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

Study identification

• EU PAS number:
  EUPAS107472
• Study ID:
  107473
• Official title and acronym:
  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
• DARWIN EU® study:
  No
• Study countries:
  Belgium

  Canada

  France

  Germany

  Italy

  Netherlands

  Spain

  United Kingdom

  United States
• Study description:
  This PASS is a non-interventional, multicenter, longitudinal study of safety with prospectively collected data in participants with Pompe disease who are receiving ERT with avalglucosidase alfa as per SoC determined by their treating physician and will be followed for safety reporting using the PASS Recommended Schedule of Assessments provided in the protocol as a guidance. ; The site selection will be worldwide, with participating sites from multiple geographical regions, particularly sites participating in the Pompe Registry and in countries where home infusion is allowed per local regulation. ; The Pompe Registry (www.registrynxt.com) is a global, multi-center, multi-national, longitudinal, observational, and voluntary program that is currently conducted to track the disease history and outcomes in patients with Pompe disease
• Study status:
  Ongoing

Research institutions and networks

Institutions

Sanofi

Study timelines

Date when funding contract was signed

Planned:06/10/2023

Actual:06/10/2023

Study start date

Planned:30/06/2024

Actual:15/07/2024

Date of final study report

Planned:31/12/2029

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Sanofi

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)