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Study identifier: NCT06636383 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Condition: Glycogen Storage Disease Type Ia  ·  Sponsor: Ultragenyx Pharmaceutical Inc

PhaseN/A
Planned participants140
Who can joinAll sexes, 2 Years to no upper limit
Healthy volunteersNo

About this study

The main objective of this observational study is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after DTX401 administration.

This description comes directly from the study's public registry record.

Talk to the study team

Patients Contact: Trial Recruitment  ·  1-888-756-8657  ·  trialrecruitment@ultragenyx.com

HCPs Contact: Medical Information  ·  1-888-756-8657  ·  medinfo@ultragenyx.com

Always discuss trial participation with your own doctor first.

Locations (19)

Children's Hospital of Orange CountyOrange, California, United StatesRecruiting
Children's Hospital ColoradoDenver, Colorado, United StatesRecruiting
University of Connecticut Health CenterHartford, Connecticut, United StatesRecruiting
University of MichiganAnn Arbor, Michigan, United StatesRecruiting
Duke University Medical CenterDurham, North Carolina, United StatesRecruiting
The Cleveland Clinic FoundationCleveland, Ohio, United StatesNot Yet Recruiting
Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania, United StatesRecruiting
University of Texas Health Science Center at HoustonHouston, Texas, United StatesRecruiting
Primary Children's HospitalSalt Lake City, Utah, United StatesNot Yet Recruiting
Hospital de Clinicas de Porto Alegre (HCPA) - PPDSPorto Alegre, Rio Grande do Sul, BrazilRecruiting
McGill University Health CenterMontreal, Quebec, CanadaRecruiting
RigshospitaletCopenhagen, DenmarkRecruiting

+ 7 more locations — full list on the registry record.

Follow this study

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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.

Source record: clinicaltrials.gov/study/NCT06636383