Evaluating Peginterferon for treating Essential Thrombocythemia and Polycythemia Vera

A Single-arm, Single-center Study to Explore the Safety and Efficacy of Pegylated Interferon Alpha in Chinese Patients With ET (Essential Thrombocythemia) and PV (Polycythemia Vera).

This study is testing if a weekly injection of Peginterferon can help people with Essential Thrombocythemia and Polycythemia Vera feel better and stay safe while using the treatment.

Quick facts

• Phase: NA
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Tongji Hospital (other)
• Locations: 1 site (Wuhan, Hubei)
• Trial ID: NCT06734637 on ClinicalTrials.gov

What this trial studies

This study is a single-arm, single-center clinical trial that aims to recruit 40 participants diagnosed with Essential Thrombocythemia (ET) and Polycythemia Vera (PV). Participants will receive a weekly subcutaneous injection of Peginterferon α-2b at a dosage of 180 mcg, and the study will assess both the efficacy and safety of this treatment. The trial follows the 2016 WHO diagnostic criteria for ET and PV to ensure appropriate patient selection. Follow-up evaluations will be conducted to monitor treatment outcomes.

Who should consider this trial

Good fit: Ideal candidates for this study are individuals who meet the diagnostic criteria for Essential Thrombocythemia or Polycythemia Vera as defined by the 2016 WHO guidelines.

Not a fit: Patients who do not meet the diagnostic criteria for ET or PV, or those with other myeloproliferative neoplasms, may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from Essential Thrombocythemia and Polycythemia Vera.

How similar studies have performed: While the specific use of Peginterferon α-2b for these conditions may be novel, similar studies involving interferon treatments have shown promise in managing myeloproliferative disorders.

Eligibility criteria

Inclusion Criteria:

1. Meet the 2016 WHO diagnostic criteria for ET (Essential Thrombocythemia) and PV (Polycythemia Vera)

   * ET (Essential Thrombocythemia) major criteria:

     1. Platelet count \> 450 \* 10\^9/L; (2) Bone marrow biopsy shows marked megakaryocytic proliferation with increased mature megakaryocyte volume and increased nuclear lobulation. There is no significant granulocytic proliferation, left shift, or erythroid proliferation, with grade 1 fibrosis observed in a few cases; (3) Does not meet the WHO diagnostic criteria for BCR-ABL1+ CML, PV, PMF, MDS, or other myeloproliferative neoplasms; (4) JAK2, CALR, or MPL mutation positive.

        Secondary criteria: (1) Presence of a clonal marker or absence of evidence for reactive thrombocytosis.

        The diagnosis of ET requires the fulfillment of all four main criteria, or the first three main criteria plus one secondary criterion.
   * PV (Polycythemia Vera) main criteria: (1) Hemoglobin \> 16.5 g/dL in males, \> 16 g/dL in females, or Hematocrit \> 49% in males, \> 48% in females, or an increase in red cell volume of 25% or more above the normal value; (2) Bone marrow biopsy shows increased cellularity inappropriate for age, with marked erythroid, granulocytic, and megakaryocytic proliferation, and the presence of mature megakaryocytes of varying sizes and morphologies; (3) JAK2 V617F mutation positive or JAK2 exon 12 mutation positive.

   Secondary criteria: Serum Epo (Erythropoietin) level below the lower limit of the normal range.

   The diagnosis of PV requires the fulfillment of three major criteria, or the first two main criteria plus one secondary criterion.
2. For ET patients, the criteria for cytoreductive therapy must be met, as follows:

(1) For patients without a history of thrombosis: Age ≥ 60 years, regardless of the presence of cardiovascular risk (CVR) or JAK2V617 mutation; Any age with a platelet count \> 1500 × 109/L. (2) For patients with a history of arterial thrombosis: Any age, regardless of the presence of CVR and JAK2V617 mutation; (3) For patients with a history of venous thrombosis: Any age, regardless of the presence of CVR and JAK2V617 mutation; 3.ECOG score ≤ 2. 4.Cardiac ejection fraction (EF) ≥ 60%. 5.The participant voluntarily signs the informed consent form.

Exclusion Criteria:

* Previous treatment with Peginterferon α-2b.
* ET patients who do not meet the criteria for cytoreductive therapy as per the 2016 Chinese Expert Consensus on Primary Thrombocythemia.
* Allergy to the active ingredient, α-interferon, or any excipients of this product.
* History of psychiatric illness, or allergy to interferon.
* Plasma total bilirubin greater than twice the normal value.
* Severe cardiac disease, liver insufficiency, chronic kidney disease, and neurological disorders (such as depression or mania).
* History of other malignant tumors within the past three years.
* Autoimmune chronic hepatitis.
* Any condition deemed unsuitable for inclusion by the investigator.

Where this trial is running

Wuhan, Hubei

• Tongji Medical College, Huazhong University of Science and Technology — Wuhan, Hubei, China (RECRUITING)

Study contacts

• Principal investigator: ZhenYa Hong, Ph.D — Tongji Medical College, Huazhong University of Science and Technology
• Study coordinator: ZhenYa Hong, Ph.D

Conditions: Essential Thrombocythemia, Polycythemia Vera, Essential thrombocythemia, Polycythemia vera, Peginterferon α-2b