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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Total Enrollment

51 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-06-23

Study Completion Date

2022-11-17

Brief Summary

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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

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Detailed Description

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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.

Conditions

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Ornithine Transcarbamylase Deficiency Wilson Disease Glycogen Storage Disease Type IA

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No