Primary Mitochondrial Disease Clinical Trial
— RePOWEROfficial title:
A Prospective Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)
NCT number | NCT03048617 |
Other study ID # | SPIMM-300 |
Secondary ID | |
Status | Completed |
Phase | |
First received | |
Last updated | |
Start date | February 13, 2017 |
Est. completion date | March 7, 2019 |
Verified date | April 2019 |
Source | Stealth BioTherapeutics Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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.
Status | Completed |
Enrollment | 215 |
Est. completion date | March 7, 2019 |
Est. primary completion date | August 2, 2018 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 16 Years to 80 Years |
Eligibility |
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 |
Country | Name | City | State |
---|---|---|---|
Australia | Calvary Health Care Bethlehem | Caulfield South | Victoria |
Australia | Royal North Shore Hospital | St Leonards | |
Canada | University of Calgary Metabolic Disease Clinica | Calgary | Alberta |
Canada | McMaster University Medical Center | Hamilton | Ontario |
Canada | Adult Metabolic Diseases Clinic | Vancouver | |
Denmark | University of Copenhagen | Copenhagen | |
Germany | University Hospital of Bonn | Bonn | |
Germany | Klinikim der Universitat Muchen, Friedrich-Baur Institute | Munich | Bavaria |
Hungary | Institute of Genomic Medicine and Rare Disorders | Budapest | |
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 | Istituto Nazionale Neurologico Carlo Besta | Milano | |
Italy | Dipartimento Ambientale di Neuroscienze | Roma | |
Italy | Istituto di Neurologia, Fondazione Policlinico Universitario A. Gemelli | Roma | |
Italy | Ospedale Pediatrico Bambin Gesu | Roma | |
Spain | Hospital Universitario 12 de Octubre | Madrid | |
Spain | Neurology Department Hospital Universitari i Politecnic La Fe | Valencia | |
United Kingdom | MRC Centre for Neuromuscular Diseases | London | |
United Kingdom | Royal Victoria Infirmary | Newcastle upon Tyne | |
United States | Akron Children's Hospital | Akron | Ohio |
United States | Children's Hospital Colorado | Aurora | Colorado |
United States | Massachusetts General Hospital | Boston | Massachusetts |
United States | Cleveland Clinic | Cleveland | Ohio |
United States | Baylor College of Medicine | Houston | Texas |
United States | The University of Texas Health Science Center | Houston | Texas |
United States | University of California San Diego | La Jolla | California |
United States | Columbia University Medical Center | New York | New York |
United States | Stanford Universtity | Palo Alto | California |
United States | The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
United States | Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania |
United States | University of Utah Hospital | Salt Lake City | Utah |
United States | Seattle Children's Hospital | Seattle | Washington |
Lead Sponsor | Collaborator |
---|---|
Stealth BioTherapeutics Inc. |
United States, Australia, Canada, Denmark, Germany, Hungary, Italy, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Assess the relationship of genotype to phenotype in patients with Primary Mitochondrial Disease | 1 year | ||
Primary | Compare local and regional differences in standard of care and management of patients with Primary Mitochondrial Disease | 1 year | ||
Secondary | Compare local and regional differences in genetic testing methodologies for Primary Mitochondrial Disease | 1 year |
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