Status:
TERMINATED
Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
Lead Sponsor:
Ultragenyx Pharmaceutical Inc
Conditions:
Ornithine Transcarbamylase Deficiency
Wilson Disease
Eligibility:
All Genders
18+ years
Brief Summary
The objective of this observational study is to evaluate the seroprevalence of anti-AAV antibodies in subjects with Ornithine Transcarbamylase (OTC) deficiency, Glycogen Storage Disease Type Ia (GSDIa...
Detailed Description
The study is primarily designed to follow a virtual model, in which Screening will take place over a telephone/video call. The study will comprise a single home health visit, either on the same day as...
Eligibility Criteria
Inclusion
- Diagnosis of OTC deficiency, GSDIa, or Wilson Disease.
- Provide informed consent after the nature of the study has been explained, and prior to any research-related procedures.
Exclusion
- Prior exposure to an AAV-based gene therapy.
- Concurrent or previous participation in another Ultragenyx clinical study.
- Recipient of a liver transplant.
- Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results.
Key Trial Info
Start Date :
June 23 2021
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
November 17 2022
Estimated Enrollment :
51 Patients enrolled
Trial Details
Trial ID
NCT04909346
Start Date
June 23 2021
End Date
November 17 2022
Last Update
June 12 2023
Active Locations (5)
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1
Synexus Clinical Research US (Virtual Trial)
Akron, Ohio, United States, 44311
2
Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil, 90560-030
3
McGill University Health Center
Montreal, Quebec, Canada, H4A 3J1
4
Hospital Clínico Universitario de Santiago de Compostela
Santiago de Compostela, Spain, 15706