Pelabresib plus ruxolitinib for adults with myelofibrosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 3, randomized study comparing pelabresib plus ruxolitinib to ruxolitinib with placebo in patients with primary or post‑polycythemia vera/post‑essential thrombocythemia myelofibrosis who are JAK inhibitor‑naive. Treatment is given in 21‑day cycles with pelabresib or placebo administered for 14 days and ruxolitinib given continuously, and participants are followed for safety and efficacy after treatment. Key entry requirements include intermediate‑1 to high DIPSS risk, spleen volume ≥450 cm3, total symptom score ≥15, platelet count ≥100 x109/L, blasts <5%, and ECOG 0–2. The study includes screening, an on‑treatment period until discontinuation for progression or toxicity, a 30‑day safety follow‑up, and a longer efficacy follow‑up phase.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

* Participants have diagnosis of primary myelofibrosis (PMF) or post-polycythemia vera myelofibrosis (post-PV MF) or post-essential thrombocythemia myelofibrosis (post-ET MF) according to the International Consensus Classification (ICC) of Myeloid Neoplasms and Acute Leukemias 2022
* DIPSS risk category of intermediate-1, intermediate-2 or high-risk
* Spleen volume ≥ 450 cm3 by CT or MRI scan (local read sufficient if no central read available)
* Have an average TSS of ≥15 within 7 days prior to randomization, using MFSAF v. 4.0 (at least 4 out of 7 TSS assessments required for average calculation)
* Participants with an Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2
* Blasts \<5% in peripheral blood. Assessment of blasts in peripheral blood is mandatory at screening
* Platelet count ≥ 100 x 10\^9/L in the absence of growth factors or transfusions for the previous 4 weeks

Key Exclusion Criteria:

* Prior splenectomy at any time or splenic irradiation in the previous 6 months
* Prior hematopoietic cell transplant or participant anticipated to receive a hematopoietic cell transplant within 24 weeks from the date of randomization
* Blasts ≥ 5% in bone marrow if results available at screening or history of accelerated phase (AP) or leukemic transformation
* History of a malignancy (other than MF, PPV-MF or PET-MF) in the past 3 years in need of systemic treatment
* Received any approved or investigational agent other than hydroxyurea or anagrelide for the treatment of MF within 14 days of first dose of study treatment or within 5 half-lives of the approved or investigational agent, whichever is longer
* Prior treatment with any JAK inhibitor or Bromodomain and extraterminal domain (BET) inhibitor

Other protocol-defined inclusion/exclusion criteria may apply.

Where it's running

• Rocky Mountain Cancer Centers
  Denver, Colorado, United States Recruiting
• Yale University School Of Medicine
  New Haven, Connecticut, United States Recruiting
• Florida Cancer Specialist South Reg
  Fort Myers, Florida, United States Recruiting
• The Anderson Family Cancer Institute
  Jupiter, Florida, United States Recruiting
• Miami NS Ins Baptist Health S FL
  Miami, Florida, United States Recruiting
• Florida Cancer Specialists-North
  St. Petersburg, Florida, United States Recruiting

+115 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Belumosudil vs. Ruxolitinib for hepatic cGVHD after HSCT

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.