Trials / Recruiting
RecruitingNCT06636383
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
GSDIa Disease Monitoring Program
- Status
- Recruiting
- Phase
- —
- Study type
- Observational
- Enrollment
- 140 (estimated)
- Sponsor
- Ultragenyx Pharmaceutical Inc · Industry
- Sex
- All
- Age
- 2 Years
- Healthy volunteers
- Not accepted
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.
Detailed description
The DTX401-CL401 Disease Monitoring Program (DMP) is a prospective, multicenter, long-term observational study to follow up participants with GSDIa for at least 10 years after the administration of DTX401.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| OTHER | No Intervention | No investigational/study product will be administered in this DMP. |
Timeline
- Start date
- 2024-11-04
- Primary completion
- 2036-12-01
- Completion
- 2036-12-01
- First posted
- 2024-10-10
- Last updated
- 2026-03-03
Locations
19 sites across 9 countries: United States, Brazil, Canada, Denmark, Germany, Italy, Japan, Netherlands, Spain
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06636383. Inclusion in this directory is not an endorsement.