Study identification

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
First published:
01/02/2024
Institution

Contact details

bluebird bio Contact

Primary lead investigator

Study timelines

Date when funding contract was signed

Planned:

Study start date

Planned:

Date of interim report, if expected

Planned:

Date of final study report

Planned:
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)