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Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

RecruitingObservational study

GSDIa Disease Monitoring Program

Who can join

Ages 2 and older · All sexes

Full eligibility criteria
Inclusion Criteria:

* Patient who had:

  * DTX401 (full or partial dose) administered in a parent clinical study (Group 1) or
  * Prescribed DTX401(full or partial dose) administered in a post-marketing setting (Group 2)
* Patient is willing and able to provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures. If a minor or an adult with cognitive limitations, the patient is willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures.

Exclusion Criteria:

* Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator

About the 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.

What is being tested

Sponsor: Ultragenyx Pharmaceutical Inc · Participants: 140 · Started: Nov 4, 2024

Contact the study team

Official record on ClinicalTrials.gov — NCT06636383

Locations in the U.S.

CaliforniaChildren's Hospital of Orange County, Orange
ColoradoChildren's Hospital Colorado, Denver
ConnecticutUniversity of Connecticut Health Center, Hartford
MichiganUniversity of Michigan, Ann Arbor
North CarolinaDuke University Medical Center, Durham
OhioThe Cleveland Clinic Foundation, Cleveland (Not yet recruiting)
PennsylvaniaChildren's Hospital of Philadelphia, Philadelphia
TexasUniversity of Texas Health Science Center at Houston, Houston
UtahPrimary Children's Hospital, Salt Lake City (Not yet recruiting)

From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.