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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 48 weeks

Awards & highlights

Study Summary

This trial is testing a new drug to treat GSD III, a rare genetic disorder that causes a build-up of glycogen in the liver. The goal is to see if the drug is safe for use in humans.

Eligible Conditions

Glycogen Storage Disease Type III

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 48 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~48 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 48 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence and severity of treatment-emergent adverse events (TEAEs), serious TEAEs, and related TEAEs in the SAD and RD Periods

Secondary outcome measures

PK parameters of AGL mRNA: AUC from time 0 to end of dosing period (AUCtau; RD cohorts only)

PK parameters of AGL mRNA: AUC from time 0 to infinity (AUCinf)

PK parameters of AGL mRNA: accumulation ratio (calculated as AUC after repeat dose / AUC after a single dose; RAUC; RD cohorts only)

+17 more

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

Group III: UX053 Dose Level DB-2RExperimental Treatment5 Interventions

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

Group VI: UX053 Dose Level 1S ->OL-1RExperimental Treatment4 Interventions

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Ultragenyx Pharmaceutical IncLead Sponsor

89 Previous Clinical Trials

179,066 Total Patients Enrolled

12 Trials studying Glycogen Storage Disease

238 Patients Enrolled for Glycogen Storage Disease

Medical Director, MD, PhDStudy DirectorUltragenyx Pharmaceutical Inc

Medical DirectorStudy DirectorUltragenyx Pharmaceutical Inc

2,777 Previous Clinical Trials

8,063,478 Total Patients Enrolled

7 Trials studying Glycogen Storage Disease

169 Patients Enrolled for Glycogen Storage Disease

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What medical conditions does UX053 typically help?

"UX053 is effective in treating catarrh and other medical complaints like seasonal allergies, runny nose, and muscle pain."

Answered by AI

Are there any opportunities for new patients to join this research project?

"The most recent information from clinicaltrials.gov suggests that this trial is looking for patients. The first posting was on October 18th, 2021 and the last update occurred on October 20th, 2022."

Answered by AI

Are there any other drugs in development that are similar to UX053?

"Currently, there are 133UX053 studies underway with 40 of them in Phase 3. Most of these trials taking place in Pittsburgh, but UX053 is also being tested at 1,081 other sites."

Answered by AI

At how many distinct sites is this research being conducted?

"There are 9 enrolling patients for this trial. Locations include Rare Disease Research in Atlanta, University of Texas, Health Science Center of Houston in Houston, and Children's Hospital of Philadelphia in Orange."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2021. "Safety, Tolerability, and Pharmacokinetics of UX053 in Patients With Glycogen Storage Disease Type III (GSD III)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04990388.