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

April 9, 2019 updated by: Stealth BioTherapeutics Inc.

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
215

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • St Leonards, Australia
        • Royal North Shore Hospital
    • Victoria
      • Caulfield South, Victoria, Australia
        • Calvary Health Care Bethlehem
  • Canada
      • Vancouver, Canada
        • Adult Metabolic Diseases Clinic
    • Alberta
      • Calgary, Alberta, Canada
        • University of Calgary Metabolic Disease Clinica
    • Ontario
      • Hamilton, Ontario, Canada
        • McMaster University Medical Center
  • Denmark
      • Copenhagen, Denmark
        • University of Copenhagen
  • Germany
      • Bonn, Germany, 53105
        • University Hospital of Bonn
    • Bavaria
      • Munich, Bavaria, Germany, 80336
        • Klinikim der Universitat Muchen, Friedrich-Baur Institute
  • Hungary
      • Budapest, Hungary
        • Institute of Genomic Medicine and Rare Disorders
  • Italy
      • Bologna, Italy
        • IRCCS Institute of Neurological Sciences of Bolgna, Bellaria Hospital
      • Brescia, Italy
        • Asst Spedali Civili Di Brescia
      • Messina, Italy, 98125
        • Azienda Ospedaliero Universitaria Policlinico G. Martino
      • Milano, Italy
        • Istituto Nazionale Neurologico Carlo Besta
      • Roma, Italy, 00168
        • Istituto di Neurologia, Fondazione Policlinico Universitario A. Gemelli
      • Roma, Italy, 56126
        • Dipartimento Ambientale di Neuroscienze
      • Roma, Italy
        • Ospedale Pediatrico Bambin Gesù
  • Spain
      • Madrid, Spain
        • Hospital Universitario 12 de Octubre
      • Valencia, Spain, 46026
        • Neurology Department Hospital Universitari i Politecnic La Fe
  • United Kingdom
      • London, United Kingdom
        • MRC Centre for Neuromuscular Diseases
      • Newcastle upon Tyne, United Kingdom
        • Royal Victoria Infirmary
  • United States
    • California
      • La Jolla, California, United States, 92093
        • University of California San Diego
      • Palo Alto, California, United States, 94304
        • Stanford Universtity
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital
    • New York
      • New York, New York, United States, 10032
        • Columbia University Medical Center
    • Ohio
      • Akron, Ohio, United States, 44308
        • Akron Children's Hospital
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104'
        • The Children's Hospital of Philadelphia
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children's Hospital of Pittsburgh of UPMC
    • Texas
      • Houston, Texas, United States, 77030
        • Baylor College of Medicine
      • Houston, Texas, United States, 77030
        • The University of Texas Health Science Center
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • University of Utah Hospital
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

16 years to 80 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Probability Sample

Study Population

Patients with clinical presentation of PMD with either signs or symptoms suggestive of myopathy

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Case-Only
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Assess the relationship of genotype to phenotype in patients with Primary Mitochondrial Disease
Time Frame: 1 year
1 year
Compare local and regional differences in standard of care and management of patients with Primary Mitochondrial Disease
Time Frame: 1 year
1 year

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Compare local and regional differences in genetic testing methodologies for Primary Mitochondrial Disease
Time Frame: 1 year
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Stealth BioTherapeutics Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 13, 2017

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 2, 2018

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 7, 2019

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 3, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 6, 2017

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 9, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 11, 2019

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 9, 2019

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • SPIMM-300

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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