MedDataX Logo

The International Registry for Leigh Syndrome

NCT ID: NCT03137355

Last Updated: 2023-12-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Total Enrollment

200 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-06-17

Study Completion Date

2030-06-17

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Leigh Syndrome Leigh Disease Leigh's Necrotizing Encephalopathy Subacute Necrotizing Encephalomyopathy Subacute Necrotizing Encephalomyelopathy

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Leigh Syndrome Leigh Disease Leigh's Necrotizing Encephalopathy Subacute Necrotizing Encephalomyelopathy Subacute Necrotizing Encephalomyopathy

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

CASE_ONLY

Study Time Perspective

OTHER

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Leigh syndrome

All people diagnosed with Leigh syndrome.

No interventions assigned to this group

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

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

Exclusion Criteria

* People without Leigh syndrome
Minimum Eligible Age

0 Days

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

The University of Texas Health Science Center, Houston

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Mary Kay Koenig

Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Mary Kay Koenig, MD

Role: PRINCIPAL_INVESTIGATOR

The University of Texas Health Science Center, Houston

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

The University of Texas Health Science Center at Houston

Houston, Texas, United States

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

United States

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Mary Kay Koenig, MD

Role: CONTACT

Phone: 713-500-7164

Email: [email protected]

William Guerra

Role: CONTACT

Phone: 713-500-7164

Email: [email protected]

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

William Guerra

Role: primary

Related Links

Access external resources that provide additional context or updates about the study.

http://peopleagainstleighs.org/registry/

Related Info

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

HSC-MS-14-0907

Identifier Type: -

Identifier Source: org_study_id