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

ConditionsPrimary MyelofibrosisMyeloproliferative Neoplasm
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorPharmaEssentia

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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;

Disqualifiers

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;

Trial design

Design model

Parallel

Treatments tested in this trial

  • Ropeginterferon alfa-2b

    Biological/Vaccine

    Pre-filled Syringe. Dosage: up to 500mcg

  • Placebo

    Other intervention

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

Treatment groups

150 Participants
are divided into 2 treatment groups
Group A: Ropeginterferon alfa-2b (P1101)Experimental treatment 1 intervention
Group B: Placebo controlPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

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

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

Time frame
80 weeks
2

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

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

Time frame
80 weeks
3

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

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

Time frame
80 weeks
4

Number of Participants absence of major thrombotic events

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

Time frame
80 weeks

Secondary outcomes

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

PharmaEssentia

Lead sponsor

Source ClinicalTrials.gov
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