PRT12396 for people with high-risk polycythemia vera or myelofibrosis
A Phase 1, Open-Label, Multi-Center, Safety and Efficacy Study of PRT12396 in Participants With Polycythemia Vera and Myelofibrosis
The team will try the oral drug PRT12396 in people with high-risk polycythemia vera or myelofibrosis to find safe doses and look for early signs of benefit.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 100 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Prelude Therapeutics Industry-sponsored |
| Locations | 1 site (Grand Rapids, Michigan) |
| Trial ID | NCT07469891 on ClinicalTrials.gov |
What this trial studies
This first-in-human, open-label, multi-center Phase 1 program tests oral PRT12396 in adults with high-risk polycythemia vera or myelofibrosis, including post-PV and post-ET MF with splenomegaly and documented JAK2 V617 mutation. Part 1 uses dose escalation to define safety, tolerability, pharmacokinetics, and the maximum tolerated dose (MTD) and recommended dose(s) for expansion; Part 2 enrolls additional participants at selected doses to further characterize safety and preliminary efficacy. Approximately up to 100 participants will be enrolled across both parts, with regular clinic visits, laboratory monitoring, and imaging to track spleen size, blood counts, adverse events, and drug levels. Participants must meet organ function, performance status, contraception, and other standard eligibility requirements.
Who should consider this trial
Good fit: Ideal candidates are adults with confirmed high-risk polycythemia vera or intermediate‑1/2 or high‑risk primary myelofibrosis (including post-PV or post-ET MF) who have a JAK2 V617 mutation, ECOG 0–2, adequate organ function, and the ability to attend study visits.
Not a fit: Patients without a JAK2 V617 mutation, with uncontrolled infection, recent other malignancy, significant organ dysfunction, or those who are pregnant or unwilling to use contraception are unlikely to qualify or benefit.
Why it matters
Potential benefit: If successful, PRT12396 could provide a new oral treatment option that reduces spleen size and disease symptoms for some patients with high-risk PV or MF.
How similar studies have performed: This is a first-in-human trial of PRT12396, while other JAK-pathway targeting drugs (for example ruxolitinib) have improved symptoms and spleen size but have shown limited disease-modifying effects, so the ultimate benefit of this novel agent is unproven.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations (including contraception requirements), and other study procedures. * Confirmed diagnosis of PV or MF according to WHO 2016 or revised ICC/WHO 2022 criteria * Documented presence of a JAK2 V617 mutation * Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 * Estimate life expectancy of ≥12 weeks per investigator assessment. * Negative serum or urine pregnancy test and agree to use contraception or maintain true abstinence. * Adequate organ function and bone marrow reserves (hematology, renal, and hepatic) Exclusion Criteria: * History of another malignancy within 3 years prior to enrollment, except for malignancy considered cured with low risk of recurrence. * Clinically significant anemia due to nutritional deficiency or hemolytic disorders. * Active or uncontrolled infection requiring systemic therapy or hospitalization. * Any other medical or psychiatric conditions that, in the Investigator's judgment, would increase risk or interfere with study participation or interpretation of results. * Clinically significant or uncontrolled medical conditions, including active infection or cardiovascular disease, that would increase risk or interfere with study participation. * Unresolved toxicity \> Grade 1 from prior anticancer therapy, except for alopecia or peripheral neuropathy ≤ Grade 2. * Pregnancy or breastfeeding * Known sensitivity or contraindication to any component of study, or the excipients of study treatment. * Prior systemic therapy for PV or MF, prior or planned allogeneic hematopoietic stem-cell transplantation, recent major surgery, prior splenectomy or prior splenic irradiation, or use of hematopoietic growth factors within protocol-defined washout periods. * Use of strong or moderate cytochrome P450 (CYP) 3A4 inhibitor or inducer, sensitive CYP3A substrates with narrow therapeutic range, or acid-reducing agents that cannot be discontinued prior to study treatment. * Participation in another interventional clinical study.
Where this trial is running
Grand Rapids, Michigan
- START Midwest, LLC — Grand Rapids, Michigan, United States (Recruiting)
Study contacts
- Study coordinator: Study Contact (Please Do Not Disclose Personal Information)
- Email: clinicaltrials@preludetx.com
- Phone: See Email
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.