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Trial record 11 of 178 for:    "Mitochondrial Diseases" OR "mitochondrial neurogastrointestinal encephalopathy disease"

An Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300) (RePOWER)

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ClinicalTrials.gov Identifier: NCT03048617
Recruitment Status : Active, not recruiting
First Posted : February 9, 2017
Last Update Posted : March 27, 2018
Sponsor:
Information provided by (Responsible Party):
Stealth BioTherapeutics Inc.

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.

Condition or disease
Primary Mitochondrial Disease

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.

Study Type : Observational
Estimated Enrollment : 300 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: A Prospective Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)
Actual Study Start Date : February 13, 2017
Estimated Primary Completion Date : May 2018
Estimated Study Completion Date : May 2018





Primary Outcome Measures :
  1. Assess the relationship of genotype to phenotype in patients with Primary Mitochondrial Disease [ Time Frame: 1 year ]
  2. Compare local and regional differences in standard of care and management of patients with Primary Mitochondrial Disease [ Time Frame: 1 year ]

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


Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 80 Years   (Child, Adult, Older 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

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.

Responsible Party: Stealth BioTherapeutics Inc.
ClinicalTrials.gov Identifier: NCT03048617     History of Changes
Other Study ID Numbers: SPIMM-300
First Posted: February 9, 2017    Key Record Dates
Last Update Posted: March 27, 2018
Last Verified: March 2018

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