NCT04990388

Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)

A Phase 1/2 First-in-human, Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses and Repeat Doses of UX053 in Patients With GSD III

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of the study is to evaluate the safety of UX053 in adults with Glycogen Storage Disease Type III (GSD III).

Detailed 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).

Conditions

• Glycogen Storage Disease Type III

Interventions

Type	Name	Description
BIOLOGICAL	UX053	mRNA-based biologic
OTHER	Placebo	consists of the same components as the formulation buffer for UX053
DRUG	Antipyretic	participants will receive oral premedication prior to infusion
DRUG	H2 Blocker	participants will receive oral premedication prior to infusion
DRUG	H1 Blocker	participants will receive oral premedication prior to infusion

Timeline

• Start date: 2021-10-18
• Primary completion: 2023-03-20
• Completion: 2023-03-20
• First posted: 2021-08-04
• Last updated: 2024-04-16
• Results posted: 2024-04-16

Locations

6 sites across 3 countries: United States, Italy, Spain

Regulatory

• FDA-regulated drug study