Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

102

Participants

Timeline

Start Date

April 29, 2022

Primary Completion Date

September 30, 2024

Study Completion Date

October 31, 2024

Conditions
Mitochondrial MyopathiesMitochondrial PathologyMitochondrial DNA MutationMitochondrial DiseasesMitochondrial DNA DeletionMitochondrial DNA DepletionMitochondrial Metabolism DefectMitochondrial Complex I Deficiency
Interventions
DRUG

Elamipretide

60 mg of elamipretide administered as once daily 0.75 mL subcutaneous injections for 48 weeks

DRUG

Placebo

Placebo administered as once daily 0.75 mL subcutaneous injections for 48 weeks

Trial Locations (32)

1023

University of Auckland - Auckland City Hospital, Neurology Department, Auckland

1083

Semmelweis Egyetem Genomikai Medicina es Ritka Betegsegek, Budapest

2065

Royal North Shore Hospital Neurology, Sydney

3162

Calvary Health Care Bethlehem, Parkdale

5021

Helse Bergen HF, Bergen

6525

Radboud University Medical Center, Nijmegen

7624

University of Pécs, Department of Neurology Klinikai Kozpont - neurologiai Klinika, Pécs

8025

Hospital de la Sta Creu i Sant Pau, Barcelona

8036

Hospital Clinic de Barcelona, Barcelona

10032

Columbia University Medical Center College of Physician and Surgeon, New York

15224

University of Pittsburgh School of Medicine Children's Hospital of Pittsburgh of UPMC Department of Genetics, Pittsburgh

19104

The Children's Hospital of Philadelphia, Philadelphia

20133

Istituto Nazionale Neurologico Carlo Besta, Milan

25064

University of Brescia, NeMO Clinical Center for Neuromuscular Diseases, Gussago

28041

Hospital Universitario 12 de Octubre, Madrid

30329

Rare Disease Research, LLC, Atlanta

44308

Akron Children's Hospital, Akron

46026

Hospital la Fe de Valencia, Valencia

56126

Azienda Ospedaliero Universitario Pisana, Dipartimento Ambientale di Neuroscienze, Pisa

63110

Washington University School of Medicine, St Louis

77030

UT Health,Center for the Treatment of Pediatric Neurodegenerative Disease, Houston

80336

Department of Neurology, University Clinics Munich, Munich

92093

University of California, San Diego, San Diego

98125

Azienda Ospedaliero Universitaria Policlinico G. Martino, Messina

02114

Massachusetts General Hospital, Boston

Unknown

Universitaetsklinikum Carl Gustav Carus Dresden Neurologie, Dresden

Univ of Tubingen, Hertie Institute for Clinical Brain Research, Tübingen

IRCCS Institute of Neorological Sciences of Bologna Bellaria Hospital, Bologna

00168

Istituto di Neurologia, Fondazione Policlinico Universitario A. Gemelli, Università Cattolica del Sacro Cuore, Rome

CB2 0QQ

University of Cambridge, Department of Clinical Neurosciences, Cambridge

WC1N 3BG

Queen Square Centre for Neuromuscular Diseases The National Hospital for Neurology and Neurosurgery, London

NE77DN

Newcastle upon Tyne Hospitals Freeman Hospital, Newcastle

All Listed Sponsors
lead

Stealth BioTherapeutics Inc.

INDUSTRY

NCT05162768 - Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) | Biotech Hunter | Biotech Hunter