Study identification

PURI

https://redirect.ema.europa.eu/resource/44514

EU PAS number

EUPAS9194

Study ID

44514

Official title and acronym

A Prospective Safety Sub-Registry to Assess Anaphylaxis and Severe Allergic Reactions, and Severe Cutaneous and Systemic Immune-Mediated Reactions with Alglucosidase Alfa Treatment (Pompe Safety Sub-Registry)

DARWIN EU® study

No

Study countries

Belgium
Germany
Italy
Taiwan
United States

Study description

The objectives of this Safety Sub-Registry are to collect uniform and meaningful data on patients with Pompe disease who experience anaphylaxis, severe allergic reactions, and/or signals of severe cutaneous and/or systemic immune-mediated reactions following treatment with alglucosidase alfa. This Safety Sub-Registry also will assess: • the symptoms, severity, outcome, and occurrence of those adverse events (AEs, anaphylaxis, severe allergic reactions, and signals of severe cutaneous and systemic immune mediated reactions), • the effect of antibody responses (both timing and pattern of responses) and cross reacting immunologic material (CRIM) status (in patients with age at symptom onset less than or equal to 12 months only) on the occurrence of such AEs.

Study status

Finalised
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:
Actual:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Genzyme, a Sanofi company
Study protocol
Initial protocol

rdct- lts13930-aglu06909-amended-protocol2-PDFA

English (466.25 KB - PDF)View document
Regulatory

Was the study required by a regulatory body?

Yes

Is the study required by a Risk Management Plan (RMP)?

EU RMP category 3 (required)