M16-191 Front-line study of Navitoclax for Myelofibrosis

• Research type

  Research Study

• Full title

  A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of Navitoclax in Combination with Ruxolitinib Versus Ruxolitinib in Subjects with Myelofibrosis (TRANSFORM-1)

• IRAS ID

  287832

• Contact name

  Donal McLornan

• Contact email

  Donal.McLornan@gstt.nhs.uk

• Sponsor organisation

  AbbVie Ltd

• Eudract number

  2020-000097-15

• Clinicaltrials.gov Identifier

  NCT04472598

• Duration of Study in the UK

  7 years, 7 months, 26 days

• Research summary

  Myelofibrosis (MF) is a bone marrow illness that affects blood-forming tissues in the body. MF disturbs the body's normal production of blood cells, causing extensive scarring in the bone marrow. This leads to severe anemia, weakness, fatigue, and an enlarged spleen. The purpose of this study is to assess safety, tolerability, and change in spleen volume when navitoclax is given in combination with ruxolitinib, as compared to best available therapy, for adult participants with MF.

  Navitoclax is an investigational drug (not yet approved) being developed for the treatment of MF. The study has 2 arms - A and B. In Arm A, participants will receive navitoclax in combination with ruxolitinib. In Arm B, participants will receive
  ruxolitinib and a placebo for navitoclax. Adult participants with a diagnosis of MF who have not been previously treated with a standard treatment such a JAK-2 inhibitor or similar medication will be enrolled. Approximately 230 participants will be enrolled in the study at approximately 17 countries worldwide. In Arm A, participants will receive oral navitoclax tablet once daily with oral ruxolitinib tablet twice daily. In Arm B, participants will receive oral ruxolitinib tablet twice daily with a placebo once daily. Treatment will continue until clinical benefit is not seen, participants cannot tolerate the study drugs, or participants withdraw consent. The approximate treatment duration is about 2 years.

  There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of treatment will be checked by medical
  assessments, blood and bone marrow tests, checking for side effects, and completing questionnaires.

• REC name

  North East - Tyne & Wear South Research Ethics Committee

• REC reference

  20/NE/0249

• Date of REC Opinion

  20 Nov 2020

• REC opinion

  Further Information Favourable Opinion