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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called Ropeginterferon Alfa-2B to determine its effectiveness for people with myelofibrosis, a condition where scar tissue accumulates in bone marrow and disrupts blood cell production. The researchers aim to assess whether this treatment can enhance the body's natural defenses and slow disease progression. Ideal candidates for this trial are those diagnosed with myelofibrosis who experience symptoms such as an enlarged spleen or low red blood cell counts that impact daily life. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group of participants, offering them a chance to contribute to significant medical advancements.

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.

Is there any evidence suggesting that Ropeginterferon Alfa-2B is likely to be safe for humans?

Research has shown that Ropeginterferon Alfa-2B is generally well-tolerated, causing few serious side effects. Studies have found it to be safe, with patients experiencing manageable side effects. In one study, some patients experienced side effects, but these were not severe for most. The treatment improved blood conditions and slowed disease progression in some cases. Overall, Ropeginterferon Alfa-2B appears to be a safe option for those considering participation in a clinical trial for myelofibrosis. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for myelofibrosis?

Ropeginterferon Alfa-2B is unique because it offers a new approach for treating myelofibrosis by using a modified form of interferon. Unlike traditional treatments like ruxolitinib, which focus on inhibiting specific pathways, Ropeginterferon Alfa-2B leverages the body's immune system to tackle the disease. This pegylated formulation allows for less frequent dosing, potentially reducing side effects and improving patient convenience. Researchers are excited because it could offer a more tolerable and effective alternative for managing myelofibrosis, especially for those who may not respond well to current therapies.

What evidence suggests that Ropeginterferon Alfa-2B might be an effective treatment for myelofibrosis?

Research has shown that Ropeginterferon Alfa-2B, the treatment under study in this trial, can benefit people with myelofibrosis. Studies found that most patients experienced improved blood counts within the first year of treatment. By week 24, 74% of patients had normal hemoglobin levels, and 83% had normal white blood cell counts. Additionally, nearly half of the patients had a reduced spleen size after 24 weeks. These results suggest that Ropeginterferon Alfa-2B may slow myelofibrosis and enhance blood health. ^{1 2 3 6 7}

Who Is on the Research Team?

Jeanne Palmer, M.D.

Principal Investigator

Mayo Clinic

Are You a Good Fit for This Trial?

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

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

I am currently breastfeeding.

Uncontrolled simultaneous illness

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1. sciencedirect.com sciencedirect.com/science/article/abs/pii/S2152265025002162

Real-World Evidence on Outcomes and Safety of ... After a median follow-up of 5.3 years, the hematological response (HR) rates at 36 months were 87% in PV, 75% in ET, 80% in preMF, and 45% in ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40683770/

Real-World Evidence on Outcomes and Safety of ... RopegIFN demonstrated hematological and molecular efficacy across Ph- MPN subtypes and was generally well tolerated.

3. ascopubs.org ascopubs.org/doi/10.1200/JCO.2025.43.16_suppl.6573

Ropeginterferon alfa-2b for pre-fibrotic primary ... Reduction of spleen size was found in 9 of 19 patients (47%) at Week 24, and 9 of 17 patients (53%) at Week 52. Reduction in MPNSAF-TSS of ≥50% ...

4. healthtree.org healthtree.org/myeloproliferative-neoplasm/community/articles/ropeginterferon-for-myelofibrosis

Ropeginterferon in Early-Stage Myelofibrosis Most people showed improved blood counts within the first year of therapy. At week 24, 74% reached a normal hemoglobin rate and 83% normalized ...

5. ashpublications.org ashpublications.org/blood/article/142/Supplement%201/4562/504717/Ropeginterferon-Alfa-2b-for-Pre-Fibrotic-Primary

Ropeginterferon Alfa-2b for Pre-Fibrotic Primary Myelofibrosis ... Reduction of MAF at 24 and 48 weeks was achieved for JAK2V617F in 50% (19/38) of patients (≥50%, N=10, 26%; complete, N=3, 8%) and 79% (22/28) ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10428496/

EFFICACY AND SAFETY OF ROPEGINTERFERON ALFA- ... The primary outcome were haematologic responses at 24 and 48 weeks. Secondary outcomes included adverse events (AEs), changes in allele burden of driver and non ...

7. link.springer.com link.springer.com/article/10.1007/s00277-024-05912-8

A randomized, double-blind, placebo-controlled phase 3 ... Ropeginterferon alfa-2b (ropeg) is a new-generation pegylated interferon-based therapy with favorable pharmacokinetics and safety profiles, ...