Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT03137355 |
| Other study ID # |
HSC-MS-14-0907 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
June 17, 2015 |
| Est. completion date |
June 17, 2030 |
Study information
| Verified date |
November 2023 |
| Source |
The University of Texas Health Science Center, Houston |
| Contact |
Mary Kay Koenig, MD |
| Phone |
713-500-7164 |
| Email |
leigh[@]uth.tmc.edu |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational [Patient Registry]
|
Clinical Trial Summary
The purpose of this study is to develop a database containing clinical and laboratory
information for patients with Leigh syndrome. The goal is to provide a greater understanding
of Leigh syndrome allowing further characterization of this disease.
Description:
Leigh syndrome, also known as juvenile sub-acute necrotizing encephalopathy, is a progressive
neurodegenerative disorder associated with dysfunction of mitochondrial oxidative
phosphorylation (OXPHOS). First described in 1951 by British neuropsychiatrist Archibald
Denis Leigh, the condition has evolved from a post mortem diagnosis to a clinical entity with
characteristic radiologic and laboratory findings.
Leigh syndrome is a rare and heterogeneous disease, finding a substantial number of patients
to study is difficult. The lack of natural history data in Leigh syndrome and the small
number of patients included in clinical reports thus far has limited the ability to fully
comprehend the progression of this disease and assess prognostic factors. A Leigh syndrome
database will help improve our understanding of this rare disease leading to an improved
ability to predict outcomes and/or improve treatment paradigms. Collecting natural history
data on Leigh syndrome and integrating this information into a database will be useful in
understanding the course of the disease and identifying trends.
