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Clinical Trials in Italy / NCT07357727
Recruiting Phase 3

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

NCT07357727 · tracked via the Priya Life Science Italy tracker
Sponsor
Novartis Pharmaceuticals
Phase
Phase 3
Started
2026-05-27
Last updated
2026-10-07

Condition(s) studied

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

Investigational drug(s) / intervention(s)

Pelabresib →Ruxolitinib →Placebo

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
No
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 (119)

FacilityCityRegionStatus
Yale University School Of Medicine New Haven Connecticut Recruiting
Florida Cancer Specialist South Reg Fort Myers Florida Recruiting
The Anderson Family Cancer Institute Jupiter Florida Recruiting
Miami NS Ins Baptist Health S FL Miami Florida Recruiting
Florida Cancer Specialists-North St. Petersburg Florida Recruiting
Florida Cancer Specialists East Stuart Florida Recruiting
Winship Cancer Institute of Emory University Atlanta Georgia Recruiting
Franciscan Health Indianapolis Indianapolis Indiana Recruiting
American Oncology Partners PA Center for Cancer and Blood Disorders Bethesda Maryland 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
TxO Austin Midtown Austin Texas Recruiting
Texas Oncology PA Dallas Presbyterian Hospital Dallas Texas Recruiting
MD Anderson Cancer Center Houston Texas Recruiting
SCRI Texas Oncology Northeast Texas Tyler Texas Recruiting
Fred Hutch Cancer Research Seattle Washington 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 Graz Austria Recruiting
Novartis Investigative Site Sankt Pölten Austria Recruiting
Novartis Investigative Site Goiânia Goiás Recruiting
Novartis Investigative Site Florianópolis Santa Catarina Recruiting
Novartis Investigative Site Barretos São Paulo 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 Nanchang Jiangxi Recruiting
Novartis Investigative Site Changchun Jilin Recruiting
Novartis Investigative Site Jinan Shandong Recruiting
Novartis Investigative Site Yantai Shandong Recruiting
Novartis Investigative Site Kunming Yunnan Recruiting

+ 79 more sites — see the full list on the official registry below.

On this site

📄 Jakafi (ruxolitinib) drug profile →

More Novartis Pharmaceuticals trials in Italy

Phase 3 Study of Pelabresib (CPI-0610) in Myelofibrosis (MF) (MANIFEST-2) Active, not recruiting
Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study Recruiting
Study of PIT565 in Relapsed and/or Refractory B-cell Malignancies Active, not recruiting
A Rollover Extension Program (REP) to Evaluate the Long-term Safety and Tolerability of Open Label Iptacopan/LNP023 in Participants With Primary IgA Nephropathy Recruiting
A Phase Ib/II Open-label Study of AMO959 With Lutetium (177Lu) Vipivotide Tetraxetan (AAA617) in Combination With ARPI in Adult Participants With PSMA-positive mCRPC Recruiting

Other trials for the same condition

Extended Access of Momelotinib in Adults With Myelofibrosis Active, not recruiting
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 Italy