PRM-151G-101
Research type
Research Study
Full title
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)
IRAS ID
188394
Contact name
Karen Carroll
Contact email
Sponsor organisation
Promedior, Inc.
Eudract number
2015-001718-80
Duration of Study in the UK
1 years, 2 months, days
Research summary
Research Summary
A study to test intravenous PRM-151 for efficacy and safety in people with Primary Myelofibrosis (PMF), Post-Polycythemia Vera MF (post-PV MF), or Post-Essential Thrombocythemia MF (post-ET MF), a a disorder of the bone marrow, in which the marrow is replaced by scar (fibrous) tissue.
Patients who meet the eligibility criteria for the study will be randomly (1:1:1) allocated to treatment with PRM-151 0.3, 3, or 10 mg/kg of body weight on days 1, 3 and 5 of the first week and then on the first day of every 4 weeks for a period of 36 weeks. Patients will receive study drug treatment for 36 weeks. Approximately 84 subjects will be included in the study. After completing 36 weeks of treatment, all patients will be offered the option to receive PRM-151
in an open-label study extension for up to an additional 96 weeks utilizing 10mg/kg dose, in the absence of disease progression or toxicity warranting discontinuation of therapy until drug is made available in another way.
The study participation will include 4 week screening period, 36 week treatment period, and an open-label treatment extension period and a 4 week follow up visit.
and a 4 week follow up visit.Summary of Results
Stage 1
In Stage 1, 27 patients took zinpentraxin alfa (eight patients in Group 1, seven patients in Group 2, six patients in Group 3 and six patients in Group 4).
The main result for Stage 1 was the Overall Response Rate, which is the percentage of patients who had either: (1) improvement of their symptoms, partial remission or complete remission, or (2) at least stable disease for three treatment cycles and improvement in the bone marrow fibrosis score at any time while their disease was stable.
Stage 1 Overall Response Rate:
● Group 1: 38% of patients had a positive response (3 out of 8 patients)
● Group 2: 14% of patients had a positive response (1 out of 7 patients)
● Group 3: 33% of patients had a positive response (2 out of 6 patients)
● Group 4: 50% of patients had a positive response (3 out of 6 patients)
Stage 1 adverse effects:
All patients in Groups 1, 3 and 4 and 86% of patients in Group 2 had adverse effects during Stage 1 but these were not necessarily related to the treatment. A total of 19% of patients (5 out of 27 patients) had an adverse effect that was described as serious; for example, those that caused death, were life-threatening, or required a patient to be admitted to hospital. In Group 4, 16.7% of patients (1 out of 6 patients) had a reaction related to the drug being injected.
Stage 1 open-label extension:
A total of 13 patients continued taking zinpentraxin alfa in the Stage 1 open-label extension.
Stage 2
In Stage 2, 97 patients took zinpentraxin alfa (33 patients in the low-dose group, 32 patients in the medium-dose group and 32 patients in the high-dose group). One patient did not take any zinpentraxin alfa.
The main result in Stage 2 was the Bone Marrow Response Rate, which is the percentage of patients who had a reduction in the amount of bone marrow fibrosis at any time during the study.
Stage 2 Bone Marrow Response Rate:
● Low-dose group: 30% of patients had a positive response (10 out of 33 patients)
● Medium-dose group: 31% of patients had a positive response (10 out of 32 patients)
● High-dose group: 25% of patients had a positive response (8 out of 32 patients)
Stage 2 adverse effects:
All patients in Stage 2 had adverse effects but these were not necessarily related to the treatment. A total of 40% of patients (39 out of 97 patients) had a serious adverse effect. Overall, 3% of patients in the low-dose group, 3% of patients in the medium-dose group and 6% of patients in the high-dose group had reactions related to the drug being injected.
Stage 2 open-label extension:
A total of 48 patients continued taking zinpentraxin alfa in the Stage 2 open-label extension.Future work
Researchers will conduct further studies to learn more about how well zinpentraxin alfa works as a treatment for MF before doctors can prescribe it to their patients.REC name
London - Westminster Research Ethics Committee
REC reference
15/LO/1787
Date of REC Opinion
18 Dec 2015
REC opinion
Further Information Favourable Opinion