An Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)

NCT ID: NCT03048617

Last Updated: 2019-04-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

215 participants

Study Classification

OBSERVATIONAL

Study Start Date

2017-02-13

Study Completion Date

2019-03-07

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Primary Mitochondrial Disease

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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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
Minimum Eligible Age

16 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Stealth BioTherapeutics Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of California San Diego

La Jolla, California, United States

Site Status

Stanford Universtity

Palo Alto, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

Akron Children's Hospital

Akron, Ohio, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

The University of Texas Health Science Center

Houston, Texas, United States

Site Status

University of Utah Hospital

Salt Lake City, Utah, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

Calvary Health Care Bethlehem

Caulfield South, Victoria, Australia

Site Status

Royal North Shore Hospital

St Leonards, , Australia

Site Status

University of Calgary Metabolic Disease Clinica

Calgary, Alberta, Canada

Site Status

McMaster University Medical Center

Hamilton, Ontario, Canada

Site Status

Adult Metabolic Diseases Clinic

Vancouver, , Canada

Site Status

University of Copenhagen

Copenhagen, , Denmark

Site Status

Klinikim der Universitat Muchen, Friedrich-Baur Institute

Munich, Bavaria, Germany

Site Status

University Hospital of Bonn

Bonn, , Germany

Site Status

Institute of Genomic Medicine and Rare Disorders

Budapest, , Hungary

Site Status

IRCCS Institute of Neurological Sciences of Bolgna, Bellaria Hospital

Bologna, , Italy

Site Status

ASST Spedali Civili di Brescia

Brescia, , Italy

Site Status

Azienda Ospedaliero Universitaria Policlinico G. Martino

Messina, , Italy

Site Status

Istituto Nazionale Neurologico Carlo Besta

Milan, , Italy

Site Status

Istituto di Neurologia, Fondazione Policlinico Universitario A. Gemelli

Roma, , Italy

Site Status

Dipartimento Ambientale di Neuroscienze

Roma, , Italy

Site Status

Ospedale Pediatrico Bambin Gesu

Roma, , Italy

Site Status

Hospital Universitario 12 de Octubre

Madrid, , Spain

Site Status

Neurology Department Hospital Universitari i Politecnic La Fe

Valencia, , Spain

Site Status

MRC Centre for Neuromuscular Diseases

London, , United Kingdom

Site Status

Royal Victoria Infirmary

Newcastle upon Tyne, , United Kingdom

Site Status

Countries

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United States Australia Canada Denmark Germany Hungary Italy Spain United Kingdom

Other Identifiers

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SPIMM-300

Identifier Type: -

Identifier Source: org_study_id

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