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

Sponsor
Ultragenyx Pharmaceutical Inc (Industry)
Overall Status
Recruiting
CT.gov ID
NCT04909346
Collaborator
(none)
200
Enrollment
3
Locations
36.2
Anticipated Duration (Months)
66.7
Patients Per Site
1.8
Patients Per Site Per Month

Study Details

Study Description

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

Condition or Disease Intervention/Treatment Phase

    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.

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    200 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    Observational Clinical Study of Anti-AAV Seroprevalence in Subjects With Ornithine Transcarbamylase Deficiency, Glycogen Storage Disorder Type Ia, and Wilson Disease
    Actual Study Start Date :
    Jun 23, 2021
    Anticipated Primary Completion Date :
    Jun 30, 2024
    Anticipated Study Completion Date :
    Jun 30, 2024

    Outcome Measures

    Primary Outcome Measures

    1. Prevalence of Anti-AAV8 Antibodies in Subjects with OTC Deficiency or GSDIa [Up to 35 days]

    2. Prevalence of Anti-AAV9 Antibodies in Subjects with Wilson Disease [Up to 35 days]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Synexus Clinical Research US (Virtual Trial) Akron Ohio United States 44311
    2 Hospital Clínico Universitario de Santiago de Compostela Santiago De Compostela Spain 15706
    3 Synexus North East Clinical Research Centre Hexham United Kingdom NE46 1QJ

    Sponsors and Collaborators

    • Ultragenyx Pharmaceutical Inc

    Investigators

    • Study Director: Medical Director, Ultragenyx Pharmaceutical Inc

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Ultragenyx Pharmaceutical Inc
    ClinicalTrials.gov Identifier:
    NCT04909346
    Other Study ID Numbers:
    • UX431-CL001
    First Posted:
    Jun 1, 2021
    Last Update Posted:
    Aug 23, 2022
    Last Verified:
    Aug 1, 2022
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Hepatolenticular Degeneration
    Ornithine Carbamoyltransferase Deficiency Disease
    Glycogen Storage Disease

    Study Results

    No Results Posted as of Aug 23, 2022