P1101 in Treating Patients With Early PMF or Overt PMF at Low or Intermediate-1 Risk (NCT06468033) | Clinical Trial Compass
RecruitingPhase 3
P1101 in Treating Patients With Early PMF or Overt PMF at Low or Intermediate-1 Risk
Japan150 participantsStarted 2025-07-18
Plain-language summary
This is a phase 3 double-blind clinical trial arm to test Ropeginterferon alfa-2b (P1101) in adult patients with Primary Myelofibrosis (PMF) at early stage or low to medium risk.
Participants will receive the study drug/placebo bi-weekly and have an assessment visit every 4 weeks. The ratio of study drug to placebo group is 2:1.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Male or female patients aged ≥18 years at the time of signing the informed consent form;
. Patients with pre-fibrotic/early PMF (Pre-PMF) or overt primary myelofibrosis at low to intermediate-1 risk according to DIPSS plus, diagnosed according to WHO 2016 or 2022 classification;
. With good liver function at screening, which is defined as total bilirubin ≤1.5 × upper limit of normal (ULN), international normalized ratio (INR) ≤1.5 × ULN, albumin \>3.5 g/dL, alanine aminotransferase (ALT) ≤2.0 × ULN, and aspartate aminotransferase (AST) ≤2.0 × ULN;
. Hgb ≥10.0 g/dL at screening;
. Neutrophil count ≥1.0 × 10\^9/L at screening;
. Creatinine clearance rate ≥30 mL/min at screening (according to the Cockcroft-Gault formula);
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants with Platelet count equal or less (≤) 400 × 10^9/L
Timeframe: 80 weeks
2
Number of Participants with White Blood Cells (WBC) count equal or less (≤) 10 × 10^9/L
Timeframe: 80 weeks
3
Number of Participants with Hemoglobin (Hgb) equal or greater (≧) 10.0 g/dL
Timeframe: 80 weeks
4
Number of Participants absence of major thrombotic events
Timeframe: 80 weeks
5
Number of Participants with no progression to secondary acute myeloid leukemia (AML).
Timeframe: 80 weeks
6
Number of Participants with no progression on the Total Symptom Score (TSS)
. Females of childbearing potential, as well as all women \<2 years after the onset of menopause, must agree to use an acceptable form of birth control until 60 days following the last dose of the study drug, and females must agree to not breastfeed during the study;
. Written informed consent obtained from the subject and ability for the subject to comply with the requirements of the study.
Exclusion criteria
. Any known contraindications to interferon α or hypersensitivity to interferon α;
. Patients with prior interferon therapy having poor tolerability or lack of efficacy to the previous interferon therapy per investigator\'s judgement;
. Patients with an ongoing cytoreduction (e.g., HU or IFN-α) at the time of screening if, in the Investigator's opinion, randomizing them into the placebo arm will lead to immediate rebound increase of peripheral blood counts and thus may jeopardize their health status;
. With severe or serious diseases that, in the Investigator's opinion, may affect the patient's participation in this study;
. History of major organ transplantation;
. Pregnant or breastfeeding women;
. Patients with any other diseases that will affect the study results or may weaken the compliance to protocol per the Investigator's judgment;
. Use any investigational drug \<4 weeks prior to the first dose of study drug, or not recovered from effects of prior administration of any investigational drug.