Clinical Trial: An Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational
Official Title: A Prospective Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)
Brief 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.
Detailed Summary: 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.
Sponsor: Stealth BioTherapeutics Inc.
Current Primary Outcome:
- Assess the relationship of genotype to phenotype in patients with Primary Mitochondrial Disease [ Time Frame: 1 year ]
- Compare local and regional differences in standard of care and management of patients with Primary Mitochondrial Disease [ Time Frame: 1 year ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Compare local and regional differences in genetic testing methodologies for Primary Mitochondrial Disease [ Time Frame: 1 year ]
Original Secondary Outcome: Same as current
Information By: Stealth BioTherapeutics Inc.
Dates:
Date Received: February 3, 2017
Date Started: February 13, 2017
Date Completion: January 2018
Last Updated: April 14, 2017
Last Verified: April 2017
