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Phase 3 Study of Pelabresib and Ruxolitinib for Myelofibrosis

This study is looking at a new combination treatment for myelofibrosis (a bone marrow disorder). It's testing if adding pelabresib to ruxolitinib works better than ruxolitinib alone for people who haven't taken a JAK inhibitor (a type of medication) before. The study will include about 460 participants with primary myelofibrosis, or myelofibrosis that developed after polycythemia vera or essential thrombocythemia. Researchers will measure how much your spleen shrinks and how your symptoms change after 24 weeks to see if the treatment is successful. The current recruitment status is unclear.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It involves comparing pelabresib plus ruxolitinib to ruxolitinib plus a placebo (an inactive pill that looks like pelabresib).
What's involved
You would go through a screening period of up to 28 days. If eligible, you would receive study drugs in 21-day cycles, with regular visits and assessments, until you stop treatment.
Compensation
Not stated in the trial record.
Follow-up
After your last dose of pelabresib or placebo, there will be a safety follow-up period for 30 days.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07357727

A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)

Recruiting
PHASE3Ages 18+InterventionalTreatment
Novartis Pharmaceuticals
~460 participants
Updated 2026-09-17 on ClinicalTrials.gov
What's tested:PelabresibRuxolitinibPlacebo

At a glance

Recruiting sites
76 of 76 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of Participants with Splenic Response (SVR35) by Central Radiology Reads at Week 24 in participants with baseline total symptom score (TSS) ≥ 25
Measured over Week 24
+3 more outcomes measured

Conditions

Primary Myelofibrosis (PMF)
Post-polycythemia Vera Myelofibrosis (PPV-MF)
Post-essential Thrombocythemia Myelofibrosis (PET-MF)

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NCT07357727

Where you'd take part

This study runs at 76 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Franciscan Health Indianapolis

    Indianapolis, Indianastudy coordinator listed

    Recruiting

  • Fred Hutch Cancer Research

    Seattle, Washingtonstudy coordinator listed

    Recruiting

  • MD Anderson Cancer Center

    Houston, Texasstudy coordinator listed

    Recruiting

  • New York Oncology Hematology P C

    Albany, New Yorkstudy coordinator listed

    Recruiting

  • Summit Medical Group Oncology

    Berkeley Heights, New Jerseystudy coordinator listed

    Recruiting

  • The Anderson Family Cancer Institute

    Jupiter, Floridastudy coordinator listed

    Recruiting

  • The Ohio State University Comprehensive Cancer Center

    Columbus, Ohiostudy coordinator listed

    Recruiting

  • Winship Cancer Institute of Emory University

    Atlanta, Georgiastudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Novartis Pharmaceuticals · STUDY_DIRECTOR · Novartis Pharmaceuticals

Who to contact

Novartis Pharmaceuticals
[email protected]
1-888-669-6682
Email the study team

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Eligibility criteria

Inclusion

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

Exclusion

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

What this trial measures

  • Number of Participants with Splenic Response (SVR35) by Central Radiology Reads at Week 24 in participants with baseline total symptom score (TSS) ≥ 25Week 24

    Spleen Response (SVR35) is defined as achieving a reduction of at least 35 percent in spleen volume from baseline to Week 24, as measured by magnetic resonance imaging (MRI) or computed tomography (CT) scan, and assessed by a centralized radiology review in participants with baseline TSS ≥ 25.

  • Absolute change from baseline in total symptom score (TSS) at Week 24 in participants with baseline TSS ≥ 25Baseline, Week 24

    Symptom improvement at Week 24 is defined as the absolute change from baseline in the total symptom score (TSS) at Week 24, as measured by the Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0) in participants with baseline TSS ≥ 25.

  • Number of Participants with Splenic Response (SVR35) by Central Radiology Reads at Week 24 in participants with baseline TSS ≥ 15Week 24

    Spleen Response (SVR35) is defined as achieving a reduction of at least 35 percent in spleen volume from baseline to Week 24, as measured by magnetic resonance imaging (MRI) or computed tomography (CT) scan, and assessed by a centralized radiology review in participants with baseline TSS ≥ 15.

  • Absolute change from baseline in total symptom score (TSS) at Week 24 in participants with baseline TSS ≥ 15Baseline, Week 24

    Symptom improvement at Week 24 is defined as the absolute change from baseline in the total symptom score (TSS) at Week 24, as measured by the Myelofibrosis Symptom Assessment Form version 4.0 (MFSAF v4.0) in participants with baseline TSS ≥ 15.