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

NCT06468033 · tracked via the Priya Life Science USA tracker

Sponsor

PharmaEssentia

Phase

Phase 3

Started

2025-07-18

Last updated

2026-08-13

Condition(s) studied

Primary MyelofibrosisMyeloproliferative Neoplasm

Investigational drug(s) / intervention(s)

Ropeginterferon alfa-2bPlacebo

Ropeginterferon alfa-2b: Pre-filled Syringe. Dosage: up to 500mcg

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

Study 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.

Eligibility

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

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.

Primary outcome measure(s)

Number of Participants with Platelet count equal or less (≤) 400 × 10^9/L — 80 weeks
Platelet count ≤400 × 10\^9/L is one of the criteria for clinically relevant complete hematologic response (CrCHR).
Number of Participants with White Blood Cells (WBC) count equal or less (≤) 10 × 10^9/L — 80 weeks
White Blood Cells (WBC) count ≤10 × 10\^9/L is one of the criteria for clinically relevant complete hematologic response (CrCHR).
Number of Participants with Hemoglobin (Hgb) equal or greater (≧) 10.0 g/dL — 80 weeks
Peripheral blood: Hemoglobin (Hgb) ≧ 10.0 g/dL is one of the criteria for clinically relevant complete hematologic response (CrCHR).
Number of Participants absence of major thrombotic events — 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).
Number of Participants with no progression to secondary acute myeloid leukemia (AML). — 80 weeks
The absence of progression to secondary acute myeloid leukemia (AML) is one of the criteria for clinically relevant complete hematologic remission (CrCHR).
Number of Participants with no progression on the Total Symptom Score (TSS) — 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 (\&amp;gt;) 10.

Trial sites (73)

Facility	City	Region
USC Norris Comprehensive Cancer Center	Los Angeles	California
Georgetown University Medical Center	Washington D.C.	District of Columbia
University of Kansas Medical Center (KUMC)	Kansas City	Kansas
American Oncology Partners of Maryland PA (Center for Cancer and Blood Disorders)	Bethesda	Maryland
Washington University School of Medicine	St Louis	Missouri
Weill Medical College of Cornell University	New York	New York
Stony Brook University Medical Center	Stony Brook	New York
Levine Cancer Institute	Charlotte	North Carolina
Duke University	Durham	North Carolina
Ohio State University	Columbus	Ohio
MD Anderson Cancer Center	Houston	Texas
Virginia Commonwealth University	Richmond	Virginia
University of Washington Medical Center - Fred Hutchinson Cancer Center	Seattle	Washington
St. Michael's Hospital	Toronto	Ontario
Princess Margaret Hospital	Toronto	Ontario
Jewish General Hospital	Montreal	Quebec
Peking Union Medical College Hospital	Beijing	Beijing Municipality
Peking University People's Hospital	Beijing	Beijing Municipality
The Second Affiliated Hospital of AMU	Chongqing	Chongqing Municipality
Southern Hospital of Southern Medical University	Guangdong	Guangdong
Guangdong Provincial People's Hospital	Guangzhou	Guangzhou
Institute of Hematology and Oncology, Harbin The First Hospital	Harbin	Heilongjiang
Henan Cancer Hospital	Zhengzhou	Henan
Union Hospital Tong Ji Medical College	Wuhan	Hubei
Jiangsu Provincial Hospital	Nanjing	Jiangsu
The First Affiliated Hospital of Soochow University	Suzhou	Jiangsu
Qilu Hospital of Shandong University	Jinan	Shandong
Ruijin Hospital, Shanghai Jiaotong University School of Medicine	Shanghai	Shanghai Municipality
Shanghai Sixth People's Hospital	Shanghai	Shanghai Municipality
West China Hospital, Sichuan University	Chengdu	Sichuan
Hematology Hospital of Chinese Academy of Medical Sciences	Tianjin	Tianjin Municipality
The First Affiliated Hospital, Zhejiang University School of Medicine	Hangzhou	Zhejiang
Queen Mary Hospital	Hong Kong	Hong Kong
Kameda General Hospital	Kamogawa	Chiba
Ehime University Hospital	Matsuyama	Ehime
Hiroshima Red Cross & Atomic-Bomb Survivors Hospital	Hiroshima	Hiroshima
Iwate Prefectural Central Hospital	Morioka	Iwate
Shonan Kamakura General Hospital	Kamakura	Kanagawa
Mie University Hospital	Tsu	Mie-ken
University of Miyazaki Hospital	Miyazaki	Miyazaki

+ 33 more sites — see the full list on the official registry below.

More PharmaEssentia trials in the USA

A Study to Assess Efficacy, Safety, and Tolerability of P1101 in Adult Patients With PV Active, not recruiting

A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET Active, not recruiting

Ropeginterferon Alfa-2b (P1101) vs. Anagrelide in Essential Thrombocythemia Patients With Hydroxyurea Resistance or Intolerance Active, not recruiting

Other trials for the same condition

A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis Active, not recruiting

A Phase 1 Study of LY5830966 in Participants With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (PPV-MF), or Post-Essential Thrombocythemia Myelofibrosis (PET-MF) Who Have Been Failed by a Type I JAK2 Inhibitor (JAK2i) Recruiting

A Study to Evaluate Safety and Efficacy of Bomedemstat (MK-3543-017) Recruiting

Study of TL-895 Combined With Ruxolitinib in JAKi Treatment-Naïve MF Subjects and Subjects With MF Who Have a Suboptimal Response to Ruxolitinib Recruiting

Safety and Tolerability Study of INCB057643 in Participants With Myelofibrosis and Other Advanced Myeloid Neoplasms Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06468033 on ClinicalTrials.gov ↗ ← All trials in the USA