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

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Brief Title

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

Official Title

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

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
    

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 Type

Observational


Primary Outcome

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


Condition

Ornithine Transcarbamylase Deficiency



Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

200

Start Date

June 23, 2021

Completion Date

June 30, 2024

Primary Completion Date

June 30, 2024

Eligibility Criteria

        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.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Medical Director, 1-888-756-8657, [email protected]

Location Countries

Canada

Location Countries

Canada

Administrative Informations


NCT ID

NCT04909346

Organization ID

UX431-CL001


Responsible Party

Sponsor

Study Sponsor

Ultragenyx Pharmaceutical Inc


Study Sponsor

Medical Director, Study Director, Ultragenyx Pharmaceutical Inc


Verification Date

September 2022