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NCT Number: NCT05885555

A Study of Ianalumab (VAY736) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least Two Lines of Therapies

The purpose of this study is to evaluate the therapeutic efficacy, safety and tolerability of ianalumab in adult patients with primary ITP previously treated with at least one corticosteroid and one TPO-RA.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Primary Immune Thrombocytopenia (ITP) Autoimmune Diseases Blood Coagulation Disorders Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders Immune System Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Purpura Purpura, Thrombocytopenic Purpura, Thrombocytopenic, Idiopathic Signs and Symptoms Skin Manifestations Thrombocytopenia Thrombotic Microangiopathies

Age range

18 year–100 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Novartis Investigative Site, CABA, Argentina

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About this study

This is a phase 2, open-label, single-arm study to evaluate the efficacy, safety and tolerability of ianalumab in participants with primary ITP (platelet count <30 G/L at screening) previously treated with at least a corticosteroid and a TPO-RA.

The study consists of the screening period, the primary endpoint assessment period, the follow-up period. The screening period will last for up to 14 days prior to the first dose of ianalumab. All eligible participants will be treated with the same dose of ianalumab and will complete the primary endpoint assessment period. After completion of the primary endpoint assessment period, all participants will continue in safety monitoring and those with a platelet count ≥30 G/L in absence of a new line of ITP therapy and rescue therapy will also continue in efficacy monitoring. The trial includes an option to offer a second course of ianalumab treatment to participants who achieved confirmed response during the initial course of ianalumab and later lost response to explore the benefit of the second course of treatment. The study will end once all participants have completed 24 months of safety follow-up since their last dose of ianalumab (including the optional second course of ianalumab treatment),or discontinued the study earlier.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Signed informed consent obtained prior to participation in the study.
  • Male or female participants aged 18 years and older on the day of signing informed consent.
  • Confirmed diagnosis of primary ITP.
  • Prior treatment with at least a corticosteroid (±IVIG) and a TPO-RA:
  • Prior additional therapies are allowed; the corticosteroid or the TPO-RA do not need to be the last treatment.
  • Prior response to IVIG/anti-D or a corticosteroid (platelet count ≥50 G/L) that was not maintained.
  • At last ITP treatment, loss of response, insufficient response, no response or intolerance.
  • Platelet count <30 G/L and assessed as needing treatment (per physician's discretion) at screening. If concomitant ITP medication is clinically indicated, the platelet assessment showing a value <30 G/L must be performed after at least 14 days on a stable dose of a corticosteroid or/and a TPO-RA (less than 10% variation from current dose) and continue stable thereafter.

Key exclusion criteria:

  • Diagnosis of secondary thrombocytopenia.
  • Platelet or whole blood transfusion, plasmapheresis, or use of any other rescue medications within 14 days before first ianalumab infusion.
  • Participants with the following conditions at screening:
  • Neutrophils <1000/mm3.
  • Immunoglobulin G (IgG) <5 g/L
  • Treatment with a B-cell depleting therapy (e.g., rituximab) or anti-B-cell Activating Factor of the TNF Family (BAFF) (e.g., belimumab) within 12 weeks prior to the first administration of ianalumab.
  • Immunosuppressant drugs other than corticosteroids within 5 times the elimination half-life of the drug or 14 days before first ianalumab infusion, whichever is longer.
  • Prior splenectomy.

Other protocol-defined inclusion/exclusion criteria may apply.

Treatment and study plan

Ianalumab

Biological

Intravenous infusion, prepared from concentrate solution

Other names: VAY736

Primary outcomes

  1. Confirmed response

    Time frame: Between Week 1 Day 1 and Week 25 Day 1

    Confirmed response is defined as a platelet count of equal or above 50 G/L at two (or more) consecutive assessments at least 7 days apart, in the absence of:

    • Rescue treatment for ≥4 weeks prior to the assessment of the platelet count, and
    • New immune thrombocytopenia (ITP) treatment before reaching a confirmed response.

Secondary outcomes

  1. Time to confirmed response

    Time frame: From Week 1 Day 1 to Week 25 Day 1

    Time from the first administration of ianalumab to the first assessment in the first sequence of two (or more) platelet assessments meeting the criteria of a confirmed response as defined by the primary endpoint.

  2. Duration of confirmed response

    Time frame: From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)

    Time from the first assessment in the first sequence of two (or more) platelet assessments meeting the criteria of a confirmed response to loss of response; with loss of response defined as the first of the following events:

    • platelet count <30 G/L,
    • start of any rescue or new ITP treatment,
    • death (whatever the cause).

  3. Complete Response rate at each timepoint

    Time frame: From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)

    Percentage of participants with a platelet count of at least 100 G/L in the absence of rescue treatment/new ITP treatment.

  4. Response rate at each timepoint

    Time frame: From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)

    Percentage of participants with a platelet count of at least 50 G/L in the absence of rescue treatment/new ITP treatment.

  5. Stable response at 6 months

    Time frame: At 6 months

    Percentage of participants with at least 75% of platelet counts collected at month 6 (between study days 121 and 183) equal to or above 50 G/L in the absence of rescue treatment/new ITP treatment.

  6. Stable response at 1 year

    Time frame: At 1 year

    Percentage of participants with at least 66% of platelet counts collected at year 1 (between study days 296 and 379) equal to or above 50 G/L in the absence of rescue treatment/new ITP treatment.

  7. Bleeding events according to the Modified World Health Organization (WHO) bleeding scale

    Time frame: From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)

    Number of participants reporting bleeding events for each grade of the World Health Organization (WHO) bleeding scale at each time point. Severity is graded from 0 to 4, with 0 = no bleeding and 4 = severe hemodynamic instability/central nervous system (CNS) bleeding/fatal.

  8. Percentage of participants with bleeding events according to the Modified World Health Organization (WHO) bleeding scale

    Time frame: From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)

    Percentage of participants reporting bleeding events for each grade of the WHO bleeding scale at each time point. Severity is graded from 0 to 4, with 0 = no bleeding and 4 = severe hemodynamic instability/central nervous system (CNS) bleeding/fatal.

  9. Number of participants who received rescue treatment

    Time frame: From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)

    Number of participants who required rescue treatment

  10. Percentage of participants who received rescue treatment

    Time frame: From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)

    Percentage of participants who required rescue treatment

  11. Change from baseline in the frequency of CD19+ B-cell counts

    Time frame: From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)

    Post-baseline frequency of CD19+ B-cell counts compared to baseline

  12. Change from baseline in the absolute number of CD19+ B-cell counts

    Time frame: From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)

    Post-baseline absolute number of CD19+ B-cell counts compared to baseline

  13. Time to first occurrence of B-cell recovery defined as ≥80% of baseline ≥50 cells/µL

    Time frame: From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)

    Time to B-cell recovery defined as ≥80% of baseline or ≥50 cells/µL

  14. Change from baseline in immunoglobulins

    Time frame: From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)

    Post-baseline immunoglobulin levels (change in titers of Total Ig, IgG, IgM, IgA) compared to baseline

  15. Incidence of anti-ianalumab antibodies in serum (ADA assay) over time

    Time frame: Up to 20 weeks after last dose of ianalumab

    Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants to assess the immunogenicity of ianalumab

  16. Titer of anti-ianalumab antibodies in serum (ADA assay) over time

    Time frame: Up to 20 weeks after last dose of ianalumab

    Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants to assess the immunogenicity of ianalumab

  17. Ianalumab serum concentrations over time

    Time frame: First dose (pre-dose, 2, 168, 336, 504, 672 hours post-dose); Subsequent doses (pre-dose and 2 hours post-dose); Last dose (pre-dose, 2 336, 672, 1344, 2016, 3360 hours post-dose)

    Ianalumab concentration in serum over time, including end of infusion and concentration at trough.

  18. Ianalumab serum PK: AUC concentrations over time

    Time frame: First dose (pre-dose, 2, 168, 336, 504, 672 hours post-dose); Subsequent doses (pre-dose and 2 hours post-dose); Last dose (pre-dose, 2 336, 672, 1344, 2016, 3360 hours post-dose)

    Ianalumab concentration in serum over time, including end of infusion and concentration at trough.

  19. Ianalumab serum PK parameters: Cmax

    Time frame: First dose (pre-dose, 2, 168, 336, 504, 672 hours post-dose); Subsequent doses (pre-dose and 2 hours post-dose); Last dose (pre-dose, 2 336, 672, 1344, 2016, 3360 hours post-dose)

    Maximum (peak) observed plasma, blood, serum or other body fluid drug concentration

  20. Ianalumab serum PK parameters: Tmax

    Time frame: First dose (pre-dose, 2, 168, 336, 504, 672 hours post-dose); Subsequent doses (pre-dose and 2 hours post-dose); Last dose (pre-dose, 2 336, 672, 1344, 2016, 3360 hours post-dose)

    Time to reach maximum (peak) observed plasma, blood, serum or other body fluid drug concentration

  21. Confirmed response (CR) in the second course

    Time frame: Second course Week 1 Day1 to second course Week 25 Day1

    Confirmed response is defined as a platelet count of equal or above 50 G/L at two (or more) consecutive assessments at least 7 days apart, in the absence of:

    • Rescue treatment for ≥4 weeks prior to the assessment of the platelet count, and
    • New ITP treatment before reaching a confirmed response.

  22. Time to confirmed response in the second course

    Time frame: Second course Week 1 Day 1 to second course Week 25 Day 1

    two (or more) platelet assessments meeting the criteria of a confirmed response.

  23. Duration of confirmed response in the second course

    Time frame: Second course Week 1 Day 1 to second course Week 25 Day 1

    Time from the first assessment, in the second course of two (or more) platelet assessments meeting the criteria of a confirmed response to loss of response; with loss of response defined as the first of the following events:

    • platelet count <30 G/L,
    • start of any rescue or new ITP treatment,
    • death (whatever the cause).

  24. Response in the second course

    Time frame: Second course Week 1 Day 1 to second course Week 25 Day 1

    Percentage of participants with a platelet count of at least 50 G/L in the absence of rescue treatment/new ITP treatment.

  25. Complete Response in the second course

    Time frame: Second course Week 1 Day 1 to second course Week 25 Day 1

    Percentage of participants with a platelet count of at least 100 G/L in the absence of rescue treatment/new ITP treatment.

  26. Bleeding events in the second course according to the Modified World Health Organization (WHO) bleeding scale

    Time frame: Second course Week 1 Day 1 to second course Week 25 Day 1

    Number of participants reporting bleeding events for each grade of the World Health Organization (WHO) bleeding scale at each time point. Severity is graded from 0 to 4, with 0 = no bleeding and 4 = severe hemodynamic instability/central nervous system (CNS) bleeding/fatal.

  27. Percentage of participants with bleeding events in the retreatment/second courseaccording to the Modified World Health Organization (WHO) bleeding scale

    Time frame: Second course Week 1 Day 1 to second course Week 25 Day 1

    Percentage of participants reporting bleeding events for each grade of the WHO bleeding scale at each time point. Severity is graded from 0 to 4, with 0 = no bleeding and 4 = severe hemodynamic instability/central nervous system (CNS) bleeding/fatal.

  28. Number of Participants who received rescue treatment after second course

    Time frame: Second course Week 1 Day 1 to second course Week 25 Day 1

    Number of participants who required rescue treatment

  29. Percentage of participants who received rescue treatment after receiving second course

    Time frame: Second course Week 1 Day 1 to second course Week 25 Day 1

    Percentage of participants who required rescue treatment

  30. Titer of anti-ianalumab antibodies in serum (ADA assay) over time for second course

    Time frame: Second course Week 1 Day 1 until 20 weeks after last dose of ianalumab

    Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants to assess the immunogenicity of ianalumab

  31. Change from start of second course in immunoglobulins

    Time frame: From second course Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)

    Post-baseline immunoglobulin levels (change in titers of Total Ig, IgG, IgM, IgA) compared to retreatment baseline

  32. Change from start of second course in the absolute number of CD19+ B-cell counts

    Time frame: From second course Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)

    Post-baseline absolute number of CD19+ B-cell counts compared to baseline

  33. Change from start of second course to end of study in the absolute number of CD19+ B-cell counts

    Time frame: From second course Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)

    Post-baseline absolute number of CD19+ B-cell counts compared to baseline

  34. Time to first occurrence of B-cell recovery defined as ≥80% of baseline ≥50 cells/µL

    Time frame: From second course Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)

    Time to B-cell recovery defined as ≥80% of baseline or ≥50 cells/µL

  35. Ianalumab serum concentrations over time in the second course

    Time frame: First dose (pre-dose, 2, 168, 336, 504, 672 hours post-dose); Subsequent doses (pre-dose and 2 hours post-dose); Last dose (pre-dose, 2 336, 672, 1344, 2016, 3360 hours post-dose) in the second course

    Ianalumab concentration in serum over time, including end of infusion and concentration at trough.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

A Phase 2 Study to Evaluate the Efficacy and Safety of Ianalumab (VAY736) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least a Corticosteroid and a Thrombopoietin Receptor Agonist (TPO-RA)

Important dates

Study start
2023
Primary completion
2025
Study completion
2029
First posted
Jun 2, 2023
Registry last updated
Apr 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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