24
Participants
Start Date
September 30, 2025
Primary Completion Date
October 2, 2026
Study Completion Date
October 2, 2026
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.
RECRUITING
Children's National Hospital, Washington D.C.
Collaborators (1)
National Center for Advancing Translational Sciences (NCATS)
NIH
Children's National Research Institute
OTHER
George Washington University
OTHER