Summary

for people ages 16-80 (full criteria)
at La Jolla, California and other locations
study started
estimated completion:

Description

Summary

This is an observational study of patients with Primary Mitochondrial Disease with either signs or symptoms suggestive of myopathy. The Investigator will identify potential patients through existing medical records and one on-site visit.

Official Title

A Prospective Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)

Details

An observational study of patients with presumed Primary Mitochondrial Disease designed to better characterize and correlate symptoms and signs of myopathy and genetic test results and the use of commonly prescribed treatments. The study will help define and identify a subject population for a future trial of an investigational product to treat primary mitochondrial disease associated with signs and symptoms of myopathy.

Keywords

Primary Mitochondrial Disease Stealth Myopathy exercise intolerance muscle pain Mitochondrial Myopathy Primary Mitochondrial Myopathy Mitochondrial Diseases

Eligibility

You can join if…

Open to people ages 16-80

  • Willing and able to provide a signed informed consent form (ICF) prior to participation in any-trial related procedures
  • Patient has clinical presentation of PMD with either signs or symptoms suggestive of myopathy
  • Patient is ambulatory and able to attempt 6MWT

You CAN'T join if...

  • Patient has symptoms of PMD due to secondary mitochondrial dysfunction
  • Patient has had prior exposure to elamipretide
  • Patient does not have the cognitive capacity to understand and complete all study assessments
  • Patient has a medical history of severe renal impairment
  • History of active alcoholism or drug addiction during the year before enrollment

Locations

  • University of California San Diego
    La Jolla California 92093 United States
  • Stanford Universtity
    Palo Alto California 94304 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Stealth BioTherapeutics Inc.
ID
NCT03048617
Study Type
Observational
Last Updated