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New treatment option for Myelofibrosis
Official title Therapeutic RSK1 Targeting in Myelofibrosis
ClinicalTrials.gov ID: NCT07379125
What this study is testing
What is PMD-026?
PMD-026 is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for myelofibrosis.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Histologically confirmed diagnosis of primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis in...
- 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...
- 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...
You likely can't join if
- 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 how well it works assessment of the...
- Currently receiving any other investigational agents or planning to receive any investigational agents within 28 days before the planned first dose...
- 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...
See the full eligibility 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...
- 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.
- 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 how well it works assessment of the investigational regimen. Patients with prior or concurrent malignancy that does...
- 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...
- 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).
The study team makes the final eligibility decision.
Where it's taking place
- St Louis, Missouri, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include St Louis, Missouri, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.