SLSMDS Natural History Study
NCT05029843 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 30
Last updated 2023-02-08
Summary
The Single Large-Scale mtDNA Deletion Sydrome: Natural History Study (PS-NHS) aims to collect data on standardized clinical outcomes, store data on the Champ Foundation Registry (CFR) and make this data available to researchers, clinicians, and industry partners who are studying SLSMDS to answer questions regarding the disease, including its causes, potential treatments, and other topics. A secondary aim is to analyze the data to understand research questions relating to the natural history of SLSMDS.
Conditions
- Pearson Syndrome
- Single Large Scale Mitochondrial DNA Deletion Syndromes (SLSMDS)
Sponsors & Collaborators
-
Children's Hospital of Philadelphia
collaborator OTHER -
The Cleveland Clinic
collaborator OTHER -
The Champ Foundation
lead OTHER
Principal Investigators
-
Sumit Parikh, MD · The Cleveland Clinic
-
Marni Falk, MD · Children's Hospital of Philadelphia
Eligibility
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2021-03-16
- Primary Completion
- 2024-10-16
- Completion
- 2024-10-16
Countries
- United States
Study Locations
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