An Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)
RePOWER
A Prospective Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)
1 other identifier
observational
215
9 countries
33
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Feb 2017
Typical duration for all trials
33 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 3, 2017
CompletedFirst Posted
Study publicly available on registry
February 9, 2017
CompletedStudy Start
First participant enrolled
February 13, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 2, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
March 7, 2019
CompletedApril 11, 2019
April 1, 2019
1.5 years
February 3, 2017
April 9, 2019
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Assess the relationship of genotype to phenotype in patients with Primary Mitochondrial Disease
1 year
Compare local and regional differences in standard of care and management of patients with Primary Mitochondrial Disease
1 year
Secondary Outcomes (1)
Compare local and regional differences in genetic testing methodologies for Primary Mitochondrial Disease
1 year
Eligibility Criteria
Patients with clinical presentation of PMD with either signs or symptoms suggestive of myopathy
You may qualify if:
- 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 may not qualify 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (33)
University of California San Diego
La Jolla, California, 92093, United States
Stanford Universtity
Palo Alto, California, 94304, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Columbia University Medical Center
New York, New York, 10032, United States
Akron Children's Hospital
Akron, Ohio, 44308, United States
Cleveland Clinic
Cleveland, Ohio, 44195, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104', United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
The University of Texas Health Science Center
Houston, Texas, 77030, United States
University of Utah Hospital
Salt Lake City, Utah, 84132, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Calvary Health Care Bethlehem
Caulfield South, Victoria, Australia
Royal North Shore Hospital
St Leonards, Australia
University of Calgary Metabolic Disease Clinica
Calgary, Alberta, Canada
McMaster University Medical Center
Hamilton, Ontario, Canada
Adult Metabolic Diseases Clinic
Vancouver, Canada
University of Copenhagen
Copenhagen, Denmark
Klinikim der Universitat Muchen, Friedrich-Baur Institute
Munich, Bavaria, 80336, Germany
University Hospital of Bonn
Bonn, 53105, Germany
Institute of Genomic Medicine and Rare Disorders
Budapest, Hungary
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, 98125, Italy
Istituto Nazionale Neurologico Carlo Besta
Milan, Italy
Istituto di Neurologia, Fondazione Policlinico Universitario A. Gemelli
Roma, 00168, Italy
Dipartimento Ambientale di Neuroscienze
Roma, 56126, Italy
Ospedale Pediatrico Bambin Gesu
Roma, Italy
Hospital Universitario 12 de Octubre
Madrid, Spain
Neurology Department Hospital Universitari i Politecnic La Fe
Valencia, 46026, Spain
MRC Centre for Neuromuscular Diseases
London, United Kingdom
Royal Victoria Infirmary
Newcastle upon Tyne, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- observational
- Observational Model
- CASE ONLY
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 3, 2017
First Posted
February 9, 2017
Study Start
February 13, 2017
Primary Completion
August 2, 2018
Study Completion
March 7, 2019
Last Updated
April 11, 2019
Record last verified: 2019-04