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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 : Recruiting
First Posted : February 9, 2017
Last Update Posted : October 25, 2017
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 : January 2018
Estimated Study Completion Date : January 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 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

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
United States, California
University of California San Diego Recruiting
La Jolla, California, United States, 92093
Contact: Jessica Reit    619-543-2816    jreit@ucsd.edu   
Stanford Universtity Recruiting
Palo Alto, California, United States, 94304
Contact: Thu Quan    650-736-8166    tquan@stanford.edu   
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Michelle Hite    720-777-4690    michelle.hite@childrenscolorado.org   
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Erica Kelly    617-724-7726    elkelly@mgh.harvard.edu   
United States, New York
Columbia University Medical Center Recruiting
New York, New York, United States, 10032
Contact: Kristin Engelstad    212-305-6834    ke4@cumc.columbia.edu   
United States, Ohio
Akron Children's Hospital Recruiting
Akron, Ohio, United States, 44308
Contact: Hilary Tonni, BSN, MSE, RN, CCRC    330-543-4743    htonni@chmca.org   
Cleveland Clinic Recruiting
Cleveland, Ohio, United States, 44195
Contact: Irys B Caristo    216-444-0173    caristi@ccf.org   
United States, Pennsylvania
The Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104`
Contact: Sara Nguyen    267-426-7165    nguyens2@email.chop.edu   
Children's Hospital of Pittsburgh of UPMC Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Sandra Braden    412-692-5969    paronishsl@upmc.edu   
United States, Texas
Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
Contact: Catherine Loffredo       catherine.loffredo@bcm.edu   
The University of Texas Health Science Center Recruiting
Houston, Texas, United States, 77030
Contact: Noemy Contress    713-500-5372    Noemy.Y.Contress@uth.tmc.edu   
United States, Utah
University of Utah Hospital Recruiting
Salt Lake City, Utah, United States, 84132
Contact: Carrie Bailey       carrie.bailey@hsc.utah.edu   
United States, Washington
Seattle Children's Hospital Not yet recruiting
Seattle, Washington, United States, 98105
Contact: Laurie Guidry       laurie.guidry@seattlechildrens.org   
Australia, Victoria
Calvary Health Care Bethlehem Not yet recruiting
Caulfield South, Victoria, Australia
Contact: Susan Caldwell    0395953288    sue.caldwell@calvarycare.org.au   
Royal North Shore Hospital Not yet recruiting
St Leonards, Australia
Contact: Fabienne Edemahildebrand    0294631827    fabienne.edemahildebrand@health.nsw.gov.au   
Canada, Alberta
University of Calgary Metabolic Disease Clinica Not yet recruiting
Calgary, Alberta, Canada
Contact: Desmond Koo       djkoo@ucalgary.ca   
Canada, Ontario
McMaster University Medical Center Not yet recruiting
Hamilton, Ontario, Canada
Contact: Erin Hatcher    9055212100 ext 76929    hatchere@hhsc.ca   
Adult Metabolic Diseases Clinic Recruiting
Vancouver, Canada
Contact: Anna Koochin       anna.koochin@vch.ca   
University of Copenhagen Recruiting
Copenhagen, Denmark
Contact: Maj-Britt Tanderup       maj-britt.tanderup.joergensen@regionh.dk   
Klinikim der Universitat Muchen, Friedrich-Baur Institute Not yet recruiting
Munich, Bavaria, Germany, 80336
Contact: Boriana Buchner    4989440057443    boriana.buchner@med.uni-muenchen.de   
University Hospital of Bonn Recruiting
Bonn, Germany, 53105
Contact: Christiane Landwehr       christiane.landwehr@ukb.uni-bonn.de   
Institute of Genomic Medicine and Rare Disorders Recruiting
Budapest, Hungary
Contact: Gyorgyi Bathori    36206632514    molneur@gmail.com   
IRCCS Institute of Neurological Sciences of Bolgna, Bellaria Hospital Not yet recruiting
Bologna, Italy
Contact: Maria Lucia Valentino    +390514966144    marialucia.valentino@unibo.it   
ASST Spedali Civili di Brescia Not yet recruiting
Brescia, Italy
Contact: Massimiliano Filosto    390303995632    massimilliano.filosto@unibs.it   
Azienda Ospedaliero Universitaria Policlinico G. Martino Recruiting
Messina, Italy, 98125
Contact: Antonio Toscano, MD    390902217178    atoscano@unime.it   
Istituto Nazionale Neurologico Carlo Besta Not yet recruiting
Milano, Italy
Contact: Lorenzo Peverelli, MD       lorenzo.peverelli@istituto-besta.it   
Istituto di Neurologia, Fondazione Policlinico Universitario A. Gemelli Recruiting
Roma, Italy, 00168
Contact: Guido Primiano, MD       guido.primiano@gmail.com   
Dipartimento Ambientale di Neuroscienze Recruiting
Roma, Italy, 56126
Contact: Vincenzo Montano       v.montano@gmail.com   
Ospedale Pediatrico Bambin Gesu Not yet recruiting
Roma, Italy
Contact: Giorgia Copponi       giorgia.copponi@opbg.net   
Hospital Universitario 12 de Octubre Not yet recruiting
Madrid, Spain
Contact: Montserrat Morales Conejo    34913908887    montserrat.morales@salud.madrid.org   
Neurology Department Hospital Universitari i Politecnic La Fe Not yet recruiting
Valencia, Spain, 46026
Contact: Marti Pimar    34677756564    marti_mpimar@gva.es   
United Kingdom
MRC Centre for Neuromuscular Diseases Recruiting
London, United Kingdom
Contact: Daleen Lopez       daleen.lopez-begg@ucl.ac.uk   
Royal Victoria Infirmary Recruiting
Newcastle upon Tyne, United Kingdom
Contact: Julia Madison    01912085982    julia.madison@ncl.ac.uk   
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: October 25, 2017
Last Verified: October 2017

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

Additional relevant MeSH terms:
Mitochondrial Diseases
Metabolic Diseases