NCT04909346

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

Observational Clinical Study of Anti-AAV Seroprevalence in Subjects With Ornithine Transcarbamylase Deficiency, Glycogen Storage Disorder Type Ia, and Wilson Disease

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 51 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Ornithine Transcarbamylase Deficiency
• Wilson Disease
• Glycogen Storage Disease Type IA

Timeline

• Start date: 2021-06-23
• Primary completion: 2022-11-17
• Completion: 2022-11-17
• First posted: 2021-06-01
• Last updated: 2023-06-12

Locations

5 sites across 5 countries: United States, Brazil, Canada, Spain, United Kingdom