A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)

NCT ID
NCT06792500
Registry status
Not yet recruiting
Study type
Interventional
Phase
Phase 1 / Phase 2
Sponsor
George Washington University
Conditions
Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus), MELAS Syndrome

Overview

ClinicalTrials.gov lists this study as Not yet recruiting.

Official title: Investigational Study of Glycerol Tributyrate in MELAS and LHON-Plus

Enrollment: 24 participants (Estimated).

What is being studied

  • Drug: Glycerol Tributyrate

Who may be eligible

Ages
18 Years to 65 Years
Sex
All
Healthy volunteers accepted
No

ClinicalTrials.gov lists the following eligibility criteria.

Inclusion Criteria: * Participants must be aged 18 to 65 years * A confirmed molecular diagnosis of MELAS or LHON-Plus * Symptomatic participants with MELAS harboring the m.3243A\>G variant or a mitochondrial pathogenic variant solely mapping in a mitochondrial gene encoding a mitochondrial subunit of Complex I * Symptomatic participants with LHON-Plus harboring the m.11778G\>A or a mitochondrial pathogenic variant only mapping in a mitochondrial gene encoding a mitochondrial subunit of Complex I * Normal enzymatic Complex II activity * Participants able to swallow capsules and comply with the requirements of the study according to the opinion of the investigator * Able to give written, informed consent * Participants who are sexually active and/or fertile must use an effective birth control during the study Exclusion Criteria: * • History of another primary mitochondrial disorder * Participants acutely ill * Positive urine pregnancy test for female subjects of childbearing potential within seven days prior to the first dose of the investigational drug * Pregnancy and/or breastfeeding * Participating in another mitochondrial disorder trial * Participated in another mitochondrial disorder trial within the last six months * On a current therapy with other investigational agents * Absence of neurological symptoms, muscle weakness, or exercise intolerance * Presence of any of the following signs or symptoms in the past six months at grade 3 or higher based on the CTCAE version 4.03: nausea, vomiting, diarrhea, hypoglycemia, hyperglycemia, dizziness, blurred vision, or syncope * A known hypersensitivity to any excipient contained in the drug formulation * Current abuse of drugs and/or alcohol * Unable to consent for themselves * Participants with an enteral feeding tube * Inability to travel to the study site

Study locations

1 study location is listed.

Site recruitment status is not specified in the registry data for these locations.

Countries: United States

  • District of Columbia, United States: 1

Sample of listed locations

  • Children's National Hospital — Washington D.C., District of Columbia, 20010, United States — Site recruitment status not specified in the registry data

Study details

Start date
March 1, 2027
Primary completion date
September 1, 2028
Completion date
November 1, 2028
First posted
January 24, 2025
Collaborators
Children's National Research Institute, National Center for Advancing Translational Sciences (NCATS)

Source and update information

Source
ClinicalTrials.gov
NCT ID
NCT06792500
Registry last updated
October 5, 2026
QualifiedStudies registry data refreshed
October 12, 2026
QualifiedStudies record fetched
October 12, 2026

View NCT06792500 on ClinicalTrials.gov

Trial information can change. Confirm current study details with the study team or the registry.

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