Recruiting

Phase 3

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

Sponsor:

Novartis Pharmaceuticals

Code:

NCT07357727

Conditions

Primary Myelofibrosis (PMF)

Post-polycythemia Vera Myelofibrosis (PPV-MF)

Post-essential Thrombocythemia Myelofibrosis (PET-MF)

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Pelabresib

Ruxolitinib

Placebo

Study Details

Brief summary:

The purpose of this trial is to evaluate whether treatment with pelabresib in combination with ruxolitinib leads to improved clinical outcomes compared to ruxolitinib alone in patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF) who have not previously received Janus kinase (JAK) inhibitor therapy.

Conditions

Primary Myelofibrosis (PMF)

Post-polycythemia Vera Myelofibrosis (PPV-MF)

Post-essential Thrombocythemia Myelofibrosis (PET-MF)

Study ID

NCT07357727

Start date

May 27, 2026

Status verified date

Oct, 2026

Completion date

Dec 27, 2030

Anticipated

Primary completion date

May 17, 2028

Anticipated

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

460 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Arm 1: Pelabresib + Ruxolitinib

Participants in this arm receive pelabresib (DAK539) orally once daily for 14 days of each 21-day cycle, in combination with ruxolitinib, which is taken orally twice daily throughout each cycle. Participants may continue receiving study treatment until they experience unacceptable toxicity, disease progression, or until either the investigator or the participant decides to discontinue treatment.

placebo comparator: Arm 2: Placebo + Ruxolitinib

Participants in this arm receive a matching placebo orally once daily for 14 days of each 21-day cycle, together with ruxolitinib, which is also taken orally twice daily throughout each cycle.

Participants may continue receiving study treatment until they experience unacceptable toxicity, disease progression, or until either the investigator or the participant decides to discontinue treatment.

Interventions

Pelabresib

Pelabresib monohydrate tablets

Ruxolitinib

Ruxolitinib phosphate tablets

Placebo

Matches pelabresib

Primary outcome measure

Number of Participants with Splenic Response (SVR35) by Central Radiology Reads at Week 24 in participants with baseline total symptom score (TSS) ≥ 25 [ Time Frame: Week 24 ]
Absolute change from baseline in total symptom score (TSS) at Week 24 in participants with baseline TSS ≥ 25 [ Time Frame: Baseline, Week 24 ]
Number of Participants with Splenic Response (SVR35) by Central Radiology Reads at Week 24 in participants with baseline TSS ≥ 15 [ Time Frame: Week 24 ]
Absolute change from baseline in total symptom score (TSS) at Week 24 in participants with baseline TSS ≥ 15 [ Time Frame: Baseline, Week 24 ]

Central Contacts and Locations

Central contacts

Novartis Pharmaceuticals

1-888-669-6682 novartis.email@novartis.com

Novartis Pharmaceuticals

+41613241111 novartis.email@novartis.com

Locations

Rocky Mountain Cancer Centers

Recruiting

Denver, Colorado, United States, 80218

Contacts

Kelly Beland

303-418-7600 Kelly.beland@usoncology.com

Principal Investigator:

Christopher Benton

Yale University School Of Medicine

Recruiting

New Haven, Connecticut, United States, 06520

Contacts

Farah Fasihuddin

farah.fasihuddin@yale.edu

Principal Investigator:

Nikolai Podoltsev

Florida Cancer Specialist South Reg

Recruiting

Fort Myers, Florida, United States, 33916

Contacts

Christina Caruso

Ccaruso@flcancer.com

Principal Investigator:

Fadi Kayali

The Anderson Family Cancer Institute

Recruiting

Jupiter, Florida, United States, 33458

Contacts

Christine Leonard

christine.leonard@jupitermed.com

Principal Investigator:

Ryan Devine

Miami NS Ins Baptist Health S FL

Recruiting

Miami, Florida, United States, 33176

Contacts

Dixie Carmona Gonzalez

786-596-2000 Dixie.CarmonaGonzalez@baptisthealth.net

Principal Investigator:

Firas El Chaer

Florida Cancer Specialists-North

Recruiting

St. Petersburg, Florida, United States, 33705

Contacts

Kelly Whitehead

kelly.whitehead@flcancer.com

Principal Investigator:

Viralkumar Bhanderi

Florida Cancer Specialists East

Recruiting

Stuart, Florida, United States, 34994

Contacts

Mary Jo Luser

MaryJo.Luser@flcancer.com

Principal Investigator:

Judah Friedman

Winship Cancer Institute of Emory University

Recruiting

Atlanta, Georgia, United States, 30322

Contacts

Danielle Alexander

danielle.oliver@emory.edu

Principal Investigator:

Anthony Michael Hunter

University of Chicago Hospital

Recruiting

Chicago, Illinois, United States, 60637

Contacts

Ellen Malone

ellen.malone@northwestern.edu

Principal Investigator:

Anand Patel

Franciscan Health Indianapolis

Recruiting

Indianapolis, Indiana, United States, 42637

Contacts

Melanie Coleman

melanie.coleman@franciscanalliance.org

Principal Investigator:

Waled Bahaj

American Oncology Partners PA Center for Cancer and Blood Disorders

Recruiting

Bethesda, Maryland, United States, 20817

Contacts

Raissa Toure

raissa.toure@aoncology.com

Principal Investigator:

Ralph Boccia

Summit Medical Group Oncology

Recruiting

Berkeley Heights, New Jersey, United States, 07922

Contacts

Michelle Mackenzie

973-436-1755 mmackenzie@summithealth.com

Principal Investigator:

Dipen Patel

New York Oncology Hematology P C

Recruiting

Albany, New York, United States, 12206

Contacts

Josephine Faruol

josephine.faruol@nyoh.com

Principal Investigator:

Lawrence Garbo

Columbia University Medical Center New York Presbyterian

Recruiting

New York, New York, United States, 10032

Contacts

Nicole Conwell

212-305-6953 nc3272@cumc.columbia.edu

Principal Investigator:

Monica Kasbekar

The Ohio State University Comprehensive Cancer Center

Recruiting

Columbus, Ohio, United States, 43221

Contacts

Hannah Davis

Hannah.Davis@osumc.edu

Principal Investigator:

Andrew Srisuwananukorn

Texas Oncology - Central South

Recruiting

Austin, Texas, United States, 78705

Contacts

Marian Heaven

marian.heaven@usoncology.com

Principal Investigator:

Jason M Melear

Texas Oncology PA Dallas Presbyterian Hospital

Recruiting

Dallas, Texas, United States, 75231

Contacts

Jessica Maner

214-265-2080 Jessica.Maner@usoncology.com

Principal Investigator:

Khoan Vu

MD Anderson Cancer Center

Recruiting

Houston, Texas, United States, 77030

Contacts

Maria Pinsoy

mbpinsoy@mdanderson.org

Principal Investigator:

Prithviraj Bose

SCRI Texas Oncology Northeast Texas

Recruiting

Tyler, Texas, United States, 75702

Contacts

Shelly Maxfield

shelly.maxfield@usoncology.com

Principal Investigator:

Shail Dalal

Fred Hutch Cancer Research

Recruiting

Seattle, Washington, United States, 98109

Contacts

Lisa Grimm

lgrimm@fredhutch.org

Principal Investigator:

Bart Scott

Jewish General Hospital

Recruiting

Montreal, Quebec, Canada, H3T 1E2

Contacts

Shireen Sirhan, Dr

514-340-8222 shireen.sirhan@mail.mcgill.ca

Principal Investigator:

Shireen Sirhan

More Information

Sponsor

Novartis Pharmaceuticals

Last update posted

Oct 22, 2026

Last verified

Oct, 2026

Keywords

Pelabresib (DAK539)
Ruxolitinib
Adult participants
Myelofibrosis (MF)

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-23. This information was provided to ClinicalTrials.gov by Novartis Pharmaceuticals on 2026-10-22. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.