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

NCT04990388 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2024-04-16

Study results available
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Summary

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

Conditions

  • Glycogen Storage Disease Type III

Interventions

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

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical Inc

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-10-18
Primary Completion
2023-03-20
Completion
2023-03-20
FDA Drug
Yes

Countries

  • United States
  • Italy
  • Spain

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04990388 on ClinicalTrials.gov