UX007 for Glycogen Storage Disease 1 - Clinical Trial

What is the Purpose of this Study?

We are doing this study to learn more about the study medicine (Triheptanoin oil) and to determine if it helps reduce times of low blood sugar that some people experience.

What is the Condition Being Studied?

Patients with Glycogen Storage Disease type 1 (GSD I)

Who Can Participate in the Study?

Adults and children diagnosed with Glycogen Storage Disease type 1 (GSD I).

Age Group
Adults, Children

What is Involved?

If you choose to join this study, you will:
- Have a physical exam
- Have blood and urine tests
- Get an ultrasound of your liver
- Take the study drug (Triheptanoin oil) at home as directed
- Complete 3 in-person study visits
- Complete 6 follow-ups on the phone

Study Details

Full Title
Anaplerotic Therapy Using Triheptanoin for Patients with Glycogen Storage Disease Type 1
Principal Investigator
Pediatric Medical Genetics Specialist
Protocol Number
IRB: PRO00103582
NCT: NCT03665636
Phase I
Enrollment Status
Open for Enrollment
Contact the Duke Recruitment Innovation Center