PMD-026 for Myelofibrosis

Overseen By Amy W Zhou, M.D.

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Washington University School of Medicine

Must not be taking: JAK inhibitors, Investigational agents

Disqualifiers: Recent stem cell transplant, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This is a phase Ib study evaluating PMD-026, an oral inhibitor of ribosomal protein S6 kinase A1 (RSK1), in participants with myelofibrosis (MF).The dose escalation portion utilizes a standard 3+3 design to evaluate two dose levels with an additional dose de-escalation portion to identify the recommended phase II dose (RP2D); subsequently, an additional 6 patients will be enrolled in the dose expansion portion evaluating the efficacy of PMD-026.

Who Is on the Research Team?

Amy W Zhou, M.D.

Principal Investigator

Washington University School of Medicine

Are You a Good Fit for This Trial?

This trial is for people with myelofibrosis, a type of bone marrow cancer that can cause an enlarged spleen and anemia. Participants should have this condition to join the study. Specific eligibility details are not provided, but typically include factors like age, disease stage, and overall health.

Inclusion Criteria

Adequate laboratory parameters: Absolute Neutrophil Count (ANC) ≥ 100/mm^3, Platelets ≥ 50,000/mm^3, Blasts ≤ 10% on manual differential

Agree to use adequate contraception if of childbearing potential

Ability to understand and willingness to sign an IRB approved written informed consent document

Exclusion Criteria

My cancer has spread to my brain or spinal cord.

I had a stem cell transplant less than a year ago.

Receiving or planning to receive other investigational agents within 28 days before the planned first dose of PMD-026

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

Participants receive PMD-026 at varying doses to determine the recommended phase II dose

Estimated 1 year and 28 days

Monthly visits (in-person)

Dose Expansion

Participants receive PMD-026 at the recommended phase II dose to evaluate efficacy

24 weeks

Monthly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• PMD-026

Trial Overview

The trial is testing PMD-026, a pill that targets RSK1 protein in patients with myelofibrosis. It starts by giving small groups different doses to find the safest one (3+3 design), then more people try it at that dose to see how well it works.

How Is the Trial Designed?

Group I: Dose Expansion - Recommended Phase II Dose (RP2D): PMD-026 Experimental Treatment 0 Interventions

PMD-026 will be taken by mouth twice daily at the recommended phase II dose every day of each 28-day cycle.

Group II: Dose Escalation Dose Level 2: PMD-026 Experimental Treatment 1 Intervention

PMD-026 will be taken by mouth twice daily at the assigned dose every day of each 28-day cycle.

Group III: Dose Escalation Dose Level 1 (Starting Dose): PMD-026 Experimental Treatment 0 Interventions

PMD-026 will be taken by mouth twice daily at the assigned dose every day of each 28-day cycle.

Group IV: Dose Escalation Dose Level -1: PMD-026 Experimental Treatment 1 Intervention

PMD-026 will be taken by mouth twice daily at the assigned dose every day of each 28-day cycle.

Who Is Running the Clinical Trial?

Washington University School of Medicine

Lead Sponsor

Trials

2,027

Recruited

2,353,000+

Phoenix Molecular Designs

Industry Sponsor

Trials

1

Recruited

60+