A Non-Interventional Registry Study of Patients with β-thalassemia to Characterise and Contextualise the Safety and Effectiveness of Betibeglogene Autotemcel

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/46901
• EU PAS number:
  EUPAS41950
• Study ID:
  46901
• Official title and acronym:
  A Non-Interventional Registry Study of Patients with β-thalassemia to Characterise and Contextualise the Safety and Effectiveness of Betibeglogene Autotemcel
• DARWIN EU® study:
  No
• Study countries:
  Austria

  Belgium

  Bulgaria

  Croatia

  Cyprus

  Czechia

  Denmark

  Estonia

  Finland

  France

  Germany

  Greece

  Hungary

  Iceland

  Ireland

  Italy

  Latvia

  Lithuania

  Luxembourg

  Malta

  Netherlands

  Norway

  Poland

  Portugal

  Romania

  Slovakia

  Slovenia

  Spain

  Sweden

  Switzerland

  United Kingdom

  United States
• Study description:
  This study has been cancelled. The European Commission withdrew the marketing authorisation for Zynteglo (betibeglogene autotemcel) in the European Union (EU). The withdrawal was at the request of the marketing authorisation holder, bluebird bio (Netherlands) B.V., which notified the European Commission of its decision to no longer market the product in the EU for commercial reasons. The withdrawal occurred before any patients were enrolled in REG-504 and, as this study was only planned to be conducted in the EU, it will no longer be conducted. REG-504 was intended to be a non-interventional, descriptive cohort study involving secondary use of product registry and transplant registry data to evaluate endpoints related to the safety and effectiveness of betibeglogene autotemcel and allogeneic-hematopoietic stem cell transplant (allo-HSCT). The study was intended to include two cohorts of patients, a betibeglogene autotemcel cohort and an allo-HSCT cohort.
• Study status:
  Planned

Research institutions and networks

Institutions

Bluebird bio

Study timelines

Date when funding contract was signed

Planned: 31/08/2021

Study start date

Planned: 30/09/2021

Date of interim report, if expected

Planned: 31/03/2022

Date of final study report

Planned: 31/12/2042

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

bluebird bio, Inc.

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)