Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I
Status:
Recruiting
Trial end date:
2021-11-30
Target enrollment:
Participant gender:
Summary
This study will be an open-label, prospective, interventional feasibility pilot project to
study the efficacy, safety, and tolerability of UX007 (triheptanoin) on reducing hypoglycemic
events in patients with GSD I. Subjects will serve as their own control. Five (5) subjects
who are treatment naïve to UX007 (triheptanoin) and are already on standard dietary therapy
for GSDI will be enrolled.
The primary objective is to evaluate the efficacy, safety, and tolerability of UX007
(triheptanoin) in patients with GSD I. The secondary objectives include evaluating the effect
of UX007 (triheptanoin) on maintaining the duration of normoglycemia between meals based on
glucose monitoring (Preventing and reducing the frequency of hypoglycemia);
reduction/stabilization of the dose of cornstarch; and the prevention of increased liver
steatosis based on ultrasound with elastography.