The Leigh Syndrome Registry
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|ClinicalTrials.gov Identifier: NCT03137355|
Recruitment Status : Recruiting
First Posted : May 2, 2017
Last Update Posted : October 25, 2018
|Condition or disease|
|Leigh Syndrome Leigh Disease Leigh's Necrotizing Encephalopathy Subacute Necrotizing Encephalomyopathy Subacute Necrotizing Encephalomyelopathy|
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.
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||200 participants|
|Target Follow-Up Duration:||10 Years|
|Official Title:||The International Database for Leigh Syndrome|
|Actual Study Start Date :||June 17, 2015|
|Estimated Primary Completion Date :||June 17, 2025|
|Estimated Study Completion Date :||June 17, 2025|
All people diagnosed with Leigh syndrome.
- Phenotypical characteristics of Leigh syndrome [ Time Frame: 10 years ]The goal of this project is to collect longitudinal data on the natural history of Leigh syndrome.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03137355
|Contact: Rahmat Adejumo, MBBSemail@example.com|
|Contact: Stephanie Maldonadofirstname.lastname@example.org|
|United States, Texas|
|The University of Texas Health Science Center at Houston||Recruiting|
|Houston, Texas, United States, 77030|
|Contact: Parisa Merhzad 713-500-7164 email@example.com|
|Principal Investigator:||Rahmat Adejumo, MBBS||The University of Texas Health Science Center, Houston|
|Study Chair:||Mary Kay Koenig, MD||The University of Texas Health Science Center, Houston|