Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07357727 | A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF) | PHASE3 | RECRUITING | 460 | — | — | May 27, 2026 | Dec 27, 2030 | Jun 4, 2026 | 11 | United States, Australia +3 |
| NCT07340138 | Study of Pelabresib add-on to Ruxolitinib in Japanese Adult Patients With Myelofibrosis | PHASE1 | RECRUITING | 6 | — | — | Apr 15, 2026 | Dec 18, 2030 | Jun 2, 2026 | 7 | Japan |
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.
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.
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.
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.
A dose-limiting toxicity (DLT) is defined as any adverse event or abnormal laboratory finding that is not attributable to the underlying disease, disease progression, intercurrent illness or injury, or concomitant medications, occurring within the first 21 days of pelabresib treatment and meeting the protocol-specified criteria. Adverse events will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), version 5.0. For Japanese safety confirmation of the 125 mg QD dose, DLTs will be included in the decision-making process.
The analysis of adverse events will include categorization by type, frequency, and severity, as graded by the NCI CTCAE version 5.0.
| Arm | Type | Description |
|---|---|---|
| Arm 1: Pelabresib + Ruxolitinib | EXPERIMENTAL | 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. |
| Arm 2: Placebo + Ruxolitinib | PLACEBO_COMPARATOR | 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. |
| Pelabresib + Ruxolitinib | EXPERIMENTAL | Eligible participants will receive pelabresib 125 mg once daily (QD) in combination with ruxolitinib at doses ranging from 5 to 25 mg twice daily (BID). |
| Name | Type | Description |
|---|---|---|
| Pelabresib | DRUG | Pelabresib monohydrate tablets |
| Ruxolitinib | DRUG | Ruxolitinib phosphate tablets |
| Placebo | DRUG | Matches pelabresib |
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 Leukemi...