World Federation of Hemophilia Gene Therapy Registry (WFH GTR)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/48684
• EU PAS number:
  EUPAS48683
• Study ID:
  48684
• Official title and acronym:
  World Federation of Hemophilia Gene Therapy Registry (WFH GTR)
• DARWIN EU® study:
  No
• Study countries:
  Australia

  Austria

  Belgium

  Canada

  Denmark

  France

  Germany

  Greece

  Ireland

  Israel

  Italy

  Japan

  Netherlands

  Saudi Arabia

  Spain

  Sweden

  Switzerland

  Türkiye

  United Kingdom

  United States
• Study description:
  The primary objective of the GTR is to determine the long-term safety of factor VIII and factor IX gene therapies in PWH. Secondary objectives are to determine the long-term efficacy and the durability of factor VIII and factor IX gene therapies in PWH and to assess long-term quality of life and burden of disease post gene-therapy infusion.
• Study status:
  Planned

Research institutions and networks

Institutions

World Federation of Hemophilia (WFH)

Canada

Study timelines

Date when funding contract was signed

Actual: 28/02/2022

Study start date

Planned: 01/12/2022

Date of final study report

Planned: 30/11/2037

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Biomarin, Pfizer, CSL Behring, Spark, Takeda

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable