Hemophilia B Patients Receiving Prophylaxis with SoC FIX Replacement

• Research type

  Research Study

• Full title

  A PROSPECTIVE STUDY TO EVALUATE DISEASE CHARACTERISTICS IN HEMOPHILIA B PARTICIPANTS RECEIVING PROPHYLAXIS WITH STANDARD OF CARE FIX REPLACEMENT THERAPY

• IRAS ID

  330032

• Contact name

  Gerard Dolan

• Contact email

  gerard.dolan@gstt.nhs.uk

• Sponsor organisation

  Regeneron Pharmaceuticals, Inc.

• Clinicaltrials.gov Identifier

  NCT05568459

• Duration of Study in the UK

  2 years, 8 months, 4 days

• Research summary

  This study is focused on males aged 16 to 65 who have haemophilia B.

  Haemophilia B is a type of blood clotting disorder. Normally, there are a number of coagulation factors circulating in the blood, lying in wait to be turned on when an injury occurs. If any one of the factors is missing from the body, blood clotting will not happen as it should. This can lead to blood loss, which can be severe & life-threatening.

  The specific coagulation factor that is missing or reduced in people with Haemophilia B is Factor IX. The severity of symptoms ranges from mild to severe depending on the amount of Factor IX present in the blood & its activity.

  The study will recruit participants who need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent & also to control their bleeding events. The aim of the study is to gather at least 6 months of information on bleeding events for each individual participant while they continue to use their usual FIX replacement therapy. We will then estimate the annualised bleeding event rates requiring on-demand treatment with FIX replacement therapy for each haemophilia B participant.

  There is no experimental treatment being tested in this study. The study is informational & does not affect any clinical care that the participant may be receiving. Participants will however undergo some procedures such as blood tests & a liver scan.

  The study is part of a larger program to understand & treat haemophilia B with a potential experimental new therapy in the future.

  The current standard of care leaves much desire to improve the quality of life of haemophilia B patients. This study aims to work towards new treatment leading to long-term sustained plasma levels of functional FIX protein.

  The study will include about 120 male participants worldwide. NHS sites will undertake the study in the UK.

  Participants will be in the study for at least 6 months, & possibly up to 2 years.

  Patient participation will involve:
  - Blood Tests
  - Vital Signs
  - Physical examinations including height & weight
  - FibroScan & Liver Ultrasound
  - Haemophilia Joint Health Exam
  - Electronic Diary
  - Questionnaires

• REC name

  South West - Cornwall & Plymouth Research Ethics Committee

• REC reference

  23/SW/0085

• Date of REC Opinion

  7 Sep 2023

• REC opinion

  Further Information Favourable Opinion