Non-Interventional Post-Authorization Safety Study- OBS17445 (SAVANT)
Research type
Research Study
Full title
A Post-Authorization Safety Study (PASS) to Assess Long-term Safety in Patients with Pompe Disease Treated with Avalglucosidase alfa in the Real-World Setting: the SAVANT (Safety assessment of AValglucosidase Alfa as a Novel Treatment) study
IRAS ID
329250
Contact name
Derralynn Hughes
Contact email
Sponsor organisation
Sanofi-Aventis Recherche & Développement
Clinicaltrials.gov Identifier
EUPAS107472, EU PAS register number
Duration of Study in the UK
5 years, 1 months, 1 days
Research summary
Enzyme Replacement Therapy (ERT) helps breakdown glycogen stores and reduce toxic buildups of glycogen in the cells. It is administered to improve muscle tone and in patients with Pompe disease. As of March 2023, avalglucosidase alfa (Nexviazyme®/Nexviadyme®) received marketing authorisation in 13 countries worldwide for long-term enzyme replacement therapy (ERT) for the treatment of patients with Pompe disease.
This Post-Authorization Safety Study (PASS) is a non-interventional global study to collected data in patients with Pompe disease who are receiving ERT with avalglucosidase alfa (Nexviazyme®/Nexviadyme®) as per standard of care determined by their treating doctor. This study is observational therefore clinical assessments and visits will be performed as per routine clinical practice. Patients enrolled in the study will be followed for up to five years after enrolment or until participant’s study discontinuation, whichever comes first.
The purpose of this study is to collect long-term safety data and evaluate the important identified risk of infusion associated reaction (IARs) including hypersensitivity and anaphylactic reactions and medication errors in participants treated with avalglucosidase alfa at home or at hospital/clinic. The data collected as part of this study will allow to improve the understanding of the benefits and potential side effects in patients treated with avalglucosidase alfa (Nexviazyme®/Nexviadyme®) in real-world setting.
This study is sponsored by Sanofi-Aventis Recherche & Development. Approximately 100 patients will be enrolled to participate in this study worldwide including seven patients from five hospitals in the UK.
REC name
South Central - Berkshire Research Ethics Committee
REC reference
24/SC/0119
Date of REC Opinion
25 Apr 2024
REC opinion
Further Information Favourable Opinion