New pill combo aims to tame bone marrow cancer
NCT ID NCT07340138
First seen Jun 27, 2026 · Last updated Sep 10, 2026 · Updated 2 times
Summary
This early-phase study tests whether adding a new drug called pelabresib to the standard treatment ruxolitinib is safe and helpful for Japanese adults with myelofibrosis, a type of bone marrow cancer. Only 6 people will take part, and the main goal is to check for side effects and how the drug moves through the body. If results are promising, larger studies may follow.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- pelabresib (a drug taken as a pill) added to ruxolitinib (another pill)
- What this could lead to
- If it works, this could point toward a better combination treatment for myelofibrosis, potentially improving symptoms or slowing the disease.
- What could go wrong
- This is a very early, small study (only 6 people) focused on safety. It may not show clear benefit, and side effects are unknown at this stage.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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3 people
The number who actually took part.
- Started
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Apr 2026
- Expected to finish
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Sep 2030
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Key Inclusion Criteria: * Participants have diagnosis of primary myelofibrosis (PMF), post-polycythemia vera MF (Post-PV MF) or post-essential thrombocythemia MF (Post-ET MF) according to the International Consensus Classification (ICC) for Myeloid Neoplasms and Acute Leukemias 2022. * DIPSS risk category intermediate-1, intermediate-2 or high-risk at screening. * Participants currently treated with ruxolitinib monotherapy AND who are likely to benefit from the addition of pelabresib to ruxolitinib in the opinion of the investigator. * Receiving ruxolitinib at a stable dose (5 to 25 mg BID) for at least 8 weeks prior to the first dose of pelabresib. * Palpable spleen (spleen length below left costal margin \[LCM\] must be recorded) or documented splenomegaly by MRI or CT (image report must be recorded) at screening. * Platelet count ≥ 100 × 10\^9/L in the absence of growth factor support (including thrombopoietin mimetics/agonists) or platelet transfusions 4 weeks prior to the first dose of pelabresib. * Blasts \< 5% in peripheral blood. Assessment of blasts in peripheral blood is mandatory at screening. Key Exclusion Criteria: * Prior splenectomy at any time or splenic irradiation in the previous 6 months * Prior hematopoietic cell transplant or participants anticipated to receive a hematopoietic cell transplant within 24 weeks from the first dose of pelabresib. * Blasts ≥ 5% in bone marrow if results available at screening or history of accelerated phase or leukemic transformation. * History of a malignancy (other than MF, PV or ET) except for adequately treated local basal cell or squamous cell carcinoma of the skin, cervical carcinoma in situ, superficial bladder cancer, asymptomatic prostate cancer without known metastatic disease and with no requirement for therapy or requiring only hormonal therapy and with normal prostate-specific antigen for ≥ 1 year prior to start of pelabresib, adequately treated Stage 1 or 2 cancer currently in complete remission, or any other cancer that has been in complete remission for ≥ 3 years * Received any approved or investigational agent for the treatment of MF except ruxolitinib within 14 days of first dose of pelabresib or within 5 half-lives of the approved or investigational agent, whichever is longer. Other protocol-defined inclusion/exclusion criteria may apply.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Novartis Investigative Site
Kamogawa, Chiba, 2968602, Japan
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Novartis Investigative Site
Sapporo, Hokkaido, 0030006, Japan
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Novartis Investigative Site
Chūō, Yamanashi, 4093898, Japan
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a new pill tame myelofibrosis?
- Scientists hunt for immune cells that could lead to new MPN therapies
- Momelotinib access expanded for myelofibrosis patients in Long-Term safety study
- New hope for rare blood cancers: first human trial of PRT12396 begins
- Massive new study aims to unlock secrets of rare blood cancers
- New combo pill aims to shrink spleens and ease symptoms in bone marrow cancer