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PMD-026 for Myelofibrosis

This study is testing an oral drug called PMD-026 for people with myelofibrosis (a bone marrow disorder). PMD-026 is taken twice a day. To join, you must be at least 18 years old and have myelofibrosis that has been confirmed by a biopsy. You also need to have tried at least one JAK inhibitor treatment for at least 12 weeks, and your disease was resistant or didn't respond well to it. The main goals of this study are to see how safe PMD-026 is, identify any serious side effects, and find the best dose to use in future studies. The current status of this study is unclear, and it plans to enroll 18 participants.

Study design
This is a Phase Ib study. It will enroll 18 participants and is designed to find the right dose of PMD-026 and then look at how well it works.
What's involved
You would take the oral drug PMD-026 every 12 hours on an outpatient basis, every day of each 28-day cycle.
Compensation
Not stated in the trial record.
Follow-up
Participants will be monitored for adverse events from the first day of treatment through 28 days after their last dose, which is estimated to be about 1 year and 28 days.

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NCT07379125

Therapeutic RSK1 Targeting in Myelofibrosis

Recruiting
PHASE1Ages 18+InterventionalTreatment
Washington University School of Medicine
~18 participants
Updated 2026-05-22 on ClinicalTrials.gov
What's tested:PMD-026

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of participants with adverse events
Measured over From cycle 1 day 1 through 28 days after last dose (estimated to be 1 year and 28 days)
+6 more outcomes measured

Conditions

Myelofibrosis

Explore related trials

Myelofibrosis

Where it's being run

1 sites across 1 states
Missouri1

Study leadership

  • Amy W Zhou, M.D. · PRINCIPAL_INVESTIGATOR · Washington University School of Medicine

Who to contact

Amy W Zhou, M.D.
[email protected]
314-362-8814
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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

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).

What this trial measures

  • Number of participants with adverse eventsFrom cycle 1 day 1 through 28 days after last dose (estimated to be 1 year and 28 days)

    Graded per CTCAE v5.0.

  • Number of participants with dose limiting toxicities (DLTs) based on occurrence of serious treatment-emergent adverse events (Dose Escalation only)During cycle 1 of treatment (each cycle is 28 days)

    Dose limiting toxicities are defined in the protocol.

  • Recommended phase II dose (RP2D) (Dose Escalation only)Completion of cycle 1 (each cycle is 28 days) of all dose-escalation patients (estimated to be 1 year and 28 days)

    The RP2D will be determined based on review of safety and tolerability endpoints in dose escalation.

  • Changes in spleen size (Dose Expansion and RP2D Cohort in Dose Escalation)Baseline and after 24 weeks of treatment (estimated to be 24 weeks)

    Measured by ultrasound or other abdominal imaging.

  • Changes in Myelofibrosis Symptom Assessment Form (MFSAF) v4.0 Total Symptom Score (Dose Expansion and PR2D Cohort in Dose Escalation)Baseline and after 24 weeks of treatment (estimated to be 24 weeks)

    The MFSAF assesses patient's symptom burden with 7-items that are scored from 0 (Absent) to 10 (Worst Imaginable). The total score can range from 0-70 with the higher score meaning more severe symptoms.

  • Bone marrow histopathologic response (Dose Expansion and RP2D Cohort in Dose Escalation)Baseline and after 24 weeks of treatment (estimated to be 24 weeks)

    Bone marrow histopathologic response will be evaluated by the International Working Group for Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria.

  • Overall response rate (ORR) (Dose Expansion and RP2D Cohort in Dose Escalation)Baseline and after 24 weeks of treatment (estimated to be 24 weeks)

    Defined as CR (complete remission/response) + PR (partial remission/response) + CI (clinical improvement). Responses are defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) consensus.