NCT07469891 · Prelude Therapeutics
A Phase 1 Study of PRT12396 in Participants With Select Myeloproliferative Neoplasms
What this study is about
This is a first-in-human, where both patients and doctors know the treatment given, multi-center Phase 1 study designed to evaluate the safety, tolerability, how the drug moves through the body, and preliminary effectiveness of PRT12396 in participants with high-risk polycythemia vera (PV) and myelofibrosis (MF), and to determine the maximum tolerated dose (MTD) and recommended dose(s) for expansion (RDE\[s\]). The study consists of a gradually increasing doses phase followed by a dose-expansion phase to further evaluate selected dose level(s).
View original scientific description
This is a first-in-human, open-label, multi-center Phase 1 study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of PRT12396 in participants with high-risk polycythemia vera (PV) and myelofibrosis (MF), and to determine the maximum tolerated dose (MTD) and recommended dose(s) for expansion (RDE\[s\]). The study consists of a dose-escalation phase followed by a dose-expansion phase to further evaluate selected dose level(s).
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
- Denver, Colorado
- Coral Springs, Florida
- Grand Rapids, Michigan
- Philadelphia, Pennsylvania
- Nashville, Tennessee
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 9, 2026 · Source of record for eligibility and locations