A prospective non-interventional post-authorization safety study (PASS), designed as a disease registry of patients with transfusion dependent IPSS low or intermediate-1-risk myelodysplastic syndromes (MDS) and isolated del(5q) (CC-5013-MDS-010)

Study identification

• EU PAS number:
  EUPAS7412
• Study ID:
  46350
• Official title and acronym:
  A prospective non-interventional post-authorization safety study (PASS), designed as a disease registry of patients with transfusion dependent IPSS low or intermediate-1-risk myelodysplastic syndromes (MDS) and isolated del(5q) (CC-5013-MDS-010)
• DARWIN EU® study:
  No
• Study countries:
  Belgium

  Denmark

  France

  Germany

  Greece

  Italy

  Luxembourg

  Netherlands

  Norway

  Spain

  Sweden

  United Kingdom
• Study description:
  This is a prospective non-interventional post-authorization safety study (PASS), designed as a disease registry. No deviation from the routine clinical practice of enrolled patients is expected as a result of this study. Patients with transfusion-dependent IPSS low or intermediate-1 myelodysplastic syndromes (MDS) and isolated del(5q) who meet the inclusion/exclusion criteria will be eligible for enrollment. This study will be conducted in countries in the EU, where it is expected that lenalidomide will be marketed in the MDS indication. Details of implementation of this condition to the marketing authorization will be agreed with each NCA in the Member States where the registry is planned. Exposure studies with uniform data collection procedures for antecedent and outcome variables can provide robust benefit/risk information through the enrollment of large numbers of patients with an uncommon condition. The enrollment will not be limited to patients receiving lenalidomide within the indication approved in the EU but will aim to include additional MDS patients receiving treatments or treatment modalities other than lenalidomide. Primary endpoints for this study include product-limit estimators of AML progression and survival for all MDS patients in the MDS registry and separately for patients in the primary population and the non-primary populations. Hazard ratios derived from Cox proportional hazards models will quantify the magnitude of risk associated with lenalidomide treatment, other risk factors, and any interaction effects derived from data obtained from patients in the primary population. Measures of effectiveness among patients in the primary population will be described as the proportion of patients who achieve the effectiveness endpoints (i.e. erythroid response, transfusion independence, and cytogenetic response).
• Study status:
  Finalised

Research institutions and networks

Institutions

Celgene International

Multiple centres: 162 centres are involved in the study

Study timelines

Date when funding contract was signed

Actual: 01/12/2013

Study start date

Planned: 01/12/2014

Actual: 17/12/2014

Date of final study report

Planned: 31/03/2023

Actual: 21/03/2023

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Celgene International Sarl

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)