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TerminatedNCT02370329Updated Oct 27, 2025Results posted

P1101 in Treating Patients With Myelofibrosis

A Phase 2 interventional study of Laboratory Biomarker Analysis and Quality-of-Life Assessment in Primary Myelofibrosis and Secondary Myelofibrosis, sponsored by Mayo Clinic. Terminated at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-10-27.

Sponsored by Mayo Clinic · Phase 2, Interventional, and Treatment

Why this study was terminated
Study drug became commercially available
Phase
Phase 2
Study type
Interventional
Enrollment
11
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study 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.

Read the detailed 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.

02

Conditions studied

  • Primary Myelofibrosis
  • Secondary Myelofibrosis

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03

In context

Primary Myelofibrosis

419 studies on the registry are indexed under Primary Myelofibrosis; 117 are open to participants now.

This study's enrollment of 11 is below the median of 44 across 347 interventional studies indexed under Primary Myelofibrosis.

Browse Primary Myelofibrosis studies →

Lead sponsor

Mayo Clinic is the lead sponsor of 3,218 studies on the registry; 670 are open to participants now.

Of its 445 completed or terminated interventional studies of FDA-regulated products, 313 (70%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • 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

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
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
11 participants (actual)

Study arms

  • Experimental
    Treatment (PEG-proline-interferon alpha-2b)

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

    Other: Laboratory Biomarker Analysis · Other: Quality-of-Life Assessment · Biological: Ropeginterferon Alfa-2B

Interventions

  • OtherLaboratory Biomarker Analysis

    Correlative studies

  • OtherQuality-of-Life Assessment

    Ancillary studies

    Also known as: Quality of Life Assessment

  • BiologicalRopeginterferon Alfa-2B

    Given SC

    Also known as: AOP2014, Besremi, P-1101, P1101, PEG-P-IFN-Alfa-2b, PEG-P-IFN-Alpha-2b, PEG-Proline-Interferon Alfa-2b

06

What researchers measure

Primary outcomes

  1. Best Overall Response (Complete Remission, Partial Remission, or Clinical Improvement) as Determined by International Working Group Criteria

    Patients will be assessed for response according to the Revised International Working Group for Myeloproliferative Neoplasms Research and Treatment criteria.

    Time frame: Up to 3 years

Secondary outcomes

  1. Progression-free Survival Time

    The distribution of survival time will be estimated using the method of Kaplan-Meier.

    Time frame: Up to 3 years

  2. Number of Patients Experiencing a Grade 3+ Adverse Event, as Measured by National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 (NCI CTCAE v4)

    The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration.

    Time frame: Up to 3 years

Other outcomes

  1. Changes in Patient-reported Symptoms and QOL as Measured by MPN-SAF

    Patient-reported symptoms and QOL will be described at each time point using the mean, confidence interval, median, and range. Changes in individual symptoms, changes in a symptom scale composed of symptoms specific to MF patients, and changes in the MPN TSS will be investigated. Graphical procedures will include stream plots of individual patient scores and plots of average values over time. Correlational analyses will be done to determine the relationships among patients-reported symptoms and QOL, as well as with clinical outcomes and clinician-assessed symptoms.

    Time frame: Baseline to up to 3 years

07

Results

Posted Oct 27, 2025

Participant flow

Participant flow — Overall Study
MilestoneTreatment (PEG-proline-interferon alpha-2b)
Started11
Completed0
Not completed11
Withdrew: Alternate treatment1
Withdrew: Adverse event1
Withdrew: Disease progression3
Withdrew: Physician decision2
Withdrew: Study closure4

Outcome measures

PrimaryBest Overall Response (Complete Remission, Partial Remission, or Clinical Improvement) as Determined by International Working Group Criteria

Patients will be assessed for response according to the Revised International Working Group for Myeloproliferative Neoplasms Research and Treatment criteria.

Time frame:
Up to 3 years
Reported as:
Count of participants · Participants
Best Overall Response (Complete Remission, Partial Remission, or Clinical Improvement) as Determined by International Working Group Criteria
ParticipantsTreatment (PEG-proline-interferon alpha-2b)
Clinical Improvement7
Stable Disease3
Progression1
SecondaryProgression-free Survival Time

The distribution of survival time will be estimated using the method of Kaplan-Meier.

Time frame:
Up to 3 years
Reported as:
Median · months
Progression-free Survival Time
monthsTreatment (PEG-proline-interferon alpha-2b)
Progression-free Survival TimeNA (31.9 to NA)
SecondaryNumber of Patients Experiencing a Grade 3+ Adverse Event, as Measured by National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 (NCI CTCAE v4)

The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration.

Time frame:
Up to 3 years
Reported as:
Count of participants · Participants
Number of Patients Experiencing a Grade 3+ Adverse Event, as Measured by National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 (NCI CTCAE v4)
ParticipantsTreatment (PEG-proline-interferon alpha-2b)
Number of Patients Experiencing a Grade 3+ Adverse Event, as Measured by National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0 (NCI CTCAE v4)10
Other pre-specifiedChanges in Patient-reported Symptoms and QOL as Measured by MPN-SAF

Patient-reported symptoms and QOL will be described at each time point using the mean, confidence interval, median, and range. Changes in individual symptoms, changes in a symptom scale composed of symptoms specific to MF patients, and changes in the MPN TSS will be investigated. Graphical procedures will include stream plots of individual patient scores and plots of average values over time. Correlational analyses will be done to determine the relationships among patients-reported symptoms and QOL, as well as with clinical outcomes and clinician-assessed symptoms.

Time frame:
Baseline to up to 3 years

Results for this outcome have not been posted.

Adverse events

Collected over 3 years. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Treatment (PEG-proline-interferon alpha-2b)3/11 (27.3%)8/11 (72.7%)11/11 (100%)
Most frequent serious events
Showing 10 of 15
Most frequent serious events
EventTreatment (PEG-proline-interferon alpha-2b)
White blood cell decreasedInvestigations2/11
Atrial fibrillationCardiac disorders1/11
Chest pain - cardiacCardiac disorders1/11
Heart failureCardiac disorders1/11
Sinus bradycardiaCardiac disorders1/11
Gastrointestinal disorders - Oth specGastrointestinal disorders1/11
Allergic reactionImmune system disorders1/11
AppendicitisInfections and infestations1/11
SepsisInfections and infestations1/11
BruisingInjury, poisoning and procedural complications1/11
Most frequent other events
Showing 10 of 27
Most frequent other events
EventTreatment (PEG-proline-interferon alpha-2b)
AnemiaBlood and lymphatic system disorders9/11
White blood cell decreasedInvestigations8/11
Neutrophil count decreasedInvestigations6/11
DiarrheaGastrointestinal disorders5/11
Lymphocyte count decreasedInvestigations5/11
FatigueGeneral disorders and administration site conditions4/11
NauseaGastrointestinal disorders3/11
Platelet count decreasedInvestigations3/11
Weight lossInvestigations3/11
DizzinessNervous system disorders2/11

Baseline characteristics

Age, Continuous
Age, Continuous(years)Treatment (PEG-proline-interferon alpha-2b)
Median69 (39 to 88)
Sex: Female, Male
Sex: Female, Male(Participants)Treatment (PEG-proline-interferon alpha-2b)
Female5
Male6
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Treatment (PEG-proline-interferon alpha-2b)
Hispanic or Latino0
Not Hispanic or Latino10
Unknown or Not Reported1
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Treatment (PEG-proline-interferon alpha-2b)
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American0
White11
More than one race0
Unknown or Not Reported0
Region of Enrollment
Region of Enrollment(participants)Treatment (PEG-proline-interferon alpha-2b)
United States11
Myelofibrosis Risk Stage
Myelofibrosis Risk Stage(Participants)Treatment (PEG-proline-interferon alpha-2b)
Low/Intermediate-1 risk stage4
Intermediate-2/high risk stage7
ECOG Performance Status
ECOG Performance Status(Participants)Treatment (PEG-proline-interferon alpha-2b)
08
12
21
08

Study locations

1 site
  • Mayo Clinic in Arizona
    Scottsdale, Arizona 85259, United States
09

References and documents

Study documents

  • Protocol and statistical analysis plan · Sep 12, 2018

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Oct 27, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02370329
Lead sponsor
Mayo Clinic
Responsible party
Sponsor
First posted
Feb 24, 2015
Start date
Aug 12, 2015
Primary completion
Nov 30, 2023
Completion
Nov 30, 2023
Results posted
Oct 27, 2025
Last update
Oct 27, 2025

Study contacts

Jeanne Palmer, M.D.
principal investigator · Mayo Clinic

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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