Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
Trial website
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT03048617