Pearson Syndrome Clinical Trial
Official title:
Single Large-scale mtDNA Deletion Syndrome Natural History Study
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.
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. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT02327364 -
Natural History of Pearson Syndrome
|
||
| Recruiting |
NCT06017869 -
Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-201 in Pediatric Patients With Pearson Syndrome
|
Phase 1 | |
| 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 | |
| Recruiting |
NCT05554835 -
Global Registry and Natural History Study for Mitochondrial Disorders
|
||
| Terminated |
NCT02104336 -
Phase 2 Study of EPI-743 in Children With Pearson Syndrome
|
Phase 2 |