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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03048617




Registration number
NCT03048617
Ethics application status
Date submitted
3/02/2017
Date registered
9/02/2017
Date last updated
11/04/2019

Titles & IDs
Public title
An Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)
Scientific title
A Prospective Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)
Secondary ID [1] 0 0
SPIMM-300
Universal Trial Number (UTN)
Trial acronym
RePOWER
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Mitochondrial Disease 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Assess the relationship of genotype to phenotype in patients with Primary Mitochondrial Disease
Timepoint [1] 0 0
1 year
Primary outcome [2] 0 0
Compare local and regional differences in standard of care and management of patients with Primary Mitochondrial Disease
Timepoint [2] 0 0
1 year
Secondary outcome [1] 0 0
Compare local and regional differences in genetic testing methodologies for Primary Mitochondrial Disease
Timepoint [1] 0 0
1 year

Eligibility
Key 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
Minimum age
16 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
13/02/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
7/03/2019
Sample size
Target
Accrual to date
Final
215
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Calvary Health Care Bethlehem - Caulfield South
Recruitment hospital [2] 0 0
Royal North Shore Hospital - St Leonards
Recruitment postcode(s) [1] 0 0
- Caulfield South
Recruitment postcode(s) [2] 0 0
- St Leonards
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
Ohio
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas
Country [8] 0 0
United States of America
State/province [8] 0 0
Utah
Country [9] 0 0
United States of America
State/province [9] 0 0
Washington
Country [10] 0 0
Canada
State/province [10] 0 0
Alberta
Country [11] 0 0
Canada
State/province [11] 0 0
Ontario
Country [12] 0 0
Canada
State/province [12] 0 0
Vancouver
Country [13] 0 0
Denmark
State/province [13] 0 0
Copenhagen
Country [14] 0 0
Germany
State/province [14] 0 0
Bavaria
Country [15] 0 0
Germany
State/province [15] 0 0
Bonn
Country [16] 0 0
Hungary
State/province [16] 0 0
Budapest
Country [17] 0 0
Italy
State/province [17] 0 0
Bologna
Country [18] 0 0
Italy
State/province [18] 0 0
Brescia
Country [19] 0 0
Italy
State/province [19] 0 0
Messina
Country [20] 0 0
Italy
State/province [20] 0 0
Milano
Country [21] 0 0
Italy
State/province [21] 0 0
Roma
Country [22] 0 0
Spain
State/province [22] 0 0
Madrid
Country [23] 0 0
Spain
State/province [23] 0 0
Valencia
Country [24] 0 0
United Kingdom
State/province [24] 0 0
London
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Newcastle upon Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Stealth BioTherapeutics Inc.
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03048617
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT03048617