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

Wilson Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04909346
• Other study ID #: UX431-CL001
• Status: Terminated
• Start date: June 23, 2021
• Est. completion date: November 17, 2022

Study information

• Verified date: June 2023
• Source: Ultragenyx Pharmaceutical Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 51
• Est. completion date: November 17, 2022
• Est. primary completion date: November 17, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

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

Locations

Country	Name	City	State
Brazil	Hospital de Clinicas de Porto Alegre	Porto Alegre	Rio Grande Do Sul
Canada	McGill University Health Center	Montréal	Quebec
Spain	Hospital Clínico Universitario de Santiago de Compostela	Santiago De Compostela
United Kingdom	Synexus North East Clinical Research Centre	Hexham
United States	Synexus Clinical Research US (Virtual Trial)	Akron	Ohio

Sponsors (1)

Lead Sponsor	Collaborator
Ultragenyx Pharmaceutical Inc

Countries where clinical trial is conducted

United States,  Brazil,  Canada,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Prevalence of Anti-AAV8 Antibodies in Subjects with OTC Deficiency or GSDIa		Up to 35 days
Primary	Prevalence of Anti-AAV9 Antibodies in Subjects with Wilson Disease		Up to 35 days