UX053 in GSD III



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Our team at the Congenital Hyperinsulinism Center at CHOP is working on a clinical drug trial that involves giving you a study drug called UX033. The FDA has not approved this drug for treatment of glycogen storage disease type III (GSD III) but has approved us to use this drug as part of this clinical trial.
The goal of the trial is to test the study drug safety, how your body processes the drug, and how the drug affects your body. You will be asked to participate in either Part 1 or Part 2 of the study:

  • Participation in Part 1 of this study will last for 3 months, will involve 10 study visits and you will receive one dose of UX053.
  • Participation in Part 2 of this study will last for up to 48 weeks, will involve 18 study visits and you will receive five doses of UX053 or a placebo.

It is possible that treatment with UX053 may improve your blood sugar levels. Travel costs will be covered by the study and you will receive compensation for participating.

Who Do I Contact?

If you are interested in participating in the study or want to learn more please contact our study team at HIResearch [at] chop.edu or 267-426-9915.

Eligibility & Criteria

IRB #:
Official Title:
A Phase 1/2 First-In-Human, 2-Part Study To Evaluate The Safety, Tolerability, and Pharmacokinetics Of Single Ascending Doses (Part 1: Open-label) And Repeat Doses (Part 2: Randomized, Double-blind, Placebo-Controlled) Of UX053 In Patients With Glycogen Storage Disease Type III (GSD III)
Study Phase:
Phase I
Phase II
Eligible Age Range:
18 - 99 Years
Study Categories:

Visit Criteria

If you agree to take part in this study, you will be assigned to one of two parts:
Part One: Your participation will last for 3 months and will involve 10 study visits. You will receive one dose of the study drug UX053 by infusion with an IV catheter.
Part Two: Your participation will last for 48 weeks and will involve 18 study visits. You will receive five doses of the study drug UX053 or placebo by infusion with an IV catheter. A placebo is a substance that does not contain the active study drug.
You will not be able to choose which part of the study to participate in. Assignment to Part 1 or Part 2 depends on when you enroll in the study.
The first group of participants who join the study will be in Part 1. After the safety data from Part 1 is reviewed and the data shows that it is safe to continue, Part 2 will begin.
There are differences between this study and your usual care. As a participant in the research, you will:

  • Receive the study drug or placebo by infusion
  • Have up to 5 hospital inpatient admissions
  • Have to wear a continuous glucose monitor (CGM) for at least 90 days
  • Have extra blood tests and urine collections
  • Have to complete an electronic diary (eDiary) daily

The main risks of this study are from the study drug UX053. These include: an immune reaction to the infusion, liver injury, and problems with blood clotting. You may benefit if drug UX053 proves to be effective at lowering the amount of glycogen in your body.