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A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)

NCT05139316 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 49

Last updated 2026-03-25

No results posted yet for this study

Summary

The primary objectives of this study are to evaluate the efficacy of DTX401 to reduce or eliminate dependence on exogenous glucose replacement therapy to maintain euglycemia and to maintain or improve the quality of glucose control.

Conditions

  • Glycogen Storage Disease Type IA

Interventions

GENETIC

DTX401

nonreplicating, recombinant, adeno-associated virus (AAV) serotype 8 (AAV8)

OTHER

Placebo

Normal Saline infusion

DRUG

Oral prednisolone

Participants who receive DTX401 solution will receive oral prednisolone

DRUG

Placebo for oral prednisolone

Participants who receive placebo will receive placebo oral prednisolone to maintain the study blind

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical Inc

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-11-08
Primary Completion
2024-02-20
Completion
2026-02-20
FDA Drug
Yes

Countries

  • United States
  • Brazil
  • Canada
  • Denmark
  • Germany
  • Italy
  • Japan
  • Netherlands
  • 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 NCT05139316 on ClinicalTrials.gov