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.
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).
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