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Enzyme Replacement Therapy

UX053 for Glycogen Storage Disease

Phase 1 & 2
Waitlist Available
Research Sponsored by Ultragenyx Pharmaceutical Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from first dose of study drug through the end of study (up to day 90)

Summary

This trial is testing a new treatment called UX053 to see if it is safe for adults with Glycogen Storage Disease Type III. The study will give different amounts of the treatment to see how the body handles it and if there are any side effects. The goal is to find a safe dose that can help people with this condition.

Eligible Conditions
  • Glycogen Storage Disease Type III
  • Debrancher Deficiency

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from first dose of study drug through the end of study (up to day 90)
This trial's timeline: 3 weeks for screening, Varies for treatment, and from first dose of study drug through the end of study (up to day 90) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, Discontinuations, and/or Dose Changes

Trial Design

6Treatment groups
Experimental Treatment
Group I: UX053 or Placebo Dose Level DB-1RExperimental Treatment5 Interventions
Participants randomized to receive a single, peripheral IV infusion of UX053 or Placebo every 2 weeks (Q2W) for 5 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker.
Group II: UX053 Dose Level DB-3RExperimental Treatment5 Interventions
Participants randomized to receive a single, peripheral IV infusion of UX053 or Placebo every 2 weeks (Q2W) for 5 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker.
Group III: UX053 Dose Level DB-2RExperimental Treatment5 Interventions
Participants randomized to receive a single, peripheral IV infusion of UX053 or Placebo every 2 weeks (Q2W) for 5 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker.
Group IV: UX053 Dose Level 3S->OL-3RExperimental Treatment4 Interventions
Participants receive a single, peripheral intravenous (IV) infusion of UX053. After completion of the 90-day Follow up Period, participants can enter the open label repeat dose (OL-RD) cohort where they will receive UX053 every 4 weeks (Q4W) for 4 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker.
Group V: UX053 Dose Level 2S->OL-2RExperimental Treatment4 Interventions
Participants receive a single, peripheral intravenous (IV) infusion of UX053. After completion of the 90-day Follow up Period, participants can enter the open label repeat dose (OL-RD) cohort where they will receive UX053 every 4 weeks (Q4W) for 4 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker.
Group VI: UX053 Dose Level 1S ->OL-1RExperimental Treatment4 Interventions
Participants receive a single, peripheral intravenous (IV) infusion of UX053. After completion of the 90-day Follow up Period, participants can enter the open label repeat dose (OL-RD) cohort where they will receive UX053 every 4 weeks (Q4W) for 4 doses. Participants will also receive premedication, consisting of oral paracetamol/acetaminophen or ibuprofen, an H2 blocker, and an H1 blocker.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Antipyretic
2022
Completed Phase 3
~20

Find a Location

Who is running the clinical trial?

Ultragenyx Pharmaceutical IncLead Sponsor
93 Previous Clinical Trials
104,327 Total Patients Enrolled
13 Trials studying Glycogen Storage Disease
383 Patients Enrolled for Glycogen Storage Disease
Medical Director, MD, PhDStudy DirectorUltragenyx Pharmaceutical Inc
1 Previous Clinical Trials
72 Total Patients Enrolled
Medical DirectorStudy DirectorUltragenyx Pharmaceutical Inc
2,885 Previous Clinical Trials
8,088,776 Total Patients Enrolled
8 Trials studying Glycogen Storage Disease
314 Patients Enrolled for Glycogen Storage Disease
~2 spots leftby Nov 2025
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