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Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III) | Clinical Trials | Clareo Health

TERMINATEDPHASE1/PHASE2

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

NCT04990388•Ultragenyx Pharmaceutical Inc

View on ClinicalTrials.gov

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).

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Confirmed diagnosis of GSD III by gene sequencing or enzymatic testing
•Alanine aminotransferase at or below 5 times normal during the three months prior to dosing
•Willing and able to comply with standard dietary management of GSD III
•If a significant rise in ALT occurs after the prior dose, ALT should show a decreasing trend toward the subject's baseline value
•Total bilirubin, platelets and international normalized ratio (INR) is within normal limits

Exclusion Criteria

•History of liver transplant or currently awaiting liver transplant
•History of cirrhosis
•Active Hepatitis B or C
•Severe kidney impairment
•History of liver cancer or large liver tumors
•History of any cancer within the past 3 years
•Known history of HIV infection
•Known severe allergy to polyethylene glycol (PEG), polysorbate, or mRNA vaccine
•Heart failure that causes marked limitation in physical activity
•Poorly controlled diabetes
+6 more criteria

Locations (6)

University of California, Irvine

Orange, California, United States

Rare Disease Research

Atlanta, Georgia, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

University of Texas, Health Science Center of Houston

Houston, Texas, United States

Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

Milan, Italy

Hospital Universitario 12 de Octubre

Madrid, Spain

Key Dates

Start Date

October 18, 2021

Primary Completion Date

March 20, 2023

Completion Date

March 20, 2023

Last Updated

April 16, 2024

Enrollment

ACTUAL participants

Conditions

Glycogen Storage Disease Type III

Interventions

UX053

BIOLOGICAL

Placebo

OTHER

Antipyretic

DRUG

H2 Blocker

DRUG

H1 Blocker

DRUG

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Glycogen Storage Disease Type III

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COMPLETEDNA

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: April 16, 2024Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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