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Pelabresib (DAK539) and Ruxolitinib vs. Placebo and Ruxolitinib in Adults With Myelofibrosis Who Are JAK Inhibitor Naive: A Phase 3 Randomized, Double-blind Study
Led by Novartis Pharmaceuticals · Updated on 2026-06-04
460
Participants Needed
11
Research Sites
136 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating whether the combination of pelabresib and ruxolitinib improves clinical outcomes compared to ruxolitinib alone in adults 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. This Phase 3 trial aims to assess how these treatments affect spleen size and symptom scores in patients with these myelofibrosis conditions. The study includes several periods: a screening phase lasting up to 28 days to confirm eligibility and collect baseline data; a treatment phase where participants are randomly assigned to receive either pelabresib plus ruxolitinib or placebo plus ruxolitinib in 21-day cycles. Pelabresib or placebo is taken orally once daily for 14 days each cycle, while ruxolitinib is taken twice daily continuously. Treatment continues until unacceptable side effects, disease progression, or withdrawal. After treatment ends, a 30-day safety follow-up monitors any late side effects, followed by ongoing efficacy monitoring every 12 weeks for participants without disease progression. Those with disease progression enter a survival follow-up phase with visits every 12 weeks, possibly remotely. Participants will undergo regular assessments including spleen imaging, laboratory tests, symptom scoring, and bone marrow biopsies to monitor treatment effects and disease status. Researchers will measure spleen response by imaging and changes in symptom scores at week 24 as primary outcomes, along with other measures such as survival and anemia response over approximately three years. The study uses a randomized, double-blind design and includes safety and efficacy follow-up to thoroughly evaluate the treatments' impact.
CONDITIONS
Brief Title
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults diagnosed with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF) according to 2022 International Consensus Classification
- DIPSS risk category of intermediate-1, intermediate-2, or high-risk
- Spleen volume of at least 450 cm3 by CT or MRI
- Average total symptom score (TSS) of 15 or higher within 7 days prior to randomization, using MFSAF v. 4.0
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2
- Peripheral blood blasts less than 5% at screening
- Platelet count of at least 100 x 10^9/L without growth factors or transfusions for 4 weeks prior
You will not qualify if you...
- Prior splenectomy at any time or splenic irradiation within the previous 6 months
- Prior hematopoietic cell transplant or planned transplant within 24 weeks from randomization
- Bone marrow blasts 5% or higher or history of accelerated phase or leukemic transformation
- History of other malignancies requiring systemic treatment in the past 3 years
- Treatment with any approved or investigational agent for myelofibrosis other than hydroxyurea or anagrelide within 14 days before first study dose
- Prior treatment with any JAK inhibitor or BET inhibitor
- Other protocol-defined exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - Up to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit
Duration - Continues until treatment discontinuation
Participants receive study drugs in repeated 21-day cycles, with pelabresib or placebo taken for 14 days and ruxolitinib taken continuously. Treatment continues until unacceptable toxicity, disease progression, or decision to discontinue.
Regular visits according to study schedule during treatment cycles
Duration - Approximately 1 month
Participants are monitored for any late-onset adverse events or safety concerns following the last dose of study treatment.
1 visit within 30 days after last dose
Duration - Variable, until disease progression or new therapy
Participants without disease progression are monitored every 12 weeks to assess efficacy endpoints until progression or new therapy initiation.
Visits every 12 weeks
Duration - Ongoing until study end
Participants with disease progression or new therapy are followed every 12 weeks, which may be done remotely, to monitor overall survival and disease status.
Visits every 12 weeks, possibly remote
Trial Site Locations
Total: 11 locations
1
Summit Medical Group
Berkeley Heights, New Jersey, United States, 07922
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2
Novartis Investigative Site
Clayton, Victoria, Australia, 3168
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3
Novartis Investigative Site
Tianjin, China, 300020
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4
Novartis Investigative Site
Suwon, Gyeonggi-do, South Korea, 442-723
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5
Novartis Investigative Site
Seoul, Yangcheon Gu, South Korea, 07985
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6
Novartis Investigative Site
Seoul, South Korea, 03080
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7
Novartis Investigative Site
Seoul, South Korea, 05505
Actively Recruiting
8
Novartis Investigative Site
Seoul, South Korea, 06351
Actively Recruiting
9
Novartis Investigative Site
Seoul, South Korea, 06591
Actively Recruiting
10
Novartis Investigative Site
Basel, Switzerland, 4058
Actively Recruiting
11
Novartis Investigative Site
Genolier, Switzerland, 1272
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
N
Novartis Pharmaceuticals
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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