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

NCT07357727 · tracked via the Priya Life Science India tracker

Sponsor

Novartis Pharmaceuticals

Phase

Phase 3

Started

2026-06-16

Last updated

2026-09-14

Condition(s) studied

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

Investigational drug(s) / intervention(s)

PelabresibRuxolitinibPlacebo

Pelabresib: Pelabresib monohydrate tablets

Ruxolitinib: Ruxolitinib phosphate tablets

Placebo: Matches pelabresib

Study 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.

Eligibility

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

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.

Primary outcome measure(s)

Number of Participants with Splenic Response (SVR35) by Central Radiology Reads at Week 24 in participants with baseline total symptom score (TSS) ≥ 25 — Week 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 ≥ 25 — Baseline, 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 ≥ 15 — Week 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 ≥ 15 — Baseline, 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.

Trial sites (70)

Facility	City	Region	Status
Yale University School Of Medicine	New Haven	Connecticut	Recruiting
The Anderson Family Cancer Institute	Jupiter	Florida	Recruiting
Winship Cancer Institute of Emory University	Atlanta	Georgia	Recruiting
Franciscan Health Indianapolis	Indianapolis	Indiana	Recruiting
Summit Medical Group Oncology	Berkeley Heights	New Jersey	Recruiting
New York Oncology Hematology P C	Albany	New York	Recruiting
The Ohio State University Comprehensive Cancer Center	Columbus	Ohio	Recruiting
MD Anderson Cancer Center	Houston	Texas	Recruiting
Novartis Investigative Site	Buenos Aires	Buenos Aires F.D.	Recruiting
Novartis Investigative Site	CABA	Argentina	Recruiting
Novartis Investigative Site	Capital Federal	Argentina	Recruiting
Novartis Investigative Site	Córdoba	Argentina	Recruiting
Novartis Investigative Site	Adelaide	South Australia	Recruiting
Novartis Investigative Site	Clayton	Victoria	Recruiting
Novartis Investigative Site	Hefei	Anhui	Recruiting
Novartis Investigative Site	Guangzhou	Guangdong	Recruiting
Novartis Investigative Site	Guangzhou	Guangdong	Recruiting
Novartis Investigative Site	Wuhan	Hubei	Recruiting
Novartis Investigative Site	Wuhan	Hubei	Recruiting
Novartis Investigative Site	Nanjing	Jiangsu	Recruiting
Novartis Investigative Site	Nantong	Jiangsu	Recruiting
Novartis Investigative Site	Changchun	Jilin	Recruiting
Novartis Investigative Site	Yantai	Shandong	Recruiting
Novartis Investigative Site	Kunming	Yunnan	Recruiting
Novartis Investigative Site	Hangzhou	Zhejiang	Recruiting
Novartis Investigative Site	Wenzhou	Zhejiang	Recruiting
Novartis Investigative Site	Beijing	China	Recruiting
Novartis Investigative Site	Chongqing	China	Recruiting
Novartis Investigative Site	Fuzhou	China	Recruiting
Novartis Investigative Site	Shanghai	China	Recruiting
Novartis Investigative Site	Shenyang	China	Recruiting
Novartis Investigative Site	Tianjin	China	Recruiting
Novartis Investigative Site	Pune	Maharashtra	Recruiting
Novartis Investigative Site	Rishikesh	Uttarakhand	Recruiting
Novartis Investigative Site	Kolkata	West Bengal	Recruiting
Novartis Investigative Site	Delhi	India	Recruiting
Novartis Investigative Site	Verona	VR	Recruiting
Novartis Investigative Site	Sapporo	Hokkaido	Recruiting
Novartis Investigative Site	Kobe	Hyōgo	Recruiting
Novartis Investigative Site	Shiwa-Gun	Iwate	Recruiting

+ 30 more sites — see the full list on the official registry below.

On this site

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07357727 on ClinicalTrials.gov ↗ ← All trials in India