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Clinical Trial Details — Status: Completed

Administrative data

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

Study information

Verified date April 2019
Source Stealth BioTherapeutics Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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.


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.


Recruitment information / eligibility

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

Study Design


Related Conditions & MeSH terms


Locations

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

Sponsors (1)

Lead Sponsor Collaborator
Stealth BioTherapeutics Inc.

Countries where clinical trial is conducted

United States,  Australia,  Canada,  Denmark,  Germany,  Hungary,  Italy,  Spain,  United Kingdom, 

Outcome

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
See also
  Status Clinical Trial Phase
Recruiting NCT05972954 - OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION) Phase 2
Recruiting NCT05250375 - Mitochondrial Myopathy Rating Scale
Terminated NCT02976038 - Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM) Phase 2
Active, not recruiting NCT05650229 - Efficacy of KL1333 in Adult Patients With Primary Mitochondrial Disease Phase 2
Completed NCT02805790 - Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study Phase 2