Glycogen Storage Disorder (GSD)

Condition Description

Glycogen storage disease (GSD) is a rare metabolic disorder where the body is not able to properly store or break down glycogen, a form of sugar or glucose. GSD affects the liver, muscles and other areas of the body, depending on the specific type.

Active Trials

Upcoming Trials

Past Trials

  • TRIAL 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 GSD III

    DESCRIPTION: This study is a phase 1/2 first-in-human (FIH), study to evaluate the safety, tolerability, and pharmacokinetic (PK) of a single ascending dose (SAD) and repeat doses (RD) of UX053 in patients with GSD III. The SAD cohorts will be open-label (OL). There will be two types of RD cohorts, an open-label (OL-RD) and a randomized, double-blind (DB), and placebo-controlled (DB-RD).

    SPONSOR: Ultragenyx Pharmaceutical Inc.

    INDICATION: Glycogen Storage Disorder Type III (GSD III)

    STUDY PROTOCOL: UX053-CL-101

    PHASE: 1/2

    STATUS: Completed

    RECRUITING PATIENTS: No