Phase 3 Study of CPI-0610 in Myelofibrosis (Amendment 1, UK V2.1)

  • Research type

    Research Study

  • Full title

    A Phase 3, Randomized, Double-blind, Active-Control Study of CPI-0610 and Ruxolitinib vs. Placebo and Ruxolitinib in JAKi Treatment Naive MF Patients

  • IRAS ID

    290367

  • Contact name

    Claire Harrison

  • Contact email

    Claire.Harrison@gstt.nhs.uk

  • Sponsor organisation

    Constellation Pharmaceuticals Inc.

  • Eudract number

    2020-001989-10

  • Clinicaltrials.gov Identifier

    NCT04603495

  • Duration of Study in the UK

    2 years, 8 months, 31 days

  • Research summary

    This study is being carried out on patients with myelofibrosis. When patients have myelofibrosis, the bone marrow is not able to make enough blood cells. The spleen in the belly is bigger than normal making the belly feel very full. Patients may also have fevers and night sweats. This study is being carried out to find out if a study medication called CPI-0610 can help decrease the spleen size.

    This Phase 3 study is intended to confirm the splenic and TSS responses observed in Phase 2 while also evaluating disease-modifying effects of CPI-0610, such as improvement in bone marrow fibrosis and other clinically relevant endpoints.

    There are two different treatment groups or arms in this study. One arm is called the ‘Experimental Arm’, those getting CPI-0610 and Ruxolitinib. The second arm is the ‘Control Arm’, those getting Ruxolitinib and Placebo.

    Following a screening period of up to 28 days, eligible patients will be randomised in a 1:1 ratio to one of two treatment groups: 1) CPI-0610 + ruxolitinib (experimental group) or 2) placebo + ruxolitinib (control group). Patients will be assessed for the primary and key secondary endpoints, splenic and total symptom score (TSS) response, respectively, at Week 24. After 24 weeks, patients in the control group who have progressive disease by radiological parameters (increase in splenomegaly) may be treated with CPI-0610 + ruxolitinib provided that they remain eligible for the study.

  • REC name

    London - Brent Research Ethics Committee

  • REC reference

    21/LO/0177

  • Date of REC Opinion

    8 Apr 2021

  • REC opinion

    Further Information Favourable Opinion