Ropeginterferon Alfa-2B for Myelofibrosis

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Mayo Clinic

Must not be taking: Antiretrovirals, Investigational agents

Disqualifiers: Pregnancy, HIV, Heart failure, Depression, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 3

Duration: Variable, up to 3 years

11 Participants Needed

Trial Summary

What is the purpose of this trial?

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

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, if you are on chemotherapy, radiation, or other investigational agents, you may need to stop them before joining the trial.

What data supports the effectiveness of the drug Ropeginterferon Alfa-2B for Myelofibrosis?

Ropeginterferon Alfa-2B has been shown to be effective in treating polycythemia vera, a related blood condition, by providing long-term blood response and reducing certain genetic markers. This suggests it may also be beneficial for myelofibrosis, as both conditions are part of a group of diseases called myeloproliferative neoplasms. ^{1 2 3 4 5}

Is Ropeginterferon Alfa-2B safe for humans?

Ropeginterferon Alfa-2B has been shown to be generally safe in humans, with common mild to moderate side effects like hair loss, fatigue, and flu-like symptoms. No new safety concerns were identified in studies with Japanese and European patients. ^{1 4 5 6 7}

What makes the drug Ropeginterferon Alfa-2B unique for treating myelofibrosis?

Ropeginterferon Alfa-2B is unique because it is a long-acting, mono-PEGylated interferon that requires less frequent dosing, with injections every two weeks, and potentially every four weeks after prolonged use. This makes it more convenient compared to other interferons, which often require more frequent administration. ^{1 2 4 6 8}

Research Team

Jeanne Palmer, M.D.

Principal Investigator

Mayo Clinic

Eligibility Criteria

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.

Inclusion Criteria

ANC >= 1000/mm^3

I have not received any treatment for myelofibrosis.

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

Exclusion Criteria

Uncontrolled simultaneous illness

Receiving any other investigational agent for the primary neoplasm

I am in cohort 1 and have a high or intermediate-2 risk disease.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

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

Variable, up to 3 years

Bi-weekly visits for treatment administration

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 years

Every 3-6 months

Treatment Details

Interventions

• Ropeginterferon Alfa-2B

Trial Overview 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.

Participant Groups

Group I: Treatment (PEG-proline-interferon alpha-2b) Experimental Treatment 3 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.

Who Is Running the Clinical Trial?

Mayo Clinic

Lead Sponsor

Trials

3,427

Recruited

3,221,000+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Findings from Research

Ropeginterferon alfa-2b is an effective treatment for polycythemia vera (PV), showing long-term hematological response and reduction in JAK2V617F allele burden, making it a promising option for patients without symptomatic splenomegaly.

This treatment offers improved tolerability and safety compared to traditional therapies, with benefits such as extended dosing intervals and a potential increase in overall survival by lowering the risk of disease progression.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A pharmacokinetic evaluation of ropeginterferon alfa-2b in the treatment of polycythemia vera. Illés, Á., Pinczés, LI., Egyed, M.[2022]

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

An alternative dosing strategy for ropeginterferon alfa-2b may help improve outcomes in myeloproliferative neoplasms: An overview of previous and ongoing studies with perspectives on the future. [2023]

2. Spain pubmed.ncbi.nlm.nih.gov

Ropeginterferon alfa-2b for the treatment of patients with polycythemia vera. [2020]

3. England pubmed.ncbi.nlm.nih.gov

A pharmacokinetic evaluation of ropeginterferon alfa-2b in the treatment of polycythemia vera. [2022]

4. New Zealand pubmed.ncbi.nlm.nih.gov

Pharmacokinetics and Pharmacodynamics of Ropeginterferon Alfa-2b in Healthy Japanese and Caucasian Subjects After Single Subcutaneous Administration. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Real world experience with ropeginterferon alpha-2b (Besremi) in essential thrombocythaemia and polycythaemia vera following exposure to pegylated interferon alfa-2a (Pegasys). [2023]

6. Japan pubmed.ncbi.nlm.nih.gov

Efficacy and safety of ropeginterferon alfa-2b in Japanese patients with polycythemia vera: an open-label, single-arm, phase 2 study. [2022]

7. England pubmed.ncbi.nlm.nih.gov

Novel long-acting ropeginterferon alfa-2b: Pharmacokinetics, pharmacodynamics and safety in a phase I clinical trial. [2022]

8. United States pubmed.ncbi.nlm.nih.gov

Study Protocol of a Randomized Controlled Clinical Trial to Evaluate the Efficacy and Safety of Ropeginterferon Alfa-2b in COVID-19 Patients with Comorbidities. [2023]