Study identification

EU PAS number

EUPAS1000000504

Study ID

1000000504

Official title and acronym

Long-term registry-based study of patients with transfusion-dependent β-thalassemia (TDT) or sickle cell disease (SCD) treated with exagamglogene autotemcel (exa-cel)

DARWIN EU® study

No

Study countries

France
Germany
Italy
United Kingdom
United States

Study description

Long-term, prospective observational cohort study using primary and secondary data collected by established international hematopoietic stem cell transplant (HSCT) registries: the European Society for Blood and Marrow Transplantation (EBMT) Registry and the Center for International Blood and Marrow Transplant Research (CIBMTR) Registry.
The study will follow patients who received exa-cel for treatment of TDT or SCD following approval of the therapy in Germany, France, Italy, and UK (via EBMT) and US (via CIBMTR). In addition, comparator populations of patients with TDT or SCD receiving an allo-HSCT from the same countries and same transplant centers will be invited to participate. All patients will be followed for safety and effectiveness outcomes for up to 15 years after HSCT.

Study status

Planned
Research institutions and networks

Institutions

Contact details

Elizabeth Hedgeman 0000-0001-5176-5378

Primary lead investigator
ORCID number:
0000-0001-5176-5378

Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:

Data analysis 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

Vertex Pharmaceutical Incorporated
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)