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