NCT06636383 Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
| NCT ID | NCT06636383 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Ultragenyx Pharmaceutical Inc |
| Condition | Glycogen Storage Disease Type Ia |
| Study Type | OBSERVATIONAL |
| Enrollment | 140 participants |
| Start Date | 2024-11-04 |
| Primary Completion | 2036-12 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 140 participants in total. It began in 2024-11-04 with a primary completion date of 2036-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
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
Contact & Investigator
Medical Director
STUDY DIRECTOR
Ultragenyx Pharmaceuticals Inc.
Frequently Asked Questions
Who can join the NCT06636383 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, studying Glycogen Storage Disease Type Ia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06636383 currently recruiting?
Yes, NCT06636383 is actively recruiting participants. Contact the research team at trialrecruitment@ultragenyx.com for enrollment information.
Where is the NCT06636383 trial being conducted?
This trial is being conducted at Orange, United States, Denver, United States, Hartford, United States, Ann Arbor, United States and 11 additional locations.
Who is sponsoring the NCT06636383 clinical trial?
NCT06636383 is sponsored by Ultragenyx Pharmaceutical Inc. The principal investigator is Medical Director at Ultragenyx Pharmaceuticals Inc.. The trial plans to enroll 140 participants.