Clinical Trials in Japan / NCT07357727
Recruiting
Phase 3
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)
NCT07357727 · tracked via the Priya Life Science Japan tracker
Condition(s) studied
Primary Myelofibrosis (PMF)Post-polycythemia Vera Myelofibrosis (PPV-MF)Post-essential Thrombocythemia Myelofibrosis (PET-MF)
Investigational drug(s) / intervention(s)
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
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 (140)
| Facility | City | Region | Status |
|---|---|---|---|
| Rocky Mountain Cancer Centers | Denver | Colorado | Recruiting |
| 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 |
| University of Chicago Hospital | Chicago | Illinois | 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 |
| Columbia University Medical Center New York Presbyterian | New York | New York | Recruiting |
| The Ohio State University Comprehensive Cancer Center | Columbus | Ohio | Recruiting |
| Texas Oncology - Central South | 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 |
| Hospital de Clinicas Jose de San Martin | Buenos Aires | Buenos Aires F.D. | Recruiting |
| Hospital Italiano de Buenos Aires | CABA | Argentina | Recruiting |
| FUNDALEU | Capital Federal | Argentina | Recruiting |
| Hospital Privado de Cordoba | 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 | Vienna | Austria | Recruiting |
| Novartis Investigative Site | Woluwe-Saint-Lambert | Brussels Capital | Recruiting |
| Novartis Investigative Site | Goiânia | Goiás | Recruiting |
| Grupo ELORA | Florianópolis | Santa Catarina | Recruiting |
| Novartis Investigative Site | Barretos | São Paulo | Recruiting |
| Jewish General Hospital | Montreal | Quebec | 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 |
+ 100 more sites — see the full list on the official registry below.
On this site
📄 Jakafi (ruxolitinib) drug profile →More Novartis Pharmaceuticals trials in Japan
Other trials for the same condition
Study of Pelabresib add-on to Ruxolitinib in Japanese Adult Patients 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 Japan