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

NCT04909346 | Terminated

• 1 other identifier: UX431-CL001
• Study Type: observational
• Target: 51
• Locations: 5 countries
• Sites: 5

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

Conditions

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

Outcome Measures

Primary Outcomes (2)

• 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

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Study population will include approximately equal proportions of subjects with OTC deficiency, GSDIa, and Wilson Disease

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Ultragenyx Pharmaceutical Inc: lead

Study Sites (5)

Synexus Clinical Research US (Virtual Trial)

Akron, Ohio, 44311, United States

Location

Hospital de Clinicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, 90560-030, Brazil

Location

McGill University Health Center

Montreal, Quebec, H4A 3J1, Canada

Location

Hospital Clínico Universitario de Santiago de Compostela

Santiago de Compostela, 15706, Spain

Location

Synexus North East Clinical Research Centre

Hexham, NE46 1QJ, United Kingdom

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Blood and urine samples for future biomarker assay development

MeSH Terms

Conditions

Ornithine Carbamoyltransferase Deficiency Disease; Hepatolenticular Degeneration; Hepatorenal form of glycogen storage disease

Condition Hierarchy (Ancestors)

Urea Cycle Disorders, Inborn; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Liver Diseases; Digestive System Diseases; Basal Ganglia Diseases; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Metal Metabolism, Inborn Errors

Study Officials

• Medical Director

  Ultragenyx Pharmaceutical Inc

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 26, 2021
• First Posted: June 1, 2021
• Study Start: June 23, 2021
• Primary Completion: November 17, 2022
• Study Completion: November 17, 2022
• Last Updated: June 12, 2023

Record last verified: 2023-06

Locations

US (1); ES (1); GB (1); CA (1); BR (1)