Clinical Trials for Mitochondrial Disease

MitoCanada is committed to curating an active list of clinical trials important to patients and mitochondrial disease research.

NuPower Study | Elampretide in PMD with nDNA Mutations, USA

Phase 3 randomized, double-blind trial evaluating the efficacy and safety of daily subcutaneous injections of Elamipretide in PMD w/nDNA mutations

Enrolment: Recruiting now

Study Site: Multi-USA site; Canadian recruits welcome

Details: 18-70 yrs, genetic confirmation of nDNA mutation

Sponsor: Stealth BioTherapeutics Inc

LC-FAOD In-Clinic Disease Monitoring Study | Global

Global observational long-term study collecting information from adult and pediatric patients with LC-FAOD

Enrolment: Recruiting now

Study Site: Multi-USA site; Canada: SickKids Hospital, Toronto

Details: 300 patients, child, adult and older adults with a confirmed diagnosis of any LC-FAOD sub-type.

Sponsor: Ultragenyx