Glycogen Storage Disease Type IA, Ornithine Transcarbamylase Deficiency, Wilson Disease
Conditions
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.
Interventions
None listed
Sponsors
Ultragenyx Pharmaceutical Inc
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 |
Countries
Brazil, Canada, Spain, United Kingdom, United States
Outcome results
None listed