New pill aims to shrink spleen and ease symptoms in hard-to-treat bone marrow cancer
NCT ID NCT07379125
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase study tests an oral drug called PMD-026 in 18 adults with myelofibrosis, a type of bone marrow cancer. Participants must have already tried a JAK inhibitor without success. The main goals are to check safety, find the best dose, and see if the drug can shrink the spleen and improve symptoms.
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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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About 18 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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May 2026
- Expected to finish
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Dec 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
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.
Inclusion Criteria: * Histologically confirmed diagnosis of primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis in chronic phase, according to the 2016 WHO criteria * Patients must have had at least 1 prior JAK inhibitor treatment for a minimum of 12 weeks and their disease was determined resistant or refractory, and/or their response was lost or intolerant to treatment. * Intermediate-2 or High-risk MF, as defined by the Dynamic International Prognostic Scoring System (DIPSS). * Presence of measurable disease as defined by: * Splenomegaly defined as estimated spleen volume of ≥450 cm3 by imaging with either MRI, CT or ultrasound, or a palpable spleen \>=5 cm from the costal margin. * Baseline MFSAF v4.0 Total Symptom Score ≥ 10 * At least 18 years of age. * ECOG performance status ≤ 2. * Adequate organ function as defined below: * Total bilirubin ≤ 1.5 x IULN (unless the participant has a history of Gilbert's syndrome) * AST(SGOT)/ALT(SGPT) ≤ 3.0 x IULN * Creatinine clearance ≥ 30 mL/min by Cockcroft-Gault * Adequate laboratory parameters: * Absolute Neutrophil Count (ANC) ≥ 100/mm\^3 * Platelets ≥50,000/mm\^3 * Blasts ≤ 10% on manual differential * The effects of PMD-026 on the developing human fetus are unknown. For this reason, women of childbearing potential and men must agree to use adequate contraception prior to study entry, for the duration of study participation, and for 30 days after completion of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study or should a man suspect he has fathered a child, s/he must inform her treating physician immediately. * Ability to understand and willingness to sign an IRB approved written informed consent document. Legally authorized representatives may sign and give informed consent on behalf of study participants. Exclusion Criteria: * Prior allogeneic or autologous stem cell transplantation within the previous 12 months * Prior splenectomy * Prior splenic irradiation if \< 3 months between last radiation and screening visit. * Prior or concurrent malignancy whose natural history has the potential to interfere with the safety or efficacy assessment of the investigational regimen. Patients with prior or concurrent malignancy that does NOT meet that definition are eligible for this trial. * Currently receiving any other investigational agents or planning to receive any investigational agents within 28 days before the planned first dose of PMD-026. * Currently receiving a JAK inhibitor or planning to receive a JAK inhibitor within 7 days before the planned first dose of PMD-026. In patients with ongoing JAK inhibitor therapy (i.e. ruxolitinib) at screening, it must be tapered over a period of at least 7 days. Patients on a low dose of ruxolitinib (e.g. 5 mg QD) may have a reduced taper period or no taper. * Known active disease involving the CNS. * QTcF \>450 msec for males, \>470 msec for females (calculated using Fridericia's formula). * A history of hypersensitivity or allergic reactions attributed to compounds of similar chemical or biologic composition to PMD-026 or other agents used in the study. * Any major surgery within 28 days prior to the first dose of PMD-026. * Uncontrolled intercurrent illness including, but not limited to: ongoing or active infection, autoimmune disease, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia. * Unable to swallow or retain oral medications. * Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 35 days of study entry (repeated on C1D1).
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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.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Washington University School of Medicine
RECRUITINGSt Louis, Missouri, 63110, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Half-Matched stem cells tested as cure for myelofibrosis
- Can blood tests predict transplant complications?
- Can a new daily pill shrink the spleen and ease myelofibrosis symptoms?
- Can a new pill tame myelofibrosis?
- MRI as a window into bone marrow disease: a new biomarker test?
- Can a menin inhibitor tame myelofibrosis when standard drugs fall short?