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
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- STATUS
- Recruiting
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- End date
- Sep 17, 2023
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- participants needed
- 30
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- sponsor
- Ultragenyx Pharmaceutical Inc
Summary
The primary objective of the study is to evaluate the safety of UX053 in adults with Glycogen Storage Disease Type III (GSD III).
Description
This study is a phase 1/2 first-in-human (FIH), 2-part study to evaluate the safety, tolerability, and pharmacokinetic (PK) of a single ascending dose (SAD; part 1) and repeat doses (RD; part 2) of UX053 in patients with GSD III. Single SAD and RD of UX053 are tested in separate cohorts. The SAD cohorts will be open-label, while the RD dose cohorts will be randomized, double-blind, and placebo-controlled.
Details
| Condition | Glycogen Storage Disease Type III |
|---|---|
| Treatment | Placebo, UX053, Antipyretic, Antipyretic, H2 Blocker, H2 Blocker, H1 Blocker, H1 Blocker |
| Clinical Study Identifier | NCT04990388 |
| Sponsor | Ultragenyx Pharmaceutical Inc |
| Last Modified on | 17 October 2022 |
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