Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease

Stopped early

Conditions studied: Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA

In brief

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), and Wilson Disease

Key facts

Study ID

NCT04909346

Run by

Ultragenyx Pharmaceutical Inc

People needed

51

Starts

2021-06-23

Expected to finish

2022-11-17

Last updated by the study team

2023-06-12

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Synexus Clinical Research US (Virtual Trial) — Akron, Ohio, United States
• Hospital de Clinicas de Porto Alegre — Porto Alegre, Rio Grande do Sul, Brazil
• McGill University Health Center — Montreal, Quebec, Canada
• Hospital Clínico Universitario de Santiago de Compostela — Santiago de Compostela, Spain
• Synexus North East Clinical Research Centre — Hexham, United Kingdom

Full record on ClinicalTrials.gov

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