Database for patients with Leigh syndrome
The International Registry for Leigh Syndrome
This study is creating a database to gather information from people with Leigh syndrome to better understand the condition and improve treatment options.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 200 (estimated) |
| Ages | 0 Days to 100 Years |
| Sex | All |
| Sponsor | The University of Texas Health Science Center, Houston Academic / other |
| Locations | 1 site (Houston, Texas) |
| Trial ID | NCT03137355 on ClinicalTrials.gov |
What this trial studies
This observational study aims to create a comprehensive database that collects clinical and laboratory information from patients diagnosed with Leigh syndrome. By gathering natural history data, the study seeks to enhance the understanding of this rare neurodegenerative disorder, which is characterized by mitochondrial dysfunction. The information collected will help identify trends and prognostic factors, ultimately improving treatment approaches and patient outcomes. The study focuses on characterizing the disease more effectively through a collaborative effort among affected individuals.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals diagnosed with Leigh syndrome.
Not a fit: Patients without a diagnosis of Leigh syndrome will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved understanding and management of Leigh syndrome, potentially enhancing patient care and treatment outcomes.
How similar studies have performed: While there have been limited studies on Leigh syndrome, the establishment of a dedicated database for such a rare condition is a novel approach that has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * All participants with a diagnosis of Leigh syndrome will be invited to participate Exclusion Criteria: * People without Leigh syndrome
Where this trial is running
Houston, Texas
- The University of Texas Health Science Center at Houston — Houston, Texas, United States (Recruiting)
Study contacts
- Principal investigator: Mary Kay Koenig, MD — The University of Texas Health Science Center, Houston
- Study coordinator: Mary Kay Koenig, MD
- Email: leigh@uth.tmc.edu
- Phone: 713-500-7164
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.