Study identification

EU PAS number

EUPAS49243

Study ID

49244

Official title and acronym

GENEr8-GTR: A Retrospective Cohort Study of Patients Treated with ROCTAVIAN™ (valoctocogene roxaparvovec): An Analysis of Patient Registries (BMN 270-801)

DARWIN EU® study

No

Study countries

Austria
Belgium
Brazil
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Finland
France
Germany
Greece
Hungary
Ireland
Italy
Latvia
Lithuania
Malta
Netherlands
Norway
Poland
Portugal
Romania
Russian Federation
Slovakia
Slovenia
Spain
Sweden
Switzerland
Türkiye
United Kingdom
United States

Study description

ROCTAVIAN™ is a commercially available gene therapy for the treatment of patients with hemophilia A (HA). As gene therapies of this type remain novel, long-term follow-up data are limited. This study is being undertaken to better characterize the long-term effectiveness and safety outcomes of patients treated with ROCTAVIAN in a real-world setting based on the safety profile outlined in the Pharmacovigilance and Risk Management Plan, based on the data collected in the World Federation of Hemophilia Gene Therapy Registry (WFH GTR) for people with HA (PwHA) administered ROCTAVIAN in a real-world setting.

Study status

Ongoing
Research institutions and networks

Institutions

Contact details

270-801 Program Director

Primary lead investigator

Study timelines

Date when funding contract was signed

Planned:

Study start date

Planned:
Actual:

Data analysis start date

Planned:
Actual:

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

BioMarin International Ltd.
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)

Regulatory procedure number

EMA/H/C/005830