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Study on Ropeginterferon Alfa-2b for Patients with Essential Thrombocythemia Intolerant or Refractory to Other Treatments

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition called Essential Thrombocythemia, which is a rare blood disorder where the body produces too many platelets. Platelets are small blood cells that help with clotting, and having too many can lead to problems like blood clots or bleeding. The treatment being tested in this study is a medication called ropeginterferon alfa-2b, which is given as an injection under the skin using a pre-filled pen. This medication is being evaluated for its effectiveness and safety in patients who cannot tolerate or do not respond well to other treatments that reduce the number of blood cells.

The purpose of the study is to see how well ropeginterferon alfa-2b works in managing Essential Thrombocythemia in patients who have limited options due to intolerance or lack of response to other treatments. Participants in the study will receive the medication over a period of time, and their response to the treatment will be monitored. The study will look at how the medication affects blood cell counts and whether it helps prevent complications like blood clots or bleeding. The study will also assess any changes in symptoms and overall health during the treatment period.

Throughout the study, participants will be regularly checked to ensure their safety and to monitor the effects of the treatment. The study aims to provide valuable information on the use of ropeginterferon alfa-2b for patients with Essential Thrombocythemia who have not had success with other treatments. This research could potentially offer a new option for managing this condition and improving the quality of life for those affected.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Screening phase

    The screening phase lasts up to 28 days. During this time, eligibility is confirmed based on specific criteria, including age, diagnosis, and previous treatment history.

    A washout period of at least 14 days is required if prior cytoreductive treatment was received. This period ensures that previous medications do not interfere with the study drug.

  2. Step 2

    Initiation of treatment

    The treatment involves the administration of ropeginterferon alfa-2b, known as Besremi. It is provided as a solution for injection in a pre-filled pen.

    The medication is administered subcutaneously, which means it is injected under the skin.

  3. Step 3

    Treatment schedule

    The dosage and frequency of ropeginterferon alfa-2b are determined by the study protocol and adjusted based on individual response and tolerance.

    Regular monitoring of blood counts and symptoms is conducted to assess the response to treatment and adjust the dosage if necessary.

  4. Step 4

    Evaluation of response

    The primary endpoint is the rate of disease response at month 12, evaluated according to specific criteria, including blood count remission and absence of disease progression.

    Secondary endpoints include response assessments at months 9, 18, 24, 30, and 36, as well as monitoring for any thromboembolic or bleeding events.

  5. Step 5

    Symptom assessment

    Symptomatic improvement is assessed using standardized questionnaires, such as the EQ-5D-5L and the 10-item MPN-SAF TSS.

    These assessments help to evaluate the impact of treatment on quality of life and symptom burden.

  6. Step 6

    End of study

    The study is estimated to conclude by December 28, 2027.

    Final evaluations will be conducted to determine the overall efficacy and safety of the treatment.

Who can join the trial?

9 criteria

  • The patient must provide written consent and be able to follow the study requirements.
  • The patient must be a male or female who is 18 years old or older.
  • The patient must have been diagnosed with **Essential Thrombocythaemia (ET)**, a condition where the body produces too many blood cells called platelets, according to specific criteria from the World Health Organization (WHO) in 2016. The diagnosis must include a bone marrow biopsy test result that is not older than 5 years.
  • The patient must need treatment to reduce blood cell counts but cannot tolerate or is not responding to, or is not eligible for, other approved treatments for ET. These treatments include medications like HU, ANA, BUS, and PB. Specific criteria for intolerance or resistance to these treatments must be met, such as high platelet counts despite treatment or side effects from the medication.
  • If the patient has taken previous treatments for ET, there must be a break of at least 14 days between the last dose of the previous treatment and the first dose of the study drug.
  • The patient must not have received interferon treatment before.
  • The patient must have adequate liver function, which means certain liver-related blood tests must be within acceptable limits.
  • The patient must have a score of 0-7 on both parts of the **Hospital Anxiety and Depression Scale (HADS)**, a questionnaire used to assess anxiety and depression levels.
  • If the patient has a HADS score of 8-10 on either or both parts, they may still be eligible if a psychiatric assessment shows that the symptoms are not significant in the context of potential treatment with interferon alfa.

Who cannot join the trial?

5 criteria

  • Patients who have a different condition than Essential Thrombocythaemia cannot participate. This is a condition where the body makes too many platelets, which are cells that help blood clot.
  • Patients who are not intolerant, refractory, or ineligible for other cytoreductive treatments cannot participate. Cytoreductive treatments are therapies that reduce the number of blood cells.
  • Patients who do not meet the age requirements for the study cannot participate. The study is open to certain age groups only.
  • Both male and female patients are eligible, but those who do not fit the gender criteria cannot participate.
  • Patients who are part of a vulnerable population, which means they might need special protection or care, cannot participate.
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Investigated drugs

Ropeginterferon alfa-2b is a medication used in this clinical trial to treat patients with essential thrombocythaemia, a condition where the body produces too many blood platelets. This medication is being tested for its effectiveness and safety in patients who cannot tolerate, do not respond to, or are not eligible for other treatments that reduce blood cell production. The goal is to see how well it helps manage the disease according to specific criteria set by the European Leukemia Net.

What is already known about the treatment

Ropeginterferon alfa-2b – This medication is administered via injection and is currently being studied in clinical trials for its effectiveness and safety in treating essential thrombocythaemia, particularly in patients who cannot tolerate or do not respond to other treatments. It is classified as an interferon, which works by modulating the immune system to reduce the production of blood cells that contribute to the disease. Ropeginterferon alfa-2b is being evaluated for its ability to meet response criteria set by the European Leukemia Net, and it represents a promising option for patients with limited treatment alternatives.

Investigated diseases

Essential Thrombocythemia – Essential Thrombocythemia is a rare blood disorder characterized by the overproduction of platelets by the bone marrow. This condition can lead to an increased risk of blood clots, which may cause complications such as strokes or heart attacks. Patients may experience symptoms like headaches, dizziness, and vision changes, although some individuals remain asymptomatic. Over time, the disease can progress, potentially leading to more severe symptoms or complications. The condition is often identified through routine blood tests showing elevated platelet counts. It is considered a chronic condition that requires ongoing monitoring.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-505160-12-00Protocol codeROP-ETEstimated enrolment132 patientsSponsorAop Orphan Pharmaceuticals GmbH

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