A prospective multicenter observational post authorization safety sub-registry to characterize the long-term safety profile of commercial use of eliglustat (Cerdelga®) in adult patients with Gaucher disease (ELISAFE)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/48611
• EU PAS number:
  EUPAS11998
• Study ID:
  48611
• Official title and acronym:
  A prospective multicenter observational post authorization safety sub-registry to characterize the long-term safety profile of commercial use of eliglustat (Cerdelga®) in adult patients with Gaucher disease (ELISAFE)
• DARWIN EU® study:
  No
• Study countries:
  Belgium

  Denmark

  Greece

  Italy

  Portugal

  Romania

  Spain

  United Kingdom
• Study description:
  This is a non-interventional, multicenter, prospective post-authorization safety study of Cerdelga. The aim of the study is to collect long term safety data from the International Collaborative Gaucher Group (ICGG) Gaucher Registry patients treated with Cerdelga and Cerezyme, and to describe the utilization of Cerdelga. The study will enroll patients who will be receiving Cerdelga or Cerezyme for the first time or as part of ongoing treatment of their Gaucher disease (GD). Since this is a sub-registry of the ICGG Gaucher Registry, Health Care Providers (HCPs) for the sub-registry will be chosen among the enrolling sites within the ICGG Gaucher Registry and in countries where Cerdelga will be available on the market. Inclusion criteria for the study are very wide to enable HCP’s to enroll as many adult patients treated or to be treated with Cerdelga or Cerezyme as possible in order to collect data in as many patients as possible. The exclusion criteria are limited only to patients currently enrolled in another clinical trial and patients treated with Cerezyme and Cerdelga in combination at the time of enrolment. At least 100 patients enrolled will be on Cerdelga. Patients will receive treatment as determined by the patient’s HCP and guided by the provisions of the prevailing locally approved product labelling and the ICGG Gaucher Registry recommendations for patient monitoring. This observational study will operate under real-world clinical practice conditions. There are no imposed protocol visits or procedures. It will collect only available data at the time of patient visits occurring as per local practice visit schedule or following the recommended schedule of assessments of the ICGG Gaucher Registry. There is no set duration for this study. Data collection will be concluded four years after the last Cerdelga patient has been enrolled in the study at which time analysis will be performed. Information will be collected in case report forms (CRFs).
• Study status:
  Ongoing

Research institutions and networks

Institutions

Sanofi

Study timelines

Date when funding contract was signed

Planned: 03/10/2016

Actual: 25/07/2017

Study start date

Planned: 09/01/2018

Actual: 18/04/2018

Date of interim report, if expected

Actual: 03/07/2023

Date of final study report

Planned: 03/07/2025

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Sanofi Genzyme

Study protocol

Initial protocol

OBS14099 protocol_PASS_PRAC03_23Aug2016

Updated protocol

rdct-obs14099-16-1-1-pass-amended-protocol04-pdfa

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 1 (imposed as condition of marketing authorisation)

Other study registration identification numbers and links

OBS14099