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Active, Not Recruiting

NCT Number: NCT06468033

P1101 in Treating Patients With Early PMF or Overt PMF at Low or Intermediate-1 Risk

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Primary Myelofibrosis Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Myeloproliferative Disorders Myeloproliferative Neoplasm

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

University of Yamanashi Hospital, Chūō, Yamanashi, Japan

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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);
  • 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.
  • Eligible for JAK inhibitor therapy at screening.

Treatment and study plan

Ropeginterferon alfa-2b

Biological

Pre-filled Syringe.

Dosage: up to 500mcg

Other names: P1101

Placebo

Other

Placebo is a look-alike substance with the intervention (Ropeginterferon alfa-2b) that contains no active drug.

Primary outcomes

  1. Number of Participants with Platelet count equal or less (≤) 400 × 10^9/L

    Time frame: 80 weeks

    Platelet count ≤400 × 10^9/L is one of the criteria for clinically relevant complete hematologic response (CrCHR).

  2. Number of Participants with White Blood Cells (WBC) count equal or less (≤) 10 × 10^9/L

    Time frame: 80 weeks

    White Blood Cells (WBC) count ≤10 × 10^9/L is one of the criteria for clinically relevant complete hematologic response (CrCHR).

  3. Number of Participants with Hemoglobin (Hgb) equal or greater (≧) 10.0 g/dL

    Time frame: 80 weeks

    Peripheral blood: Hemoglobin (Hgb) ≧ 10.0 g/dL is one of the criteria for clinically relevant complete hematologic response (CrCHR).

  4. Number of Participants absence of major thrombotic events

    Time frame: 80 weeks

    The absence of major thrombotic events during the observation time frame is one of the criteria for clinically relevant complete hematologic response (CrCHR).

  5. Number of Participants with no progression to secondary acute myeloid leukemia (AML).

    Time frame: 80 weeks

    The absence of progression to secondary acute myeloid leukemia (AML) is one of the criteria for clinically relevant complete hematologic remission (CrCHR).

  6. Number of Participants with no progression on the Total Symptom Score (TSS)

    Time frame: 80 weeks

    The TSS score is utilized to evaluate clinical symptoms, which is based on the MFSAF Total Symptom Score (TSS) form v4.0.

    No progression is defined as:

    • The participants who still have a TSS score equal to or less than (≤) 10 If the baseline score is ≤ 10.
    • The participants with a TSS score no increase than 50% If the baseline score is greater than (>) 10.

Sponsors and collaborators

Lead sponsor

PharmaEssentia

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2027
Study completion
2029
First posted
Jun 21, 2024
Registry last updated
Jul 22, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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