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

Terminated

• Conditions: Ornithine Transcarbamylase Deficiency; Glycogen Storage Disease Type IA; Wilson Disease

• Registration Number: NCT04909346
• Lead Sponsor: Ultragenyx Pharmaceutical Inc

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), and Wilson Disease

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 Screening or within 30 days of enrollment. A safety follow-up may occur up to 5 days after Day 1 assessments are completed.

Study Timeline

• Study First Submitted: 2021-05-26
• Study First Submitted QC: 2021-05-26
• Study First Posted: 2021-06-01
• Study Start: 2021-06-23
• Primary Completion: 2022-11-17
• Study Completion: 2022-11-17
• Status Verified: 2023-06
• Last Update Submitted: 2023-06-09
• Last Update Posted: 2023-06-12

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 51

Inclusion Criteria

• 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 Criteria

• 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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Prevalence of Anti-AAV8 Antibodies in Subjects with OTC Deficiency or GSDIa	Up to 35 days
Prevalence of Anti-AAV9 Antibodies in Subjects with Wilson Disease	Up to 35 days

Trial Locations

Locations (5)

Hospital Clínico Universitario de Santiago de Compostela; 🇪🇸 Santiago De Compostela, Spain

Hospital de Clinicas de Porto Alegre; 🇧🇷 Porto Alegre, Rio Grande Do Sul, Brazil

McGill University Health Center; 🇨🇦 Montréal, Quebec, Canada

Synexus North East Clinical Research Centre; 🇬🇧 Hexham, United Kingdom

Synexus Clinical Research US (Virtual Trial); 🇺🇸 Akron, Ohio, United States