Ropeginterferon for JAK2 Mutation

Overseen By Chi-Joan How

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Brigham and Women's Hospital

Must not be taking: Investigational agents

Disqualifiers: MPN, Pregnancy, Active malignancy, others

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

Compensation: Varies

Number of Visits: 7

Duration: 2 years

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether the drug ropeginterferon is safe and effective for individuals with a specific genetic change, the JAK2 mutation, who are at high risk but have not yet developed a blood condition called myeloproliferative neoplasm (MPN). Participants will receive an injection of ropeginterferon every four weeks and attend regular checkups. The trial seeks individuals with a JAK2 mutation who have experienced blood clots or have certain lab results higher than normal but do not yet meet the criteria for an MPN diagnosis. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that ropeginterferon is likely to be safe for humans?

Research has shown that ropeginterferon is generally well-tolerated by patients with blood disorders. One study found that patients treated with ropeginterferon responded well, and the treatment was safe for up to five years. Another analysis showed that patients with polycythemia vera (a type of blood cancer) who took ropeginterferon had good results and avoided serious side effects.

These studies suggest that ropeginterferon is safe for people with certain blood conditions. However, since this trial is in its early stages, researchers are still collecting specific safety data for people with JAK2 mutations who do not yet have myeloproliferative neoplasms (a group of diseases where the bone marrow makes too many blood cells). The focus will be on ensuring the treatment is safe for this specific group of patients. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Ropeginterferon is unique because it targets the JAK2 mutation, which is a key player in certain blood disorders. Unlike standard treatments like hydroxyurea or ruxolitinib, which generally focus on controlling symptoms, ropeginterferon works by directly addressing the genetic mutation. This approach could potentially offer more effective control over the disease by targeting its root cause. Researchers are excited because this could lead to better long-term outcomes and fewer side effects compared to conventional therapies.

What evidence suggests that ropeginterferon might be an effective treatment for JAK2 mutation?

Research has shown that ropeginterferon alfa-2b can significantly lower the JAK2V617F allele burden, indicating reduced disease activity in conditions like polycythemia vera. In some studies, it achieved high response rates, with 75% to 87% of patients showing improvements in blood cell levels. Additionally, ropeginterferon reduced spleen size, benefiting patients with blood disorders. While these results are promising, they primarily come from studies on similar conditions, not the specific group in this trial. However, the positive effects on JAK2 mutation-related conditions suggest potential benefits for participants in this new research. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

This trial is for individuals with a JAK2 mutation and high-risk features but who have not developed a myeloproliferative neoplasm. The study aims to enroll 12 such patients to determine if ropeginterferon can be safely used as a treatment.

Inclusion Criteria

I am 18 years old or older.

I have a JAK2 mutation with another high-risk feature.

Agreement to use adequate contraception

Exclusion Criteria

I have had a major organ transplant.

I have severe seizures that are not controlled.

I have a history of HIV, hepatitis C, or hepatitis B.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ropeginterferon by subcutaneous injection every 4 weeks for up to 2 years

2 years

Clinic visits every 1-3 months for checkups and tests

Follow-up

Participants are monitored for safety and effectiveness after treatment, including evaluation of response durability

2 years

Follow-up every 6 months through year 2, and again at years 3 and 4

What Are the Treatments Tested in This Trial?

Interventions

• Ropeginterferon

Trial Overview The trial is testing the safety of ropeginterferon alfa-2b in patients at high risk due to JAK2 mutations. Participants will receive injections under the skin every four weeks and undergo regular checkups and tests every one to three months.

How Is the Trial Designed?

Group I: Ropeginterferon Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Brigham and Women's Hospital

Lead Sponsor

Trials

1,694

Recruited

14,790,000+

Citations

1. nature.com nature.com/articles/s41375-022-01528-x

Long-term outcomes of polycythemia vera patients treated ... The median JAK2V617F allele burden declined continuously during ropeginterferon alfa-2b treatment, from 37.3% at baseline (before treatment in ...

2. 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 ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12623536/

A comparative study of hematologic response to ... At 48 weeks, ropeginterferon alfa-2b achieved significantly higher CHR rates (52%; 95% confidence interval [CI]: 35–70%) than historical ...

4. thejh.org thejh.org/index.php/jh/article/view/1245/806

Effective Management of Polycythemia Vera With ... After the 52-week ropeginterferon alfa-2b treatment, the mean spleen index reduced to 50.2 cm2 with a mean spleen size reduction of 5.6 cm2, as ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT07249840

Ropeginterferon for High Risk JAK2 Clonal Hematopoiesis The goal of this clinical trial is to learn if the drug ropeginterferon alfa-2b can be used safely to treat patients with a JAK2 mutation ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38500445/

Exposure-efficacy and exposure-safety analyses of ... Our analyses have shown that patients with PV treated with ropeginterferon alfa-2b had an increased probability of achieving CHR and a molecular response with ...

7. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2021/761166Orig1s000MedR.pdf

BESREMI (ropeginterferon alfa-2b-njft) Review The updated safety data provides for 60 month safety data from the CONTINUATION-PV Study. (up to 60 months of observation) whose data base lock was May 29, 2020 ...