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

NCT ID: NCT06468033

Last Updated: 2025-08-13

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 150 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-07-18
• Study Completion Date: 2028-09-30

Brief 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.

Conditions

Primary Myelofibrosis; Myeloproliferative Neoplasm

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Ropeginterferon alfa-2b (P1101)

Ropeginterferon alfa-2b (P1101) is administrated subcutaneously (SC) every two weeks (± 3 days) until 80 weeks.

Group Type: EXPERIMENTAL

Ropeginterferon alfa-2b

Intervention Type: BIOLOGICAL

Pre-filled Syringe.

Dosage: up to 500mcg

Placebo control

Placebo is administrated subcutaneously (SC) every two weeks (± 3 days) until 80 weeks.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

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

Interventions

Ropeginterferon alfa-2b

Pre-filled Syringe.

Dosage: up to 500mcg

Intervention Type: BIOLOGICAL

Placebo

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

Intervention Type: OTHER

Other Intervention Names

P1101

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

PharmaEssentia

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Toshiaki Sato, MD/PhD

Role: STUDY_DIRECTOR

PharmaEssentia JP

Locations

University of Yamanashi Hospital

Chūō, Yamanashi, Japan

Site Status: RECRUITING

Countries

Japan

Central Contacts

Sandy Kan

Role: CONTACT

sandy_kan@pharmaessentia.com

+886-2-26557688 ext. 7855

Aypiin Shen

Role: CONTACT

aypiin_shen@pharmaessentia.com

+886-2-26557688 ext. 7896

Other Identifiers

A23-302

Identifier Type: -

Identifier Source: org_study_id