Prospective, multinational, non-interventional post-authorisation study to document the long-term immunogenicity, safety, and efficacy of Human-cl rhFVIII (simoctocog alfa) in patients with haemophilia A treated in routine clinical practice (GENA-99)

Study identification

• EU PAS number:
  EUPAS7905
• Study ID:
  43614
• Official title and acronym:
  Prospective, multinational, non-interventional post-authorisation study to document the long-term immunogenicity, safety, and efficacy of Human-cl rhFVIII (simoctocog alfa) in patients with haemophilia A treated in routine clinical practice (GENA-99)
• DARWIN EU® study:
  No
• Study countries:
  Argentina

  Belarus

  Czechia

  Ecuador

  France

  Guatemala

  Italy

  Lithuania

  Norway

  Portugal

  Slovakia

  United Kingdom

  United States
• Study description:
  The number of patients enrolled in the pre-authorisation clinical studies with Human-cl rhFVIII was 135 (see Section 2.2.1), which is considered adequate to provide relevant information on general safety aspects and to demonstrate the efficacy of Human-cl rhFVIII in terms of its ability to restore factor VIII levels and stop or prevent bleeding. However, data from pre-authorisation studies are insufficient to estimate all aspects of therapy with FVIII products, especially with respect to immunogenicity (EMA/CHMP/BPWP/144533/2009). Therefore, to estimate the likelihood of rare side effects, such as the occurrence of FVIII inhibitors, and to bridge between the outcome from clinical trials and long-term clinical use, the purpose of this study is to assess the long-term immunogenicity, safety, and efficacy of Human-cl rhFVIII in patients with haemophilia A treated in routine clinical practice. Specifically, the study is designed to meet the requirements for post-authorisation studies as outlined in ‘Guideline on the clinical investigation of recombinant and human plasma-derived factor VIII products’ by the European Medicines Agency (EMA), EMA/CHMP/BPWP/144533/2009
• Study status:
  Finalised

Research institutions and networks

Institutions

Multiple centres: 38 centres are involved in the study

Study timelines

Date when funding contract was signed

Planned:26/06/2014

Actual:22/07/2014

Study start date

Planned:01/02/2016

Actual:21/01/2016

Date of final study report

Planned:31/03/2020

Actual:26/03/2021

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Octapharma AG

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)