Actively Recruiting
A Phase 1, Open-Label, Multi-Center, Safety and Efficacy Study of PRT12396 in Participants With Polycythemia Vera and Myelofibrosis
Led by Prelude Therapeutics · Updated on 2026-05-29
100
Participants Needed
3
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the safety, tolerability, how the body processes it, and early signs of effectiveness of an investigational drug called PRT12396 in people with high-risk polycythemia vera (PV) and myelofibrosis (MF). These conditions are types of myeloproliferative neoplasms that affect blood cell production. The study aims to find the highest dose that can be tolerated and to recommend doses for further testing. It is a first-in-human, open-label Phase 1 trial conducted at multiple centers. The trial has two parts: first, a dose-escalation phase where participants receive increasing oral doses of PRT12396 twice daily to assess safety and determine the recommended dose. Second, a dose-expansion phase enrolls more participants to further evaluate the selected dose's safety, tolerability, and preliminary effects. PRT12396 capsules are taken by mouth with water, either an hour before or two hours after meals. Participants will attend scheduled visits and undergo laboratory tests to monitor their health and response to treatment. The research team will assess dose-limiting toxicities, adverse events, and various blood-related measures such as hematologic response and symptom scores over about two years. The study plans to enroll up to 100 participants and includes careful monitoring of safety and drug levels in the body throughout the trial period.
CONDITIONS
Brief Title
A Phase 1 Study of PRT12396 in Participants With Select Myeloproliferative Neoplasms
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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 6512 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)
You will not qualify if you...
- 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 64 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.
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Repeated 28-day cycles until disease progression or discontinuation
Participants with polycythemia vera or myelofibrosis receive PRT12396, an investigational oral capsule administered twice daily at the assigned dose level or recommended dose for expansion. Treatment includes a dose escalation phase followed by a dose expansion phase to evaluate safety, tolerability, and preliminary efficacy.
Weekly visits for up to 4 weeks during dose escalation; visit frequency may vary during dose expansion
Duration - Up to 2 years
Participants are monitored for safety, treatment response, and adverse events after completing treatment with PRT12396, with assessments continuing for an average of 2 years.
Regular follow-up visits scheduled over 2 years
Trial Site Locations
Total: 3 locations
1
BRCR Global - Coral Springs
Coral Springs, Florida, United States, 33065
Actively Recruiting
2
START Midwest, LLC
Grand Rapids, Michigan, United States, 49546
Actively Recruiting
3
Tristar BMT
Nashville, Tennessee, United States, 37203
Actively Recruiting
Research Team
S
Study Contact (Please Do Not Disclose Personal Information)
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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