Bomedemstat vs BAT for Essential Thrombocythaemia

• Research type

  Research Study

• Full title

  A Phase 3, Randomized, Open-label, Active-Comparator-Controlled Clinical Study to Evaluate the Safety and Efficacy of Bomedemstat (MK-3543/IMG-7289) versus Best Available Therapy (BAT) in Participants With Essential Thrombocythemia who have an Inadequate Response to or are Intolerant of Hydroxyurea.

• IRAS ID

  1008631

• Contact name

  - -

• Contact email

  n/a

• Sponsor organisation

  Merck Sharp & Dohme LLC

• Eudract number

  2022-002850-13

• Clinicaltrials.gov Identifier

  NCT06079879

• Research summary

  Essential Thrombocythemia (ET) is a blood disorder whereby an increased number of platelets are formed in the blood. Historically, therapy for ET was aimed at reducing the platelet count to lower the risk of bleeding and clotting events.
  Bomedemstat is a drug that slows down the process of platelet production. This trial is testing the safety and efficacy of bomedemstat (MK-3543/IMG-7289) in comparison to existing best available therapies (BAT) in people who currently have an inadequate response to or are intolerant of hydroxyurea.

  Approximately 300 participants will be randomly assigned to either receive bomedemstat (Arm A) or one of the Best Available Therapies (Arm B) at a 1:1 ratio. The choice of which specific BAT is most appropriate for each participant will be made by the research team before randomisation. The therapies offered include:
  • Anagrelide
  • Busulfan
  • Interferon alfa/pegylated interferon alfa, or
  • Ruxolitinib

  All participants must provide a bone marrow aspirate and biopsy sample for review. This may be repeated at any time point if there is suspicion of relapse or disease progression. Blood samples will need be taken for monitoring throughout as well as for genetic analysis and genomic profiling. A cheek swab will also be required for genomic profiling at the screening appointment.

  The treatment phase is initially for 52 weeks, participants who have not discontinued trial treatment by Week 52 will be eligible to continue receiving trial treatment in the Extended Treatment Phase for up to Week 156. Participants in the BAT arm who have not discontinued trial treatment after Week 52 are eligible to crossover and receive bomedemstat during the Extended Treatment Phase at the investigator’s discretion, pending eligibility and consultation.

  Participants will be followed up at the end of their treatment, 30 days after their last dose. Only participants taking busulfan will be followed up again at 180 days after their last dose.

• REC name

  London - Harrow Research Ethics Committee

• REC reference

  24/LO/0008

• Date of REC Opinion

  12 Mar 2024

• REC opinion

  Further Information Favourable Opinion