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

Ultragenyx

Status: Terminated

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

Study type: Observational

Funder types: Industry

Identifiers

NCT04909346

UX431-CL001

Details and patient eligibility

About

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

Full 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.

Enrollment: 51 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.