Ropeginterferon alfa-2b (P1101) for early or low‑risk primary myelofibrosis

A Randomized, Double-Blind, Placebo-Controlled Multicenter Phase III Study to Assess Efficacy and Safety of Ropeginterferon Alfa-2b (P1101) in Adult Patients With Pre-fibrotic/Early Primary Myelofibrosis (PMF) or Overt PMF at Low or Intermediate-1 Risk According to DIPSS Plus (HOPE-PMF): The Core Study and Its Extension Study

This trial tests whether ropeginterferon alfa-2b (P1101), given every two weeks, helps adults with early or low‑intermediate‑1 risk primary myelofibrosis.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 150 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: PharmaEssentia (Industry-sponsored)
• Locations: 1 site (Chūō, Yamanashi)
• Trial ID: NCT06468033 on ClinicalTrials.gov

What this trial studies

This is a phase 3, double‑blind, randomized (2:1) trial comparing ropeginterferon alfa‑2b (P1101) with placebo in adults who have pre‑fibrotic/early PMF or overt PMF at low to intermediate‑1 DIPSS risk. Participants receive study drug or placebo bi‑weekly and attend assessment visits about every four weeks for clinical and laboratory monitoring. The protocol includes regular blood counts, liver and kidney function tests, symptom and spleen evaluations, and safety surveillance to record hematologic and clinical outcomes. The trial aims to document response rates, tolerability, and signals of disease modification over the treatment period.

Who should consider this trial

Good fit: Adults aged 18 or older with pre‑fibrotic/early primary myelofibrosis or overt PMF at low to intermediate‑1 DIPSS risk and adequate blood counts and liver/kidney function are the intended participants.

Not a fit: Patients with advanced/high‑risk PMF, significant cytopenias, major liver or renal impairment, or who cannot tolerate interferon are unlikely to benefit from this intervention.

Why it matters

Potential benefit: If successful, the drug could improve blood counts, reduce symptoms, and potentially slow progression in patients with early or low‑risk PMF.

How similar studies have performed: Interferons including ropeginterferon have shown hematologic and molecular responses in related myeloproliferative neoplasms such as polycythemia vera, but robust phase 3 evidence specifically in PMF is limited.

Eligibility criteria

Inclusion Criteria:

1. Male or female patients aged ≥18 years at the time of signing the informed consent form;
2. 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;
3. 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;
4. Hgb ≥10.0 g/dL at screening;
5. Neutrophil count ≥1.0 × 10\^9/L at screening;
6. Creatinine clearance rate ≥30 mL/min at screening (according to the Cockcroft-Gault formula);
7. 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;
8. Written informed consent obtained from the subject and ability for the subject to comply with the requirements of the study.

Exclusion Criteria:

1. Any known contraindications to interferon α or hypersensitivity to interferon α;
2. Patients with prior interferon therapy having poor tolerability or lack of efficacy to the previous interferon therapy per investigator\'s judgement;
3. 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;
4. With severe or serious diseases that, in the Investigator's opinion, may affect the patient's participation in this study;
5. History of major organ transplantation;
6. Pregnant or breastfeeding women;
7. Patients with any other diseases that will affect the study results or may weaken the compliance to protocol per the Investigator's judgment;
8. 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.
9. Eligible for JAK inhibitor therapy at screening.

Where this trial is running

Chūō, Yamanashi

• University of Yamanashi Hospital — Chūō, Yamanashi, Japan (Recruiting)

Study contacts

• Study coordinator: Sandy Kan

Conditions Primary Myelofibrosis; Myeloproliferative Neoplasm; P1101; PMF; Ropeginterferon; PharmaEssentia; Myeloproliferative neoplasms; MPN