805790
(ClinicalTrials.gov)
August 22, 201613/6/2016Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER StudyA Phase 2 Randomized, Double-Blind, Placebo-Controlled Crossover Study to Evaluate Safety, Tolerability, and Efficacy of Subcutaneous Injections of MTP-131 in Subjects With Mitochondrial Myopathy Previously Treated in the SPIMM-201 StudyPrimary Mitochondrial DiseaseDrug: Elamipretide;Drug: PlaceboStealth BioTherapeutics Inc.NULLCompleted16 YearsN/AAll30Phase 2United States
5NCT02367014
(ClinicalTrials.gov)
February 20159/2/2015Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial MyopathyPhase 1/2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Clinical Study for the Safety, Tolerability, and Efficacy of IV MTP-131 for Mitochondrial Myopathy in Genetically Confirmed Mitochondrial DiseaseMitochondrial MyopathyDrug: elamipretide (low dose);Drug: elamipretide (intermediate dose);Drug: elamipretide (high dose);Drug: PlaceboStealth BioTherapeutics Inc.NULLCompleted16 Years65 YearsAll36Phase 1/Phase 2United States

324. Methylglutaconic aciduria


Clinical trials : 4 Drugs : 4 - (DrugBank : 3) / Drug target gene : 1 - Drug target pathways : 10
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04689360
(ClinicalTrials.gov)
December 23, 202023/12/2020An Intermediate Size Expanded Access Protocol of ElamipretideAn Intermediate Size Expanded Access Protocol of Elamipretide for Subcutaneous Injection in Patients With Genetically Confirmed Rare Diseases With Known Mitochondrial DysfunctionMitochondrial Diseases;Barth SyndromeDrug: elamipretideStealth BioTherapeutics Inc.NULLAvailable1 Year80 YearsAllNULL
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.NULLCompleted12 YearsN/AMale12Phase 2/Phase 3United States