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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
Sponsor:
Information provided by (Responsible Party):
Rahmat Adejumo, The University of Texas Health Science Center, Houston

Brief 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.

Condition or disease
Leigh Syndrome Leigh Disease Leigh's Necrotizing Encephalopathy Subacute Necrotizing Encephalomyopathy Subacute Necrotizing Encephalomyelopathy

Detailed 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.

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 200 participants
Observational Model: Case-Only
Time Perspective: Other
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


Group/Cohort
Leigh syndrome
All people diagnosed with Leigh syndrome.



Primary Outcome Measures :
  1. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 100 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
People with a diagnosis of Leigh syndrome.
Criteria

Inclusion Criteria:

  • All participants with a diagnosis of Leigh syndrome will be invited to participate

Exclusion Criteria:

  • People without Leigh syndrome

Information from the National Library of Medicine

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


Contacts
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Contact: Rahmat Adejumo, MBBS 713-500-7164 leigh@uth.tmc.edu
Contact: Stephanie Maldonado 713-500-7164 leigh@uth.tmc.edu

Locations
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United States, Texas
The University of Texas Health Science Center at Houston Recruiting
Houston, Texas, United States, 77030
Contact: Parisa Merhzad    713-500-7164    leigh@uth.tmc.edu   
Sponsors and Collaborators
The University of Texas Health Science Center, Houston
Investigators
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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
Additional Information:
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Responsible Party: Rahmat Adejumo, Instructor In Pediatrics, The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier: NCT03137355    
Other Study ID Numbers: HSC-MS-14-0907
First Posted: May 2, 2017    Key Record Dates
Last Update Posted: October 25, 2018
Last Verified: October 2018

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Rahmat Adejumo, The University of Texas Health Science Center, Houston:
Leigh Syndrome
Leigh Disease
Leigh's Necrotizing Encephalopathy
Subacute Necrotizing Encephalomyelopathy
Subacute Necrotizing Encephalomyopathy
Additional relevant MeSH terms:
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Mitochondrial Encephalomyopathies
Brain Diseases
Leigh Disease
Syndrome
Disease
Pathologic Processes
Central Nervous System Diseases
Nervous System Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Pyruvate Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors
Metabolic Diseases
Mitochondrial Diseases
Mitochondrial Myopathies
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases