What is the mechanism of action of this treatment?

The most common treatments for Primary Myelofibrosis (PMF) include JAK inhibitors and interferon therapies. JAK inhibitors, such as ruxolitinib and fedratinib, work by blocking the Janus kinase (JAK) pathway, which is often overactive in PMF, leading to reduced spleen size and alleviation of symptoms. Interferon therapies, like PEG-proline-interferon alpha-2b, enhance the body's immune response and may slow the progression of myelofibrosis by targeting malignant cells. These treatments are crucial for PMF patients as they help manage symptoms, improve quality of life, and potentially modify the disease course.

What side effects are associated with this type of intervention?

Common treatments for Primary Myelofibrosis, including interferon therapies like PEG-proline-interferon alpha-2b, can lead to side effects such as flu-like symptoms, fatigue, and depression. Ruxolitinib, another frequently used treatment, is associated with anemia and thrombocytopenia, with grade ≥3 anemia occurring in 38% of patients and thrombocytopenia in 22%. Fedratinib, another option, also carries risks of anemia and gastrointestinal symptoms. Patients should discuss these potential side effects with their healthcare provider to make an informed decision.

What prior FDA approvals exist?

The FDA has approved several treatments for Primary Myelofibrosis, including ruxolitinib, which is a JAK1/JAK2 inhibitor that helps reduce spleen size and alleviate symptoms. Another treatment, fedratinib, also targets JAK2 and is used for patients resistant or intolerant to ruxolitinib. While these treatments focus on symptom management and disease progression, interferon-based therapies like PEG-proline-interferon alpha-2b, which are being studied in trials, aim to improve the body's natural response and potentially slow the growth of myelofibrosis. These interferon therapies have shown promise in related conditions such as polycythemia vera and essential thrombocythemia.

What other types of research exist?

Promising novel alternatives to P1101 for Primary Myelofibrosis patients include ruxolitinib, a JAK1/2 inhibitor that has shown efficacy in reducing spleen size and alleviating symptoms, and pegylated interferon alpha-2a, which has demonstrated high rates of hematologic and molecular response in myeloproliferative disorders. Both therapies have been investigated in clinical trials and offer potential benefits for PMF patients.

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Interferon

Ropeginterferon Alfa-2B for Myelofibrosis

Phase 2

Waitlist Available

Led By Jeanne Palmer, M.D.

Research Sponsored by Mayo Clinic

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

No prior treatment for myelofibrosis (for cohort 1 only)

For cohort 1: early stage MF without currently available treatment options

Must not have

Nursing women

Men or women of childbearing potential unwilling to employ adequate contraception

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 3 years

Awards & highlights

No Placebo-Only Group

Summary

This study is evaluating whether a substance can improve the body's natural response to myelofibrosis.

Who is the study for?

This trial is for patients with myelofibrosis who have symptoms like anemia and enlarged spleen, and are in different stages of the disease. Some should not have had treatment before, while others may have tried a drug called ruxolitinib without success. Participants need to be relatively healthy otherwise, able to answer questions about their health, and willing to provide blood samples.

What is being tested?

The trial is testing P1101 (PEG-proline-interferon alpha-2b), which might help the body fight myelofibrosis better by slowing its growth. The study includes tests on how this treatment affects patients' quality of life and tracks changes through lab biomarker analysis.

What are the potential side effects?

While specific side effects for PEG-proline-interferon alpha-2b aren't listed here, similar drugs can cause flu-like symptoms, fatigue, mood changes or depression, irritation at injection site, and sometimes changes in blood counts leading to increased risk of infections.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have not received any treatment for myelofibrosis.

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I have early stage mycosis fungoides with no treatment options left.

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My kidneys are functioning well.

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My spleen is enlarged due to myelofibrosis.

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I have intermediate-2 or high-risk myelofibrosis and cannot take or did not respond to ruxolitinib.

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I can take care of myself and am up and about more than half of my waking hours.

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I have been diagnosed with myelofibrosis according to WHO standards.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently breastfeeding.

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I am of childbearing age and not using birth control.

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I had a heart attack less than 6 months ago or need ongoing treatment for heart failure.

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I have had radiation treatment to my spleen within the last 3 months.

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I have moderate to severe numbness, tingling, or pain in my hands or feet.

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I am immunocompromised or HIV positive and on antiretroviral therapy.

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I have had a stem cell transplant before.

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I haven't had chemotherapy or radiation in the last 2 weeks.

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I do not have any other active cancer.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 3 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 3 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Best overall response (CR, PR, or CI) as determined by International Working Group Criteria

Secondary study objectives

Incidence of adverse events, as measured by National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 (NCI CTCAE v4)

Survival time

Other study objectives

Changes in patient-reported symptoms and QOL as measured by MPN-SAF

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (PEG-proline-interferon alpha-2b)Experimental Treatment3 Interventions

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Ropeginterferon Alfa-2B

2015

Completed Phase 2

~20

0 / 16Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Primary Myelofibrosis (PMF) include JAK inhibitors and interferon therapies. JAK inhibitors, such as ruxolitinib and fedratinib, work by blocking the Janus kinase (JAK) pathway, which is often overactive in PMF, leading to reduced spleen size and alleviation of symptoms. Interferon therapies, like PEG-proline-interferon alpha-2b, enhance the body's immune response and may slow the progression of myelofibrosis by targeting malignant cells. These treatments are crucial for PMF patients as they help manage symptoms, improve quality of life, and potentially modify the disease course.

Targeting myeloproliferative neoplasms with JAK inhibitors.