Observational Study of Anti-AAV for OTC, GSDIa or Wilson Disease
Research type
Research Study
Full title
Observational Clinical Study of Anti-AAV Seroprevalence in Subjects With Ornithine Transcarbamylase Deficiency, Glycogen Storage Disorder Type Ia, and Wilson Disease
IRAS ID
305198
Contact name
Shalini Iyengar
Contact email
Sponsor organisation
Ultragenyx Pharmaceutical, Inc
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 3 months, 2 days
Research summary
Study UX431-CL001 is sponsored by Ultragenyx Pharmaceutical, Inc. The purpose of this Observational study is to understand the incidence of Anti-adeno-associated virus (AAV) antibodies in participants diagnosed with Ornithine Transcarbamylase Deficiency (OTC), Glycogen Storage Disorder Type Ia (GSDIa), or Wilson Disease. AAV vectors are frequently used for gene therapy. Currently, the prevalence of neutralising antibodies against the AAV vector in participants with OTC deficiency, GSDIa, and Wilson Disease is unknown.
Approximately 200 participants aged 18 years and above with OTC, GSDIa or Wilson Disease will participate in the study globally. The total duration of the study for each participant will be up to 35 days.
The study will consist of two visits and a follow up phone call.
• Screening: The screening visit will be completed by a PPD VRCC nurse over the phone. After obtaining consent, the nurse will ask participants about their medical history, height, weight. This information will decide if a participant is eligible to take part in the study.• Day 1 visit: The Day 1 Visit will occur within 30 days of the screening visit and will take place at participant home. A study home health care provider will obtain blood and possibly urine samples, ask if participants have had any changes in their health and will ask if there have been any changes in medications
A PPD VRCC nurse will contact participants by phone 5 days after the home visit to ask if they have had any changes to their health since the home visit and will ask if there have been any changes in medications.
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
21/YH/0298
Date of REC Opinion
6 Jan 2022
REC opinion
Further Information Favourable Opinion