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Glycerol Tributyrate for MELAS and LHON-Plus

This study is testing a drug called glycerol tributyrate for people with Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus). The goal is to see if glycerol tributyrate is safe and if it can help improve how your body uses energy (oxidative phosphorylation metabolism). You could be eligible if you are 18 to 65 years old and have a confirmed diagnosis of MELAS or LHON-Plus with specific genetic changes. The study plans to enroll 24 participants. The current recruitment status is unclear.

Study design
This is an open-label, dose-escalation study, meaning everyone will know they are receiving glycerol tributyrate, and the dose may increase over time. It is a basket study, enrolling participants with MELAS and LHON-Plus at the same time, and plans to include 24 participants.
What's involved
Participants will take glycerol tributyrate orally three times a day on an empty stomach. Before starting the drug, there will be a two-month period to gather your medical history, perform physical exams, and conduct various tests, including blood and urine tests, and specialized exams for MELAS or LHON-Plus.
Compensation
Not stated in the trial record.
Follow-up
The safety and potential effectiveness of glycerol tributyrate will be assessed over 20 months.

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NCT06792500

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)

Not Yet Recruiting
PHASE1Ages 18–65InterventionalTreatment
George Washington University
~24 participants
Updated 2026-06-04 on ClinicalTrials.gov
What's tested:Glycerol Tributyrate

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Assessment of Dose Safety for Glycerol Tributyrate
Measured over 20 months
+1 more outcome measured

Conditions

MELAS Syndrome
Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)

Explore related trials

LHON-PlusMELAS SyndromeSuccinate Dehydrogenase

Where it's being run

1 sites across 1 states
District of Columbia1

Study leadership

  • Debra Regier, M.D., Ph.D. · PRINCIPAL_INVESTIGATOR · Children's National Hospital; Children's National Rare Disease Institute
  • Wei-Liang Chen, M.D. · STUDY_CHAIR · Children's National Research Institute
  • Anne Chiaramello, Ph.D. · STUDY_DIRECTOR · George Washington University School of Medicine and Health Sciences

Who to contact

Anne Chiaramello, Ph.D.,
[email protected]
(202) 994-2173
Email the study team

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Eligibility criteria

Inclusion

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

• 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

What this trial measures

  • Assessment of Dose Safety for Glycerol Tributyrate20 months

    To assess the dose safety of the investigational drug, glycerol tributyrate, the investigators will measure Incidence of Treatment-Emergent Adverse Events \[Safety and Tolerability\] using the CTCAE version 4.03

  • Potential Efficacy of Glycerol Tributyrate on the oxidative phsophorylation metabolism20 months

    The investigators will assess whether participant-specific maximum tolerated dose (MTD) of glycerol tributyate results in increase of the participant's bioenergetic parameters for the oxidative phosphorylation pathway and/or mitochondrial ATP rate production above the participant's baseline determined from a dermal fibroblasts derive from a skin biopsy during the two-month-long baseline lead-in phase.