A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
NCT ID: NCT05139316
Last Updated: 2025-11-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
ACTIVE_NOT_RECRUITING
PHASE3
49 participants
INTERVENTIONAL
2021-11-08
2026-02-28
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Study of Recombinant Human Insulin-Like Growth Factor I in Patients With Severe Insulin Resistance
NCT00004419
Intraoperative Dexamethasone and Its Effect on Hyperglycemia
NCT03646916
Insulinotropic Effect of GLP-1 and GIP After Dexamethasone
NCT02235584
Safety Study of Repeated Doses of Glucagon on Animal Starch in the Liver
NCT01986231
Somatostatin Analogue SOM230 (Pasireotide) in Healthy Male Volunteers
NCT01128192
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Participants will be randomized 1:1 to DTX401 or placebo group, and followed closely for 48 weeks. At week 48 eligible participants will cross over and receive DTX401 if they had previously received placebo or placebo if they had previously received DTX401, and will be followed closely for an additional 96 weeks. After completion of week 144 or early withdrawal, participants will be offered enrollment into a Disease Monitoring Program (DMP) where they will be followed for at least 10 years post DTX401 infusion.
In Japan, there will be a single open label study arm and all participants will be treated with DTX401. At week 48, Japanese participants will be offered enrollment into a Disease Monitoring Program (DMP) where they will be followed for at least 10 years post DTX401 infusion.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
CROSSOVER
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
DTX401, Then Placebo
Participants receive single peripheral intravenous (IV) infusion of DTX401 in solution. At week 48 participants receive single peripheral IV infusion of Placebo.
DTX401
nonreplicating, recombinant, adeno-associated virus (AAV) serotype 8 (AAV8)
Placebo
Normal Saline infusion
Oral prednisolone
Participants who receive DTX401 solution will receive oral prednisolone
Placebo for oral prednisolone
Participants who receive placebo will receive placebo oral prednisolone to maintain the study blind
Placebo, Then DTX401
Participants receive single peripheral IV infusion of Placebo. At week 48 eligible participants receive single peripheral IV infusion of DTX401 solution.
DTX401
nonreplicating, recombinant, adeno-associated virus (AAV) serotype 8 (AAV8)
Placebo
Normal Saline infusion
Oral prednisolone
Participants who receive DTX401 solution will receive oral prednisolone
Placebo for oral prednisolone
Participants who receive placebo will receive placebo oral prednisolone to maintain the study blind
DTX401 (Japan Only)
Participants receive single peripheral intravenous (IV)infusion of DTX401 in solution.
DTX401
nonreplicating, recombinant, adeno-associated virus (AAV) serotype 8 (AAV8)
Oral prednisolone
Participants who receive DTX401 solution will receive oral prednisolone
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
DTX401
nonreplicating, recombinant, adeno-associated virus (AAV) serotype 8 (AAV8)
Placebo
Normal Saline infusion
Oral prednisolone
Participants who receive DTX401 solution will receive oral prednisolone
Placebo for oral prednisolone
Participants who receive placebo will receive placebo oral prednisolone to maintain the study blind
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Currently receiving a therapeutic regimen of cornstarch (or equivalent), following international guidance/recommendations with stable nutrition, glycemic, and clinical status.
* Willing and able to complete the informed consent process and to comply with study procedures and visit schedule
* Females of childbearing potential and fertile males must consent to use highly effective contraception from the period following informed consent through the duration of the 144-week study and in cases of early withdrawal at least 48 weeks after the last dose of investigational product (IP). Female subjects must agree not to become pregnant. Male subjects must agree not to father a child or donate sperm
Exclusion Criteria
* History of liver transplant, including hepatocyte cell therapy/ transplant
* History of liver disease
* Presence of liver adenoma \>5 cm in size
* Presence of liver adenoma \>3 cm and ≤5 cm in size that has a documented annual growth rate of ≥0.5 cm per year
* Significant hepatic inflammation or cirrhosis as evidenced by imaging or any of the following laboratory abnormalities: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> upper limit of normal (ULN), total bilirubin \>1.5 × ULN, alkaline phosphatase \>2.5 × ULN
* Non-fasting triglycerides ≥1000 mg/dL
* Pregnant, breastfeeding, or planning to become pregnant (self or partner) at any time during the study.
* Current or previous participation in another gene transfer study
* History of illicit drug use within 60 days prior to screening or positive results from an 8-panel urine drug screen during the Screening Period completed at 2 time points at least 4 weeks apart
8 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Ultragenyx Pharmaceutical Inc
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Medical Director
Role: STUDY_DIRECTOR
Ultragenyx Pharmaceutical Inc
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Children's Hospital of Orange County
Orange, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
University of Connecticut Health Center
Farmington, Connecticut, United States
Mount Sinai
The Bronx, New York, United States
Duke University
Durham, North Carolina, United States
Cleveland Clinic
Cleveland, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Texas
Houston, Texas, United States
Primary Children's Hospital
Salt Lake City, Utah, United States
Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil
McGill University
Montreal, Quebec, Canada
Righospitalet
Copenhagen, Capital, Denmark
University Medical Center Eppendorf
Hamburg, , Germany
Istituto Giannina Gaslini
Genova, Linguria, Italy
University of Naples
Naples, , Italy
Kumamoto University Hospital
Kumamoto, , Japan
Osaka City General Hospital
Osaka, , Japan
Fujita Health University Hospital
Toyoake, , Japan
Groningen University
Groningen, , Netherlands
Hospital Clinico Universitario de Santiago
Santiago de Compostela, A Coruna, Spain
Countries
Review the countries where the study has at least one active or historical site.
Related Links
Access external resources that provide additional context or updates about the study.
Ultragenyx Transparency Commitment
Ultragenyx Patient Advocacy/GSDIA Disease Information
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2020-004184-12
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2023-508750-25-00
Identifier Type: CTIS
Identifier Source: secondary_id
DTX401-CL301
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.