Clinical Trials in Japan / NCT06468033
Active, not recruiting
Phase 3
P1101 in Treating Patients With Early PMF or Overt PMF at Low or Intermediate-1 Risk
NCT06468033 · tracked via the Priya Life Science Japan tracker
Condition(s) studied
Primary MyelofibrosisMyeloproliferative Neoplasm
Investigational drug(s) / intervention(s)
Ropeginterferon alfa-2b →Placebo
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
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 | Status |
|---|---|---|---|
| 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.
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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 Japan