Therapeutic RSK1 Targeting in Myelofibrosis
ConditionMyelofibrosis
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorWashington University School of Medicine
About 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.
Eligibility criteria
Qualifiers
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).
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.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- PMD-026
Treatment groups
18 Participants
are divided into 4 treatment groupsSponsors and collaborators
Washington University School of Medicine
Lead sponsor
Phoenix Molecular Designs
Collaborator
The Foundation for Barnes-Jewish Hospital
Collaborator
Swim Across America
Collaborator
Source ClinicalTrials.gov