Essential Thrombocythemia Clinical Trials

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A Phase 3, Randomized, Double-blind, Active-control Study of Pelabresib (DAK539) and Ruxolitinib vs. Placebo and Ruxolitinib in Adult Patients With Myelofibrosis Who Are JAK Inhibitor Naive

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• 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

Locations
Other Locations
Republic of Korea
Novartis Investigative Site
RECRUITING
Seoul
Contact Information
Primary
Novartis Pharmaceuticals
novartis.email@novartis.com
1-888-669-6682
Backup
Novartis Pharmaceuticals
novartis.email@novartis.com
+41613241111
Time Frame
Start Date: 2026-04-15
Estimated Completion Date: 2030-11-28
Participants
Target number of participants: 460
Treatments
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.
Sponsors
Leads: Novartis Pharmaceuticals

This content was sourced from clinicaltrials.gov