GENEr8-COAS: A Non-Interventional, Multi-National, Longitudinal Study of Patients Treated with ROCTAVIAN™ (valoctocogene roxaparvovec) (GENEr8-COAS Observational Cohort Study)

Study identification

• EU PAS number:
  EUPAS49071
• Study ID:
  49072
• Official title and acronym:
  GENEr8-COAS: A Non-Interventional, Multi-National, Longitudinal Study of Patients Treated with ROCTAVIAN™ (valoctocogene roxaparvovec) (GENEr8-COAS Observational Cohort Study)
• DARWIN EU® study:
  No
• Study countries:
  Germany

  Italy

  United States
• Study description:
  This study is a non-interventional, multi national, longitudinal cohort study. The study will enroll patients diagnosed with HA and treated with ROCTAVIAN™. Patients will be offered the opportunity to enroll in this study only after a clinical decision has been made that they will receive ROCTAVIAN™. Only subjects who are eventually administered ROCTAVIAN™ will be included in the analysis. The study will enroll subjects until the total number of 200 planned subjects administered ROCTAVIAN™ has been reached. Data will be collected directly from subjects and health care providers as part of routine clinical practice. Where possible to avoid duplication of entry, necessary data may be extracted from relevant electronic health record systems where variables match, however, the source data will remain the initial written case history. Bleed events, FVIII levels, exogenous factor and non-factor replacement treatments, and COAs (e.g. PROs and HJHS) will be utilized to describe effectiveness. Measurements of effectiveness endpoints at or prior to, the index date (i.e. baseline data) will be utilized to quantify changes over time, as warranted. SAEs (regardless of causality or assessed relatedness), suspected ADRs of any type, and TAEs of hepatotoxicity, thromboembolic events, infusion reactions (including hypersensitivity), new malignancies, and development of FVIII inhibitors, during or after administration of ROCTAVIAN™ (index date) will be collected to describe safety endpoints. The end of the study is defined as the date on which the last subject is assessed for the purposes of the final collection of data for the primary endpoints, appropriate follow-up of safety events as required by applicable regulations and described herein notwithstanding.
• Study status:
  Planned

Research institutions and networks

Institutions

BioMarin Pharmaceuticals

Study timelines

Date when funding contract was signed

Planned: 15/12/2022

Study start date

Planned: 13/09/2023

Data analysis start date

Planned: 01/01/2027

Date of interim report, if expected

Planned: 30/06/2027

Date of final study report

Planned: 30/12/2042

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

BioMarin Pharmaceutical 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)
• Regulatory procedure number:
  EMA/H/C/005830