NCT00947960 - Triheptanoin Treatment Trial for Patients With Adult Polyglucosan Body Disease | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

June 30, 2009

Primary Completion Date

July 31, 2015

Study Completion Date

July 31, 2015

Conditions

Adult Polyglucosan Body DiseaseGlycogen Brancher Enzyme DeficiencyGlycogen Storage Disease Type IV

Interventions

DRUG

Triheptanoin

1-2 grams triheptanoin (drug)/kilogram body weight divided into 4 equal doses per day taken with meals and a snack for 6 months followed by 1-2 grams vegetable oil (placebo)/kilogram body weight divided into 4 equal doses per day taken with meals and a snack for 6 months.

OTHER

Vegetable Oil

1-2 grams vegetable oil (placebo)/kilogram body weight divided into 4 equal doses per day taken with meals and a snack for 6 months followed by 1-2 g triheptanoin (drug)/kilogram body weight for 6 months.

Trial Locations (1)

75013

Department of Genetics, Groupe Hospitalier Pitié-Salpêtrière, Paris