NCT03642860View on ClinicalTrials.gov

The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses

PHASE2CompletedINTERVENTIONAL
Enrollment

3

Participants

Timeline

Start Date

August 15, 2018

Primary Completion Date

August 28, 2019

Study Completion Date

August 28, 2019

Conditions
Tarui DiseaseDebrancher DeficiencyGYG1 DEFICIENCY
Interventions
DRUG

Triheptanoin

Daily treatment with Triheptanoin oil for 14 days (7 days titration period in addition to 7 days full dose period with 1g/kg/day).

DRUG

Placebo Oil

Daily treatment with Safflower oil for 14 days (7 days titration period in addition to 7 days full dose period with 1g/kg/day).

Trial Locations (1)

2200

Copenhagen Neuromuscular Center, Copenhagen

All Listed Sponsors
collaborator

Ultragenyx Pharmaceutical Inc

INDUSTRY

lead

Rigshospitalet, Denmark

OTHER

NCT03642860 - The Effect of Triheptanoin on Fatty Acid Oxidation and Exercise Tolerance in Patients With Glycogenoses | Biotech Hunter | Biotech Hunter