A MULTICENTER, MULTICOUNTRY, POSTMARKETING ACTIVE SURVEILLANCE TALIGLUCERASE ALFA REGISTRY IN PATIENTS WITH GAUCHER DISEASE

Study identification

• EU PAS number:
  EUPAS4721
• Study ID:
  47627
• Official title and acronym:
  A MULTICENTER, MULTICOUNTRY, POSTMARKETING ACTIVE SURVEILLANCE TALIGLUCERASE ALFA REGISTRY IN PATIENTS WITH GAUCHER DISEASE
• DARWIN EU® study:
  No
• Study countries:
  Albania

  Israel

  Türkiye

  United States
• Study description:
  To gather data on the long term safety and effectiveness of taliglucerase alfa in the real world post-marketing setting, Pfizer will conduct a prospective non-interventional active surveillance drug registry of patients with Gaucher disease undergoing taliglucerase alfa treatment (referred to as the “Drug Registry”). The registry will be open for at least ten years. The pregnancy and lactation exposure related sub-study will be nested within the Drug Registry (and is referred to as the “Pregnancy/Lactation Sub Study”). The Pregnancy/Lactation Sub-Study will be open for the maximum of 11 years (ie, will extend a maximum of 1 year beyond the end of data collection in the Drug Registry if a woman becomes pregnant during the last 9 months of the Drug Registry).
• Study status:
  Finalised

Research institutions and networks

Institutions

Parexel International

United States

Study timelines

Date when funding contract was signed

Planned:01/10/2013

Study start date

Planned:31/10/2013

Actual:18/09/2013

Date of interim report, if expected

Planned:31/07/2019

Actual:31/07/2019

Date of final study report

Planned:24/07/2024

Actual:19/04/2024

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Pfizer

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  Non-EU RMP only