Beyond-9

• Research type

  Research Study

• Full title

  A Two-Part Open-Label Study of REGV131 LNP1265, a CRISPR/Cas9-Based Coagulation Factor IX Gene Insertion Therapy in Participants with Hemophilia B (BEYOND-9)

• IRAS ID

  1009750

• Contact name

  Lynsey Kennedy

• Contact email

  irasuk@iconplc.com

• Sponsor organisation

  Regeneron Pharmaceuticals Inc.

• Research summary

  Haemophilia B is a type of blood clotting disorder causing people to bleed longer than others due to a genetic disorder.
  The current main medication to treat haemophilia B is concentrated FIX product, called clotting factor or simply, factor. Patients with severe haemophilia may require frequent treatment to maintain enough clotting factor in their bloodstream to prevent bleeds. These factor therapies are infused intravenously.
  REGV131-LNP1265, a new investigational drug, is made up of 2 drug components to be administered one after the other:
  - REGV131
  - LNP1265
  Both drug components work together to create a new working Factor 9 gene in patients DNA. Clotting factor should begin to be made by liver cells about a week later. The intent is that this therapy can be a lifelong treatment for haemophilia B, enabling the body to permanently produce functional clotting factor by giving it the genetic instructions to do so.
  Pre-screening will occur where a single blood test will be taken. This test has been developed to measure possible antibodies to a manufactured virus called adeno-associated virus (AAV) and this will determine whether potential participants can take part in the screening phase. This blood test is an investigational in vitro diagnostic device.
  This study will enrol male participants with severe & moderately severe haemophilia B, aged 18+ years. The study duration ranges from approx. 2 years, 1 month - 4 years, 2 months, depending on the length of time in the lead-in period.
  NHS sites will undertake the trial in the UK. Some of these sites also took part in the Lead in study: R0000-HEMB-2187.
  Enrolled patients will undergo:
  - Blood Tests
  - Questionnaires
  - Intravenous Infusions
  - Sampling bodily fluids
  - Electrocardiograms (ECG)
  - Vital Signs
  - Physical Examination
  - Fibroscan & liver ultrasound
  - Haemophilia joint health exam
  - Electronic diary will be used to record bleeding events
  - Questionnaires

• REC name

  North East - York Research Ethics Committee

• REC reference

  24/NE/0055

• Date of REC Opinion

  22 May 2024

• REC opinion

  Further Information Favourable Opinion