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Primary MyelofibrosisPhase 2NCT02370329

P1101 in Treating Patients With Myelofibrosis

A Phase 2 study testing Laboratory Biomarker Analysis, Quality-of-Life Assessment and Ropeginterferon Alfa-2B in people with Primary Myelofibrosis and Secondary Myelofibrosis, run by Mayo Clinic at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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 more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Laboratory Biomarker Analysis, Quality-of-Life Assessment and Ropeginterferon Alfa-2B
Could I receive a placebo?
No, everyone receives an active treatment
How many people
11 participants
Where
1 siteUnited States
Study dates
12 Aug 2015to 30 Nov 2023
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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

Who cannot join

  • 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
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  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Mayo Clinic in Arizona

    Scottsdale, Arizona, 85259

Other trials in United States

About this study

Official title: Phase II Study of P1101 in Early Myelofibrosis

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:

  1. 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:

  1. 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:

  1. 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.

Being tested
Laboratory Biomarker Analysis, Quality-of-Life Assessment, Ropeginterferon Alfa-2B
Medical subject headings
Primary Myelofibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Laboratory Biomarker AnalysisOther

    Correlative studies

  • Quality-of-Life AssessmentOther

    Also called Quality of Life Assessment

    Ancillary studies

  • Ropeginterferon Alfa-2BBiological

    Also called AOP2014, Besremi, P-1101, P1101

    Given SC

Study groups in NCT02370329
Study groupRoleWhat they receive
Treatment (PEG-proline-interferon alpha-2b)ExperimentalPatients 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.
Receives: Other: Laboratory Biomarker Analysis; Other: Quality-of-Life Assessment; Biological: Ropeginterferon Alfa-2B

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Up to 3 years

Secondary outcomes (2)

  1. Progression-free Survival Time

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

    Measured: 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.

    Measured: Up to 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Feb 2015 (estimated)
  2. Study start12 Aug 2015
  3. Primary completion30 Nov 2023
  4. Study completion30 Nov 2023

Why it stopped: Study drug became commercially available

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
11 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02370329
Sponsor's protocol ID
MC138F
Other IDs
NCI-2015-00183, MC138F, 14-001548
Status last verified
Sep 2024
First submitted
17 Feb 2015
Last updated
27 Oct 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Laboratory Biomarker Analysis, Quality-of-Life Assessment and Ropeginterferon Alfa-2B in people with Primary Myelofibrosis and Secondary Myelofibrosis, run by Mayo Clinic at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 19 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 27 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 27 Oct 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Best Overall Response (Complete Remission, Partial Remission, or Clinical Improvement) as Determined by International Working Group Criteria (time frame: Up to 3 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02370329.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Oct 2025.

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