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A Study of Ianalumab and Eltrombopag for Patients with Primary Immune Thrombocytopenia Who Did Not Respond Well to or Relapsed After Steroid Treatment

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What is this trial about?

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

This study involves patients with primary immune thrombocytopenia, which is a condition where the body's defense system mistakenly attacks and destroys its own platelets. Platelets are blood cells that help stop bleeding by forming clots. When platelet levels become too low, patients may experience bleeding problems. The study focuses on patients who did not respond well enough to or had their condition return after initial treatment with steroid medications. The study will test a medicine called ianalumab, which is also known by its code name VAY736, given as an infusion into a vein. This medicine will be compared to placebo, and both will be given together with another medicine called eltrombopag, which is taken by mouth. Some patients may also receive other supportive medications during the study, including antihistamines, pain relievers, normal human immunoglobulins, medicines that reduce stomach acid, glucocorticoids, and antiviral medicines like entecavir if needed.

The purpose of the study is to find out if adding ianalumab to eltrombopag can help patients go longer without their treatment failing compared to adding placebo to eltrombopag. Treatment failure means that platelet counts remain too low, that rescue treatments are needed, that new treatments for the condition must be started, that the eltrombopag dose cannot be reduced or stopped as planned, or that death occurs. The study will also look at how many patients achieve normal or near-normal platelet counts, how long these improvements last, how safe the treatment is, whether bleeding events occur, and how the treatment affects patients' daily symptoms like tiredness and overall quality of life.

Patients who join the study will be randomly assigned to receive either ianalumab or placebo, both given along with eltrombopag. The study treatment period will last for several months, during which patients will have regular visits to check their platelet counts, monitor their overall health, and assess any side effects. Blood samples will be taken to measure the levels of certain immune cells and to check how the body processes the study medicine. After the main treatment period ends, patients will continue to be followed to see how long the benefits of treatment last and to monitor their safety.

The research process

The trial runs in 10 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Randomization and treatment assignment

    After joining the study, you will be randomly assigned to one of three treatment groups. This process is called randomization and ensures fairness in the study.

    You will receive either ianalumab (at one of two different doses) combined with eltrombopag, or placebo (an inactive substance) combined with eltrombopag.

    Neither you nor your doctor will know which treatment group you are in. This is called a double-blind study design.

  2. Step 2

    Treatment period with study medications

    You will receive ianalumab or placebo through an intravenous infusion. This means the medication will be given directly into your vein.

    You will also take eltrombopag by mouth. Eltrombopag is a medication used to help increase platelet counts in patients with immune thrombocytopenia.

    The planned treatment period will last until the end of week 24.

    Your doctor may decide to reduce the dose of eltrombopag or stop it during the study, depending on your response to treatment.

  3. Step 3

    Regular monitoring and assessments

    Throughout the study, your platelet count will be checked regularly. Platelets are blood cells that help your blood clot.

    Blood samples will be taken to measure your B-cell levels. B-cells are a type of white blood cell that plays a role in your immune system.

    Blood samples will also be taken to measure immunoglobulin levels. Immunoglobulins are proteins that help your body fight infections.

    In some participants, blood samples will be collected to measure the concentration of ianalumab in the blood and to check for antibodies against ianalumab.

    You will be monitored for any side effects or adverse events throughout the study period.

  4. Step 4

    Assessment of bleeding events

    Any bleeding events will be assessed using the World Health Organization Bleeding Scale. This scale helps measure the severity of bleeding.

    Your doctor will record whether you experience any bleeding and how serious it is.

  5. Step 5

    Rescue treatment if needed

    If your platelet count drops too low (below 30 billion cells per liter) or if you need urgent treatment, you may receive rescue treatment.

    Rescue treatment refers to additional medications given to quickly increase your platelet count in case of an emergency.

    The number of times you receive rescue treatment will be recorded.

  6. Step 6

    Quality of life questionnaires

    You will be asked to complete questionnaires about how you feel during the study.

    These include the PROMIS Fatigue questionnaire, which asks about your level of tiredness.

    You will also complete the ITP Patient Assessment Questionnaire, which asks about your symptoms, fatigue, how much these bother you, and your daily activities.

    These questionnaires help measure how the treatment affects your daily life and well-being.

  7. Step 7

    Monitoring for treatment failure

    Throughout the study, your doctor will monitor for signs of treatment failure.

    Treatment failure is defined as: platelet counts remaining below 30 billion cells per liter, needing rescue treatment after 8 weeks from the start, starting a new treatment for immune thrombocytopenia, being unable to reduce or stop eltrombopag, or death.

    The time from the start of the study until treatment failure occurs will be recorded. This is called time to treatment failure.

  8. Step 8

    Assessment of treatment response

    Your doctor will assess whether you have a response to treatment. A response means your platelet count is at least 50 billion cells per liter without needing rescue treatment or new immune thrombocytopenia treatment.

    Your doctor will also assess whether you have a complete response. A complete response means your platelet count is at least 100 billion cells per liter without needing rescue treatment or new immune thrombocytopenia treatment.

    The duration of your response and whether you maintain a stable response at 6 months and at 1 year will be recorded.

  9. Step 9

    Safety monitoring

    Throughout the study, any side effects or adverse events will be carefully monitored and recorded.

    Particular attention will be paid to severe infections. The number and proportion of participants experiencing severe infections will be tracked.

    All safety information will be collected to evaluate the safety of the study treatments.

  10. Step 10

    End of treatment period

    The planned treatment period will end at week 24.

    At this time, your doctor will assess your overall response to treatment and whether you experienced treatment failure.

    Additional assessments and follow-up visits may be scheduled after the treatment period ends.

Who can join the trial?

4 criteria

  • You must be 18 years old or older at the time you sign the consent form to join this study
  • You must sign a consent form that shows you agree to take part in the study before any study activities begin
  • You must have a diagnosis of primary immune thrombocytopenia, which is a condition where your body does not have enough platelets (small blood cells that help your blood clot and stop bleeding). This diagnosis should have shown that treatment with corticosteroids (medicines that reduce inflammation and calm down your immune system) either did not work well enough or the condition came back after initial treatment. You may have also received IVIG (intravenous immunoglobulin, which is a medicine given through a vein that contains antibodies to help your immune system)
  • Your platelet count must be less than 30 G/L (a measurement showing you have a low number of platelets in your blood), and your doctor believes that treatment with eltrombopag (a medicine that helps your body make more platelets) is appropriate for you, and you have no medical reasons that would prevent you from taking eltrombopag

Who cannot join the trial?

3 criteria

  • The specific exclusion criteria (reasons why a patient cannot participate in this study) have not been provided in the available information.
  • Generally, clinical trials have exclusion criteria to ensure patient safety and that the study results are reliable, but the detailed list for this particular study is not available in the source data.
  • Patients interested in this study should discuss with their doctor whether they meet the requirements to participate, as the doctor will have access to the complete list of criteria.
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Investigated drugs

  • Ianalumab

    is an investigational medication being tested in this study. It is given in addition to standard treatment to see if it can help patients with primary immune thrombocytopenia maintain their response to treatment for a longer period of time.

  • Eltrombopag

    is a medication used as standard treatment for patients with primary immune thrombocytopenia. It works by helping the body produce more platelets, which are blood cells that help with clotting and prevent bleeding.

  • Placebo

    is an inactive substance that looks like the study medication but contains no active ingredients. It is used to compare the effects of the actual medication being tested.

What is already known about the treatment

  • Ianalumab

    Ianalumab is an investigational medication currently being studied in clinical trials for the treatment of primary immune thrombocytopenia (ITP), a condition where the immune system mistakenly destroys blood platelets, leading to increased bleeding risk. This medication is given in combination with eltrombopag, another drug that helps increase platelet counts, and is being tested in patients who did not respond well to or experienced a return of symptoms after initial steroid treatment. Ianalumab works at the molecular level by targeting specific immune cells called B cells, which are involved in producing the antibodies that attack platelets in ITP patients. It belongs to a class of medications known as monoclonal antibodies, which are designed to precisely target specific components of the immune system.

  • Eltrombopag

    Eltrombopag is an approved medication used to treat primary immune thrombocytopenia (ITP) by helping to increase the number of platelets in the blood, which are essential for normal blood clotting. It is taken orally as a tablet and works by stimulating the bone marrow to produce more platelets through activation of a specific receptor on platelet-producing cells. This medication belongs to a class called thrombopoietin receptor agonists, which mimic the natural hormone that controls platelet production in the body. Eltrombopag is commonly prescribed for patients with ITP who have not responded adequately to other treatments such as steroids or immune-suppressing medications.

Investigated diseases

Primary Immune Thrombocytopenia – Primary immune thrombocytopenia, also known as ITP, is a blood disorder in which the immune system mistakenly destroys platelets, which are cells that help blood clot. People with this condition have lower than normal platelet counts in their blood, typically below 100,000 platelets per microliter. The disease can develop after a first-line treatment with corticosteroids, and some patients may have an insufficient response to this initial therapy or experience a relapse. When platelet counts drop below 30,000 per microliter, the risk of bleeding increases. The condition can progress to a point where patients need rescue treatment or additional therapies to manage their platelet levels. ITP affects the body's ability to control bleeding, and patients may experience various bleeding events of different severity levels.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-512890-28-00Protocol codeCVAY736Q12301Estimated enrolment147 patientsSponsorNovartis Pharma AG

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