Glycerol-Phenylbutyrate Treatment in Children With MCT Mutation (Allan-Herndon- Dudley Syndrome)

PHASE2/PHASE3UnknownINTERVENTIONAL
Enrollment

6

Participants

Timeline

Start Date

June 30, 2021

Primary Completion Date

August 30, 2022

Study Completion Date

August 30, 2022

Conditions
Monocarboxylate Transporter 8 Deficiency
Interventions
DRUG

Glycerol Phenylbutyrate 1100 MG/ML

Daily adminisitraion of the study drug (three times a day) in escalting dose over 4 months

Trial Locations (1)

76100

RECRUITING

Pediatric Endocrinology Unit, Kapan Medical Center, Rehovot

Sponsors
All Listed Sponsors
collaborator

Weizmann Institute of Science

OTHER

lead

Kaplan Medical Center

OTHER

NCT05019417 - Glycerol-Phenylbutyrate Treatment in Children With MCT Mutation (Allan-Herndon- Dudley Syndrome) | Biotech Hunter | Biotech Hunter