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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 : September 6, 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
Actual Primary Completion Date : August 2, 2018
Estimated Study Completion Date : December 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


  Hide Study Locations
Locations
United States, California
University of California San Diego
La Jolla, California, United States, 92093
Stanford Universtity
Palo Alto, California, United States, 94304
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
United States, New York
Columbia University Medical Center
New York, New York, United States, 10032
United States, Ohio
Akron Children's Hospital
Akron, Ohio, United States, 44308
Cleveland Clinic
Cleveland, Ohio, United States, 44195
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104`
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
The University of Texas Health Science Center
Houston, Texas, United States, 77030
United States, Utah
University of Utah Hospital
Salt Lake City, Utah, United States, 84132
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Australia, Victoria
Calvary Health Care Bethlehem
Caulfield South, Victoria, Australia
Australia
Royal North Shore Hospital
St Leonards, Australia
Canada, Alberta
University of Calgary Metabolic Disease Clinica
Calgary, Alberta, Canada
Canada, Ontario
McMaster University Medical Center
Hamilton, Ontario, Canada
Canada
Adult Metabolic Diseases Clinic
Vancouver, Canada
Denmark
University of Copenhagen
Copenhagen, Denmark
Germany
Klinikim der Universitat Muchen, Friedrich-Baur Institute
Munich, Bavaria, Germany, 80336
University Hospital of Bonn
Bonn, Germany, 53105
Hungary
Institute of Genomic Medicine and Rare Disorders
Budapest, Hungary
Italy
IRCCS Institute of Neurological Sciences of Bolgna, Bellaria Hospital
Bologna, Italy
ASST Spedali Civili di Brescia
Brescia, Italy
Azienda Ospedaliero Universitaria Policlinico G. Martino
Messina, Italy, 98125
Istituto Nazionale Neurologico Carlo Besta
Milano, Italy
Istituto di Neurologia, Fondazione Policlinico Universitario A. Gemelli
Roma, Italy, 00168
Dipartimento Ambientale di Neuroscienze
Roma, Italy, 56126
Ospedale Pediatrico Bambin Gesu
Roma, Italy
Spain
Hospital Universitario 12 de Octubre
Madrid, Spain
Neurology Department Hospital Universitari i Politecnic La Fe
Valencia, Spain, 46026
United Kingdom
MRC Centre for Neuromuscular Diseases
London, United Kingdom
Royal Victoria Infirmary
Newcastle upon Tyne, United Kingdom
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: September 6, 2018
Last Verified: September 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