Actively Recruiting
A Phase 1b Study of Pelabresib (DAK539) Added to Stable Dose of Ruxolitinib in Japanese Adults With Myelofibrosis
Led by Novartis Pharmaceuticals · Updated on 2026-06-02
6
Participants Needed
7
Research Sites
213 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, pharmacokinetics, and early effects of pelabresib added to ruxolitinib in Japanese adults with myelofibrosis. This Phase 1b, multicenter, open-label study focuses on patients diagnosed with primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis, with intermediate to high risk levels. Participants will first go through a screening period of up to 28 days. Those eligible will receive pelabresib 125 mg once daily alongside a stable dose of ruxolitinib (5 to 25 mg twice daily). Pelabresib treatment continues until disease progression, unacceptable toxicity, death, or decisions by the participant or investigator. After stopping pelabresib, a 30-day safety follow-up is conducted, followed by long-term follow-up visits approximately every 12 weeks for at least three years or two years after the last dose, whichever is longer. During the study, participants will have regular safety assessments including monitoring for leukemic transformation. Researchers will evaluate dose-limiting toxicities within 21 days and track adverse events throughout the roughly four-year study. Pharmacokinetics of pelabresib will be measured during the first treatment cycle. Changes in spleen size and symptom scores will be assessed up to 48 weeks. Participation may last several years with ongoing safety and efficacy monitoring.
CONDITIONS
Brief Title
Study of Pelabresib add-on to Ruxolitinib in Japanese Adult Patients With Myelofibrosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 18 years or older
- Diagnosis of primary myelofibrosis, post-polycythemia vera MF, or post-essential thrombocythemia MF according to 2022 International Consensus Classification
- DIPSS risk category intermediate-1, intermediate-2, or high-risk at screening
- Currently treated with ruxolitinib monotherapy at a stable dose of 5 to 25 mg twice daily for at least 8 weeks prior to pelabresib start
- Palpable spleen or documented splenomegaly by MRI or CT at screening
- Platelet count of at least 100 x 10^9/L without growth factor support or platelet transfusions within 4 weeks prior to pelabresib
- Less than 5% blasts in peripheral blood at screening
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 of pelabresib start
- 5% or more blasts in bone marrow if available at screening or history of accelerated phase or leukemic transformation
- History of malignancy other than MF, PV, or ET except certain treated cancers in remission
- Use of any approved or investigational MF treatment other than ruxolitinib within 14 days or 5 half-lives prior to pelabresib
- 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 28 days
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Until disease progression, unacceptable toxicity, death, participant decision, or investigator decision
Participants receive pelabresib 125 mg orally once daily on Days 1-14 of each 21-day cycle in addition to a stable dose of ruxolitinib twice daily. Treatment continues until disease progression, unacceptable toxicity, death, participant decision, or investigator decision.
Repeated 21-day cycles with pelabresib dosing on Days 1-14 of each cycle
Duration - At least 3 years from first dose and at least 2 years after last dose
After treatment ends, participants have a 30-day safety follow-up visit and then long-term follow-up visits approximately every 12 weeks for at least three years from first dose and at least two years after last dose to monitor safety and disease status.
Safety follow-up visit 30 days after last dose and long-term follow-up visits approximately every 12 weeks
Trial Site Locations
Total: 7 locations
1
Novartis Investigative Site
Kamogawa, Chiba, Japan, 296-8602
Actively Recruiting
2
Novartis Investigative Site
Sapporo, Hokkaido, Japan, 0030006
Actively Recruiting
3
Novartis Investigative Site
Kamakura, Kanagawa, Japan, 247-8533
Actively Recruiting
4
Novartis Investigative Site
Kurashiki, Okayama-ken, Japan, 7108602
Actively Recruiting
5
Novartis Investigative Site
Bunkyo Ku, Tokyo, Japan, 1138431
Actively Recruiting
6
Novartis Investigative Site
Chūō, Yamanashi, Japan, 409-3898
Actively Recruiting
7
Novartis Investigative Site
Kumamoto, Japan, 862-8655
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
N
Novartis Pharmaceuticals
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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