A study to test intravenous PRM-151 to see how different doses of PRM-151 act in the body and blood of people with myelofibrosis - a disorder of the bone marrow, in which the marrow is replaced by scar (fibrous) tissue.

A Phase 2, Prospective Study Of PRM-151 In Subjects With Primary Myelofibrosis (PMF), Post-Polycythemia Vera MF (post-PV MF), Or Post-Essential Thrombocythemia MF (post-ET MF) - PRM-151G-101

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2015-001718-80-FR

Enrollment

Registered

2015-12-11

Start date

2016-02-17

Completion date

Unknown

Last updated

2020-10-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Primary Myelofibrosis (PMF), Post-Polycythemia Vera MF (post-PV MF), Or Post-Essential Thrombocythemia MF (post-ET MF) MedDRA version: 18.1 Level: LLT Classification code 10074689 Term: Post polycythemia vera myelofibrosis System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) MedDRA version: 18.1 Level: LLT Classification code 10074690 Term: Post essential thrombocythemia myelofibrosis System Organ Class: 10029104 - Neoplasms benign, malignant and u

Interventions

Product Code: PRM-151 Pharmaceutical Form: Concentrate and solvent for solution for infusion

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Subjects must be =18 years of age at the time of signing the Informed Consent Form (ICF); 2. Subjects must voluntarily sign an ICF; 3. Subjects must have a pathologically confirmed diagnosis of PMF as per the WHO diagnostic criteria or post ET/PV MF; 4. At least Grade 2 marrow fibrosis according to the WHO Grading of Bone Marrow Fibrosis; 5. Intermediate-1, intermediate -2, or high risk disease according to the IWG-MRT Dynamic International Prognostic Scoring System; 6. A bone marrow biopsy must be performed within four weeks prior to Cycle 1 Day 1 treatment to establish the baseline fibrosis score; 7. Subjects must not be candidates for ruxolitinib based on EITHER: a. Platelet count 55 years, must have a negative serum pregnancy test within four weeks prior to the first dose of study drug and must agree to use adequate methods of birth control throughout the study. Adequate methods of contraception include use of oral contraceptives or Depo-Provera, with an additional barrier method (diaphragm with spermicidal gel or condoms with spermicide), double-barrier methods (diaphragm with spermicidal gel and condoms with spermicide), partner vasectomy, and total abstinence; 13. Ability to adhere to the study visit schedule and all protocol requirements; 14. Must have adequate organ function as demonstrated by the following: • ALT (SGPT) and/or AST (SGOT) = 3x upper limit of normal (ULN), or = 4 x ULN (if upon judgment of the treating physician, it is believed to be due to extramedullary hematopoiesis [EMH] related to MF); • Direct bilirubin = 1.5 x ULN; or = 2x ULN (if upon judgment of the treating physician, it is believed to be due to EMH related to MF); • Serum creatinine = 2.5 mg/dL x ULN. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 42 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 42

Exclusion criteria

Exclusion criteria: 1. White blood cell count > 25 x 10^9/L or > 10% peripheral blood blasts; 2. Other invasive malignancies within the last 3 years, except non-melanoma skin cancer and localized cured prostate and cervical cancer; 3. History of stroke, unstable angina, myocardial infarction, or ventricular arrhythmia requiring medication or mechanical control within the last 6 months; 4. Presence of active serious infection; 5. Any serious, unstable medical or psychiatric condition that would prevent, (as judged by the Investigator) the subject from signing the informed consent form or any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study; 6. Known history of human immunodeficiency virus (HIV), or known active hepatitis A, B, or C infection; 7. Organ transplant recipients other than bone marrow transplant; 8. Women who are pregnant or lactating.

Design outcomes

Primary

Measure	Time frame
Main Objective: To determine the effect size of three different doses of PRM-151 on reduction in bone marrow fibrosis by = 1 grade in intermediate-1, intermediate-2, and high risk subjects with PMF, post-PV MF, or post ET-MF who are anemic or thrombocytopenic and who are ineligible for, intolerant of, or have had an inadequate response to ruxolitinib. ;Secondary Objective: To determine if there is a difference in efficacy between the three doses of PRM-151 used in the study To evaluate the safety and tolerability of three different doses of PRM-151 To assess the duration of effect of three doses of PRM-151 on reduction in bone marrow fibrosis To assess the effect and duration of effect of three doses of PRM-151 on disease related anemia, thrombocytopenia, and constitutional symptoms To assess IWG-MRT response (Complete Response, Partial Response, Clinical Improvement), stable and progressive disease in subjects treated with three doses of PRM-151;Primary end point(s): Bone marrow response rate, defined as the percent of subjects with a reduction in bone marrow fibrosis score by at least one grade according to WHO criteria at any time during the study as determined by a central adjudication panel of expert hematopathologists, blinded to subject, treatment, and time of biopsy ;Timepoint(s) of evaluation of this end point: At any time during the study	—

Measure

Time frame

Main Objective: To determine the effect size of three different doses of PRM-151 on reduction in bone marrow fibrosis by = 1 grade in intermediate-1, intermediate-2, and high risk subjects with PMF, post-PV MF, or post ET-MF who are anemic or thrombocytopenic and who are ineligible for, intolerant of, or have had an inadequate response to ruxolitinib. ;Secondary Objective: To determine if there is a difference in efficacy between the three doses of PRM-151 used in the study To evaluate the safety and tolerability of three different doses of PRM-151 To assess the duration of effect of three doses of PRM-151 on reduction in bone marrow fibrosis To assess the effect and duration of effect of three doses of PRM-151 on disease related anemia, thrombocytopenia, and constitutional symptoms To assess IWG-MRT response (Complete Response, Partial Response, Clinical Improvement), stable and progressive disease in subjects treated with three doses of PRM-151;Primary end point(s): Bone marrow response rate, defined as the percent of subjects with a reduction in bone marrow fibrosis score by at least one grade according to WHO criteria at any time during the study as determined by a central adjudication panel of expert hematopathologists, blinded to subject, treatment, and time of biopsy ;Timepoint(s) of evaluation of this end point: At any time during the study

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Secondary

Measure	Time frame
Secondary end point(s): Comparison of primary and secondary efficacy parameters between doses Incidence of adverse events (AEs), serious adverse events (SAEs), and changes in laboratory test results Bone marrow improvement: Bone marrow response rate at weeks 12, 24, and 36 Duration of bone marrow response Hemoglobin improvement Platelet improvement Hematologic improvement Symptom improvement: Percent of subjects with 25% and 50% reduction in MPN-SAF Total Symptom Score from baseline at Week 36 Mean change from baseline in EORTC QLQ-C30 at 36 weeks Duration of all improvement parameters listed above Percent of subjects with complete response, partial response, clinical improvement, stable disease, and progressive disease according to IWG-MRT criteria ;Timepoint(s) of evaluation of this end point: At the end of Cycle 9 of the study patient.	—

Measure

Time frame

Secondary end point(s): Comparison of primary and secondary efficacy parameters between doses Incidence of adverse events (AEs), serious adverse events (SAEs), and changes in laboratory test results Bone marrow improvement: Bone marrow response rate at weeks 12, 24, and 36 Duration of bone marrow response Hemoglobin improvement Platelet improvement Hematologic improvement Symptom improvement: Percent of subjects with 25% and 50% reduction in MPN-SAF Total Symptom Score from baseline at Week 36 Mean change from baseline in EORTC QLQ-C30 at 36 weeks Duration of all improvement parameters listed above Percent of subjects with complete response, partial response, clinical improvement, stable disease, and progressive disease according to IWG-MRT criteria ;Timepoint(s) of evaluation of this end point: At the end of Cycle 9 of the study patient.

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Countries

Canada, France, Germany, Italy, Netherlands, United Kingdom, United States

Contacts

Public ContactProject Manager

Venn Life Sciences

Peter.Winkel@venncro.com31524712 456

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026