Study identification

PURI

https://redirect.ema.europa.eu/resource/107473

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
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
First published:
01/02/2024
Institution

Contact details

Trial Transparency Team Trial Transparency Team

Primary lead investigator
Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:
Actual:

Date of final study report

Planned:
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)