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

Ultragenyx Pharmaceutical Inc51 enrolled

Overview

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

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 Type

OBSERVATIONAL

Enrollment

Conditions

Ornithine Transcarbamylase Deficiency Wilson Disease Glycogen Storage Disease Type IA

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (5)

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

Outcomes

Primary Outcomes

Prevalence of Anti-AAV8 Antibodies in Subjects with OTC Deficiency or GSDIa

Time frame: Up to 35 days

Prevalence of Anti-AAV9 Antibodies in Subjects with Wilson Disease

Time frame: Up to 35 days

Data from ClinicalTrials.gov

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