Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
Purpose
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.
Condition
- Glycogen Storage Disease Type Ia
Eligibility
- Eligible Ages
- Over 2 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
| Group 1: DTX401 in Prior Clinical Study | Patients administered DTX401(pariglasgene brecaparvovec), full or partial dose, in prior clinical study involving DTX401 |
|
| Group 2: DTX401 in Post-Marketing Setting | Patients administered prescribed DTX401(pariglasgene brecaparvovec), full or partial dose, in a post-marketing setting |
|
Recruiting Locations
Children's Hospital of Orange County
Orange 5379513, California 5332921 92868
Orange 5379513, California 5332921 92868
Children's Hospital Colorado
Denver 5419384, Colorado 5417618 80045
Denver 5419384, Colorado 5417618 80045
University of Connecticut Health Center
Hartford 4835797, Connecticut 4831725 06106
Hartford 4835797, Connecticut 4831725 06106
University of Michigan
Ann Arbor 4984247, Michigan 5001836 48109
Ann Arbor 4984247, Michigan 5001836 48109
Duke University Medical Center
Durham 4464368, North Carolina 4482348 27710
Durham 4464368, North Carolina 4482348 27710
Children's Hospital of Philadelphia
Philadelphia 4560349, Pennsylvania 6254927 19104
Philadelphia 4560349, Pennsylvania 6254927 19104
University of Texas Health Science Center at Houston
Houston 4699066, Texas 4736286 77030
Houston 4699066, Texas 4736286 77030
More Details
- Status
- Recruiting
- Sponsor
- Ultragenyx Pharmaceutical Inc
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.