NCT03137355

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
200

participants targeted

Target at P75+ for all trials

Timeline
50mo left

Started Jun 2015

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress73%
Jun 2015Jun 2030

Study Start

First participant enrolled

June 17, 2015

Completed
1.9 years until next milestone

First Submitted

Initial submission to the registry

April 28, 2017

Completed
4 days until next milestone

First Posted

Study publicly available on registry

May 2, 2017

Completed
13.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 17, 2030

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 17, 2030

Last Updated

December 1, 2023

Status Verified

November 1, 2023

Enrollment Period

15 years

First QC Date

April 28, 2017

Last Update Submit

November 30, 2023

Conditions

Leigh SyndromeLeigh DiseaseLeigh's Necrotizing EncephalopathySubacute Necrotizing EncephalomyopathySubacute Necrotizing Encephalomyelopathy

Keywords

Leigh SyndromeLeigh DiseaseLeigh's Necrotizing EncephalopathySubacute Necrotizing EncephalomyelopathySubacute Necrotizing Encephalomyopathy

Outcome Measures

Primary Outcomes (1)

  • Phenotypical characteristics of Leigh syndrome

    The goal of this project is to collect longitudinal data on the natural history of Leigh syndrome.

    10 years

Study Arms (1)

Leigh syndrome

All people diagnosed with Leigh syndrome.

Eligibility Criteria

Age0 Days - 100 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

People with a diagnosis of Leigh syndrome.

You may qualify if:

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

You may not qualify if:

  • People without Leigh syndrome

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

RECRUITING

Related Links

MeSH Terms

Conditions

Leigh DiseaseNecrotizing encephalopathy, infantile subacute, of Leigh

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesPyruvate Metabolism, Inborn ErrorsCarbohydrate Metabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic DiseasesMitochondrial Diseases

Study Officials

  • Mary Kay Koenig, MD

    The University of Texas Health Science Center, Houston

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Mary Kay Koenig, MD

CONTACT

713-500-7164leigh@uth.tmc.edu

William Guerra

CONTACT

713-500-7164leigh@uth.tmc.edu

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
OTHER
Target Duration
10 Years
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

April 28, 2017

First Posted

May 2, 2017

Study Start

June 17, 2015

Primary Completion (Estimated)

June 17, 2030

Study Completion (Estimated)

June 17, 2030

Last Updated

December 1, 2023

Record last verified: 2023-11

Locations

US(1)