What is the mechanism of action of this treatment?

The most common treatments for Essential Thrombocythemia (ET) include agents like Ropeginterferon alfa-2b-njft, which modulate the immune system and inhibit abnormal cell proliferation. Ropeginterferon alfa-2b-njft works by enhancing the body's immune response to target and reduce the proliferation of abnormal megakaryocytes, the cells responsible for excessive platelet production in ET. This is crucial for ET patients as it helps to control platelet counts, reduce the risk of thrombotic events, and manage symptoms. Other treatments, such as hydroxyurea, also work by inhibiting DNA synthesis in rapidly dividing cells, thereby reducing platelet production. These mechanisms are vital for managing ET, as they help prevent complications like blood clots and bleeding, improving patient outcomes and quality of life.

What side effects are associated with this type of intervention?

Common treatments for Essential Thrombocythemia, such as interferon therapies including Ropeginterferon alfa-2b-njft, often have side effects like flu-like symptoms (fever, chills, fatigue), injection site reactions, and gastrointestinal issues (nausea, vomiting). Other potential side effects include hematologic abnormalities (anemia, leukopenia), liver enzyme elevations, and neuropsychiatric symptoms (depression, anxiety). These treatments aim to modulate the immune system and inhibit abnormal cell proliferation, which can lead to these varied adverse effects.

What prior FDA approvals exist?

The FDA has approved several treatments for Essential Thrombocythemia (ET) that focus on immune modulation and inhibition of abnormal cell proliferation. One notable example is interferon alfa-2b, which has been used to manage ET by modulating the immune system and reducing the proliferation of abnormal cells. Another treatment is hydroxyurea, which inhibits DNA synthesis and reduces platelet counts. These treatments aim to control the disease by targeting the underlying mechanisms of abnormal cell growth and immune response, similar to the investigational drug Ropeginterferon alfa-2b-njft.

What other types of research exist?

Promising novel alternatives to Ropeginterferon alfa-2b-njft for Essential Thrombocythemia patients include JAK inhibitors such as ruxolitinib, which target abnormal cell proliferation and have shown efficacy in myeloproliferative disorders. Additionally, novel agents like luspatercept, which modulate the immune response and improve hematologic parameters, are being explored. Clinical trials and ongoing research into these and other targeted therapies may provide new options for ET management in 2024.

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Interferon

P1101 for Essential Thrombocythemia

Phase 2

Waitlist Available

Research Sponsored by PharmaEssentia

Eligibility Criteria Checklist

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

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new medication called Ropeginterferon alfa-2b-njft (P1101) for adults with Essential Thrombocythemia, a blood disorder. The medication helps the body control blood cell production to prevent the disease from worsening. Interferons have been studied in similar conditions for over 30 years, but earlier versions had safety and tolerability issues.

Who is the study for?

Adults diagnosed with Essential Thrombocythemia (ET) as per WHO criteria, with a platelet count over 450 × 10^9/L and good liver and kidney function. Participants must be at least 18 years old, agree to use birth control, and not breastfeed during the study. Those previously treated with ANA or HU are eligible but not if they've had poor responses or intolerance to interferon alfa therapy.

What is being tested?

The trial is testing Ropeginterferon alfa-2b-njft (P1101) in adults with ET to evaluate its effectiveness, safety, and how well patients tolerate it. This single-arm study means all participants will receive P1101 without comparison to another treatment.

What are the potential side effects?

Potential side effects of P1101 may include reactions related to the immune system such as flu-like symptoms, fatigue, headache, muscle pain; there might also be injection site reactions or changes in blood tests that monitor liver function.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

To assess efficacy of ropeginterferon alfa-2b-njft (P1101) in adult USA/Canadian patients with ET

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: Ropeginterferon alfa-2b (P1101)Experimental Treatment1 Intervention

Pre-filled Syringe, Q2W, SC injection

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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 Essential Thrombocythemia (ET) include agents like Ropeginterferon alfa-2b-njft, which modulate the immune system and inhibit abnormal cell proliferation. Ropeginterferon alfa-2b-njft works by enhancing the body's immune response to target and reduce the proliferation of abnormal megakaryocytes, the cells responsible for excessive platelet production in ET. This is crucial for ET patients as it helps to control platelet counts, reduce the risk of thrombotic events, and manage symptoms. Other treatments, such as hydroxyurea, also work by inhibiting DNA synthesis in rapidly dividing cells, thereby reducing platelet production. These mechanisms are vital for managing ET, as they help prevent complications like blood clots and bleeding, improving patient outcomes and quality of life.