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)

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

24

Participants

Timeline

Start Date

September 30, 2025

Primary Completion Date

October 2, 2026

Study Completion Date

October 2, 2026

Conditions
MELAS SyndromeLebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
Interventions
DRUG

Glycerol Tributyrate

Participants will be administered orally three times a day enteric hard capsules containing 500 mg of glycerol tributyrate with an 8-ounce glass of water on an empty stomach: morning, noon, and evening during the dose-escalation phase. Each MELAS participant will undergo a six-month dose escalation phase of glycerol tributyrate, starting at a dose of 1,000 mg (tid) per day during the first month followed by a monthly increase of 500 mg (tid) per dose of glycerol tributyrate with the maximal oral dose of glycerol tributyrate 3,500 mg (tid) per dose at the end of six-month-long dose escalation phase. Once the participant-specific fixed maximum tolerated dose (MTD) is determined, MELAS participants will take an oral administration of this MTD three times a day during the 12-month-long clinical phase.

Trial Locations (1)

20010

RECRUITING

Children's National Hospital, Washington D.C.

All Listed Sponsors
collaborator

National Center for Advancing Translational Sciences (NCATS)

NIH

collaborator

Children's National Research Institute

OTHER

lead

George Washington University

OTHER