P1101 in Treating Patients With Myelofibrosis

  • STATUS
    Recruiting
  • End date
    Aug 1, 2022
  • participants needed
    19
  • sponsor
    Mayo Clinic
Updated on 18 April 2021
platelet count
chronic myeloid leukemia
myeloid leukemia
anemia
ruxolitinib
interferon
leukemia
neutrophil count
essential thrombocythemia
thrombocytosis
polycythemia

Summary

This pilot phase II trial studies P1101 (polyethyleneglycol [PEG]-proline-interferon alpha-2b) in treating patients with myelofibrosis. PEG-proline-interferon alpha-2b is a substance that can improve the body's natural response and may slow the growth of myelofibrosis.

Description

PRIMARY OBJECTIVE:

I. To evaluate for clinical response (complete remission [CR], partial remission [PR], or clinical improvement [CI]) as defined by International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria in a cohort of intermediate-2/high risk myelofibrosis (MF) patients. Response in a second cohort of early stage MF patients will also be described.

SECONDARY OBJECTIVES:

I. To evaluate the adverse event profile of P1101 in patients with myelofibrosis by cohort (early vs intermediate-2/high risk).

II. To evaluate the tolerability of P1101 in patients with myelofibrosis by cohort (early vs intermediate-2/high risk).

EXPLORATORY AND CORRELATIVE RESEARCH OBJECTIVES:

I. To evaluate quality of life (QOL) and patient-reported symptoms using the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) with P1101 for patients with myelofibrosis by cohort (early vs intermediate-2/high risk).

II. To evaluate the impact of P1101 on bone marrow and histological features of myelofibrosis including cytogenetics, blast percentage, fibrosis, and JAK2-V617F allele burden by cohort (early vs intermediate-2/high risk).

OUTLINE

Patients receive PEG-proline-interferon alpha-2b subcutaneously (SC) on days 1 and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 3-6 months for 3 years.

Details
Condition Myelosclerosis with myeloid metaplasia, Secondary Myelofibrosis, Myelofibrosis
Treatment laboratory biomarker analysis, quality-of-life assessment, PEG-Proline-Interferon Alfa-2b, ropeginterferon alfa-2b
Clinical Study IdentifierNCT02370329
SponsorMayo Clinic
Last Modified on18 April 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Is your age greater than or equal to 18 yrs?
Gender: Male or Female
Do you have any of these conditions: Myelofibrosis or Secondary Myelofibrosis or Myelosclerosis with myeloid metaplasia?
Do you have any of these conditions: Myelofibrosis or Myelosclerosis with myeloid metaplasia or Secondary Myelofibrosis?
Do you have any of these conditions: Secondary Myelofibrosis or Myelosclerosis with myeloid metaplasia or Myelofibrosis?
Do you have any of these conditions: Myelofibrosis or Secondary Myelofibrosis or Myelosclerosis with myeloid metaplasia?
Do you have any of these conditions: Secondary Myelofibrosis or Myelofibrosis or Myelosclerosis with myeloid metaplasia?
Do you have any of these conditions: Secondary Myelofibrosis or Myelofibrosis or Myelosclerosis with myeloid metaplasia?
Evaluable myelofibrosis by IWG-MRT criteria including one or more of the following
Spleen >= 5 cm below the left costal margin
MPN-SAF total symptom score (TSS) > 10 at baseline
Hemoglobin < 10 g/dL
Confirmed diagnosis of myelofibrosis (primary myelofibrosis or myelofibrosis secondary to essential thrombocythemia or polycythemia vera) by World Health Organization (WHO) diagnostic criteria (3 major and 2 minor criteria: major criteria: megakaryocyte proliferation and atypia with either reticulin and/or collagen fibrosis, not meeting criteria for chronic myelogenous leukemia [CML], polycythemia vera [PV], myelodysplastic syndrome [MDS], or other myeloid neoplasm, JAK2V617F or other clonal marker or no evidence of reactive marrow fibrosis; minor criteria: leukoerythroblastosis, increased lactate dehydrogenase [LDH], anemia, palpable splenomegaly)
For cohort 1: early stage MF (low or intermediate 1 stage as defined by Dynamic International Prognostic Scoring System [DIPSS]) without currently available treatment options
For cohort 2: intermediate-2 or high risk MF patients as defined by DIPSS either not eligible for ruxolitinib or having failed under ruxolitinib
No prior treatment for myelofibrosis (for cohort 1 only)
Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0, 1 or 2
Platelet count >= 100,000/mm^3 (obtained =< 14 days prior to registration)
Absolute neutrophil count (ANC) >= 1000/mm^3 (obtained =< 14 days prior to registration)
Aspartate transaminase (AST) =< 2.5 x upper limit of normal (ULN) (obtained =< 14 days prior to registration)
Alanine aminotransferase (ALT) =< 2.5 x ULN (obtained =< 14 days prior to registration)
Calculated creatinine clearance must be >= 50 ml/min using the Cockcroft-Gault formula (obtained =< 14 days prior to registration)
Negative pregnancy test done =< 7 days prior to registration, for women of childbearing potential only
Ability to complete questionnaire(s) by themselves or with assistance
Provide informed written consent
Willing to return to enrolling institution for follow-up
Willing to provide blood samples for correlative research purposes

Exclusion Criteria

Patients who have had chemotherapy or radiation =< 2 weeks of registration
For cohort 1 only: patients with evidence of intermediate 2 or high risk disease (according to DIPSS)
For cohort 1 only: patients with a bone marrow biopsy with < 15% cellularity, evidence of collagen fibrosis, osteosclerosis, or blasts > 10% in peripheral blood or marrow (demonstrating advanced disease)
Patients who have received a prior stem cell transplant
Patients who have received radiation to the spleen within 3 months prior to registration
Patients with intolerance to compounds similar to pegylated interferon alpha-2b
Patients with evidence of >= grade 2 peripheral sensory neuropathy
Any of the following
Pregnant women
Nursing women
Men or women of childbearing potential who are unwilling to employ adequate contraception
Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens
Immunocompromised patients or patients known to be human immunodeficiency virus (HIV) positive and currently receiving antiretroviral therapy
Uncontrolled simultaneous illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, history of depression, or psychiatric illness/social situations that would limit compliance with study requirements
Receiving any other investigational agent which would be considered as a treatment for the primary neoplasm
History of myocardial infarction =< 6 months prior to registration, or congestive heart failure requiring use of ongoing maintenance therapy for life-threatening ventricular arrhythmias
History of significant or major funduscopic findings including, but not limited to, retinal exudates, hemorrhage, detachment, neovascularization, papilledema, optic atrophy, micro-aneurysm or macular changes
Other active malignancy at time of registration; EXCEPTIONS: non-melanotic skin cancer or carcinoma-in-situ of the cervix
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