M16-109 Phase 2 Single Arm Study on Patients with Myelofibrosis

  • Research type

    Research Study

  • Full title

    A Phase 2 Single-Arm, Open-Label Study Evaluating Tolerability and Efficacy of Navitoclax in Combination with Ruxolitinib in Subjects with Myelofibrosis

  • IRAS ID

    229938

  • Contact name

    Claire Harrison

  • Contact email

    Claire.Harrison@gstt.nhs.uk

  • Sponsor organisation

    AbbVie Ltd

  • Eudract number

    2017-001398-17

  • Duration of Study in the UK

    2 years, 5 months, 9 days

  • Research summary

    Myelofibrosis (MF) is an uncommon type of illness that affects the blood-forming tissues in the body. MF is a serious bone marrow illness that disrupts the body's normal production of blood cells. It results in extensive scarring in the bone marrow, leading to severe anaemia, weakness, fatigue and an enlarged spleen.

    Many people with myelofibrosis get progressively worse, and some may develop a form of leukaemia.

    An accepted treatment for MF is a drug called Ruxolitinib that helps with symptoms. However when ruxolitinib is stopped, the patient develops the MF symptoms again. The only treatment that currently controls all the symptoms of MF is a stem cell transplant but this treatment is not suitable for most patients; therefore there is a real need to develop new strategies including trying combinations of treatments.

    Navitoclax is an experimental drug that kills cancer cells by blocking a protein that allows cancer cells to stay alive. It has been shown to have an effect on cells that cause MF

    This study is in patients with MF who are currently receiving ruxolitinb and becoming symptomatic and is designed to see if adding navitoclax to ruxolitinib works better than ruxolitinb on its own. All the patients will receive both treatments. AbbVie is funding this study which will enrol about 34 patients in 15 sites in the USA and the UK.

    The effect of the treatment on MF will be checked by taking MRI measurements of spleen size, blood and bone marrow samples, measuring side effects and by completing symptom questionnaires. Patients will continue to have study visits and receive treatment for as long as their improvements are greater than their side effects.

  • REC name

    South Central - Berkshire B Research Ethics Committee

  • REC reference

    17/SC/0493

  • Date of REC Opinion

    4 Oct 2017

  • REC opinion

    Further Information Favourable Opinion