Study on AAV8 antibodies and other biomarkers in Pompe Disease
Research type
Research Study
Full title
A Study to Evaluate Seroprevalence of Antibodies to AAV8 and Assessment of Biomarkers in Patients with Late-Onset Pompe Disease
IRAS ID
333223
Contact name
Jordi Diaz Manera
Contact email
Sponsor organisation
Astellas Gene Therapies Inc.
Duration of Study in the UK
3 years, 0 months, 4 days
Research summary
Study AT845-02 is sponsored by Astellas Gene Therapies. The purpose of this study is to see if you have antibodies to a very small virus called adeno associated virus (AAV) in patients with Late Onset Pompe Disease (LOPD). The virus does not cause any disease in your body, however when you are exposed to the virus, the body may recognise the virus as a foreign substance and create antibodies against the virus. Acid Alpha-glucosidase (GAA) is required in the body to breakdown glycogen, which is a stored form of sugar used for energy. GAA is stored in an intracellular part of a cell called lysosome. When GAA is not present in the body, it causes the glycogen to build up in the lysosome, which causes damage to the cell, leading to muscle weakness. This study will help to increase the understanding of the occurrence of antibodies against AAV8 and GAA and to assess if the levels changes over time in participants with LOPD.
Approximately 100 participants from roughly 50 different study sites, aged 16 to 70 years will take part in the study. Duration of the study will be roughly 2 years and will include 7 visits to the hospital or clinic, each visit comprising roughly 1 day.
Study will involve participants having a Screening and Baseline study visit to confirm participants eligibility to take part in the study, informed consent will be taken, blood and urine samples will be collected, participants will be asked about their medical history, current and past medications, physicals exams, height and weight will be taken. The visits will be repeated roughly every 4 months for up to 2 years. The study team will let participants know when to attend the study visits and which visits may occur in participants home or at alternate location.
REC name
South Central - Hampshire B Research Ethics Committee
REC reference
24/SC/0027
Date of REC Opinion
26 Mar 2024
REC opinion
Further Information Favourable Opinion