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

• Status: Recruiting
• Trial end date: 2023-09-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ultragenyx Pharmaceutical Inc
• Treatments: Acetaminophen; Antipyretics; Cetirizine; Famotidine; Ibuprofen

Criteria

Key Inclusion Criteria:

• Confirmed diagnosis of GSD III by gene sequencing or enzymatic testing

• History of significant hypoglycemia or elevation in liver enzyme (alanine aminotransferase) in the past year

• 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

Key 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

• Poorly controlled hypothyroidism

• Treatment with immunosuppressive medications such as those used to treat chronic autoimmune conditions and solid organ transplants

• Pregnant or nursing, or planning to become pregnant during the study

Note: Additional inclusion/exclusion criteria may apply, per protocol