Imetelstat VS BAT in Patients with MF Refractory to JAK Inhibitor

• Research type

  Research Study

• Full title

  A Randomized Open-Label, Phase 3 Study to Evaluate Imetelstat (GRN163L) Versus Best Available Therapy (BAT) in Patients with Intermediate-2 or High-risk Myelofibrosis (MF) Relapsed/ Refractory (R/R_ to Janus Kinase (JAK)-Inhibitor

• IRAS ID

  1003434

• Contact name

  Andrew Grethlein

• Sponsor organisation

  Geron Corporation

• Eudract number

  2020-003288-24

• Clinicaltrials.gov Identifier

  NCT04576156

• Research summary

  Myelofibrosis is a type of cancer which affects bone marrow and its ability to produce normal blood cells. Aside from stem cell transplant and treatments to address the symptoms, there is only one available type of treatment for MF, Janus Kinase inhibitors (JAK inhibitors). Many patients are ineligible for stem cell transplant and some do not respond to JAK inhibitor treatment, resulting in a high unmet need for more safe and effective treatments.
  This study aims to test an experimental treatment, Imetelstat, which is thought to stop cancer cells from rapidly replicating and increasing in number by blocking a specific enzyme called telomerase. Blocking this enzyme may be an important mechanism in killing cancer cells and improving survival.
  This study will recruit patients who have not responded well to JAK inhibitor treatment and need alternative treatments. Patients will be randomly assigned to receive Imetelstat or the best available available treatment (BAT) in the UK. By comparing outcomes of treatments we aim to identify whether Imetelstat is a better treatment option for prolonging survival and treating symptoms.
  Specialist centres worldwide will run this study, recruiting ~320 patients. Patients will be tested to check if they are eligible to participate. Eligible patients will be randomly assigned to Imetelstat or BAT. Patients will receive treatment until their condition worsens or they have side effects. During treatment patients will have tests and scans to monitor their health. If a patient’s condition worsens while on the BAT arm, they may be eligible to cross over to receive Imetelstat. All patients will be monitored after stopping treatment to ensure their wellbeing and to follow for survival even after they may have started a new treatment until death, withdrawal of consent or loss to follow up. Duration of participation will depend on survival and/or time until withdrawal.

• REC name

  East of Scotland Research Ethics Service REC 2

• REC reference

  21/ES/0017

• Date of REC Opinion

  8 Mar 2021

• REC opinion

  Further Information Favourable Opinion