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Clinical Trial Summary

The Pearson Syndrome 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 Pearson syndrome.


Clinical Trial Description

This study is a prospective, observational, and longitudinal study intended to track the course of Pearson syndrome and single large scale mitochondrial DNA deletion syndromes (SLSMDS) to identify demographic, genetic, environmental, and other variables that correlate with the diseases development and outcomes. If available, retrospective clinical data may be accessed and used in analyses as well. The PS-NHS will be conducted at two Center of Excellence sites: the Cleveland Clinic and Children's Hospital of Philadelphia (CHOP). All PS-NHS data will be entered and stored on the CFR. The CFR exists entirely online. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05029843
Study type Observational
Source The Champ Foundation
Contact Elizabeth Reynolds, PhD
Phone 727-612-4606
Email [email protected]
Status Recruiting
Phase
Start date March 16, 2021
Completion date March 16, 2023

See also
  Status Clinical Trial Phase
Completed NCT02327364 - Natural History of Pearson Syndrome
Completed NCT03384420 - A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome Phase 1/Phase 2
Terminated NCT02104336 - Phase 2 Study of EPI-743 in Children With Pearson Syndrome Phase 2