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Trial record 1 of 1 for:    Repower | Recruiting, Not yet recruiting Studies
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An Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300) (RePOWER)

This study is currently recruiting participants.
Verified October 2017 by Stealth BioTherapeutics Inc.
Sponsor:
ClinicalTrials.gov Identifier:
NCT03048617
First Posted: February 9, 2017
Last Update Posted: October 25, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Stealth BioTherapeutics Inc.
  Purpose
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.

Condition
Primary Mitochondrial Disease

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: A Prospective Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)

Resource links provided by NLM:


Further study details as provided by Stealth BioTherapeutics Inc.:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • Compare local and regional differences in genetic testing methodologies for Primary Mitochondrial Disease [ Time Frame: 1 year ]

Estimated Enrollment: 300
Actual Study Start Date: February 13, 2017
Estimated Study Completion Date: January 2018
Estimated Primary Completion Date: January 2018 (Final data collection date for primary outcome measure)
Detailed Description:
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.
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 65 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Patients with clinical presentation of PMD with either signs or symptoms suggestive of myopathy
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03048617


  Show 33 Study Locations
Sponsors and Collaborators
Stealth BioTherapeutics Inc.
  More Information

Responsible Party: Stealth BioTherapeutics Inc.
ClinicalTrials.gov Identifier: NCT03048617     History of Changes
Other Study ID Numbers: SPIMM-300
First Submitted: February 3, 2017
First Posted: February 9, 2017
Last Update Posted: October 25, 2017
Last Verified: October 2017

Keywords provided by Stealth BioTherapeutics Inc.:
Primary Mitochondrial Disease
Stealth
Myopathy
exercise intolerance
muscle pain
Mitochondrial Myopathy
Primary Mitochondrial Myopathy

Additional relevant MeSH terms:
Mitochondrial Diseases
Metabolic Diseases