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NCT Number: NCT05653349

Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP)

The purpose of this study is to evaluate the effect of two different doses of ianalumab versus placebo in addition to first-line corticosteroids in increasing the rate of stable response off treatment at 12 months (SROT-12) in adult participants with primary ITP.

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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 3

Primary location

Novartis Investigative Site, CABA, Buenos Aires, Argentina

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About this study

This is a multi-center, randomized, double-blind Phase 3 study to assess the efficacy and safety of two different doses of ianalumab compared to placebo in adults with primary ITP (platelets count <30 G/L) who require first-line standard-of-care corticosteroids.

After completion of the screening period, the participants will enter the randomized treatment period (ianalumab/placebo with standard of care corticosteroids).

After the treatment period, all participants will enter the follow-up period to be monitored for efficacy and safety or safety only depending on how they respond to the study treatment.

The efficacy and safety follow-up period will last until treatment failure or up to 39 months after randomization of the last patient, whichever occurs first. Safety follow-up period will be performed for at least 20 weeks and up to 2 years after the last ianalumab/placebo dose.

The primary endpoint was updated in May 2026 to stable response off treatment at 12 months (SROT-12). The previous primary endpoint, time to treatment failure (TTF), remains in the study as a key secondary objective.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Signed informed consent prior to participation in the study.
  • Male or female participants aged 18 years and older on the day of signing informed consent
  • Primary ITP diagnosed within 3 months before initiating first-line ITP therapy (corticosteroids, IVIG)
  • Platelet count below 30 G/L before starting any first-line ITP therapy (corticosteroids, IVIG)
  • Response (platelet count >=50 G/L) to corticosteroids (+/- IVIG) at any time prior to randomization. Note: Platelet count measured within 7 days of platelet transfusion will not be considered as response.

Key Exclusion Criteria:

  • Evans syndrome or any other cytopenia (patients with low grade anemia related to bleeding or iron deficiency are eligible)
  • Current life-threatening bleeding
  • Previous ITP treatment, including splenectomy, except for corticosteroids and/or IVIG initiated as first-line therapy for up to 28 days before randomization and rescue corticosteroids and/or IVIG given prior to confirmed diagnosis of primary ITP .
  • Prior use of B-cell depleting therapy (e.g., rituximab).
  • Absolute neutrophil count below 1.0 G/L at randomization
  • Participants with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication with an exemption of low dose of acetylsalicylic acid

Other protocol-defined Inclusion/Exclusion may apply.

Treatment and study plan

Ianalumab

Biological

Intravenous infusion, prepared from concentrate solution

Other names: VAY736

Placebo

Drug

Intravenous infusion, prepared from matching placebo

Corticosteroids

Drug

Oral or parental (if clinically justified)

Primary outcomes

  1. Rate of Stable Response off treatment at 12 months (SROT-12)

    Time frame: 12 months after last patient completed treatment

    A participant will be considered in SROT-12 if at least 75% of the platelet counts collected between visits Week 53 Day 1 and Week 65 Day 1 (Study Days 352 and 463) qualify as Response. Response is any platelet counts of at least 50 G/L in the absence of rescue treatment or new immune thrombocytopenia (ITP) treatment).

Secondary outcomes

  1. Time from randomization to Treatment Failure (TTF) (Key Secondary Endpoint)

    Time frame: Randomization to end of study (up to 39 months after randomization of last patient

    Time to Treatment Failure (TTF) is defined as the time from randomization until the first of the following events/situation indicative of treatment failure: platelet count below 30 G/L later than 8 weeks from randomization, need for a rescue treatment later than 8 weeks from randomization, intake of rescue treatment (e.g., corticosteroids, Intravenous immunoglobulins (IVIG), or platelet transfusion) given later than 8 weeks from randomization, start of new Immune Thrombocytopenia (ITP) treatment whenever it occurs, death (whatever the cause).

  2. Complete Response (CR) rate in each treatment group

    Time frame: Randomization to end of study (up to 39 months after randomization of last patient)

    Complete Response (CR) rate at each timepoint defined as the proportion of participants with any platelet count of at least 100 G/L in the absence of rescue treatment or new ITP treatment.

  3. Response (R) rate in each treatment group

    Time frame: Randomization to end of study (up to 39 months after randomization of last patient)

    Response (R) rate at each timepoint defined as the proportion of participants with any platelet count of at least 50 G/L in the absence of rescue treatment or new ITP treatment.

  4. Time to complete response in each treatment group

    Time frame: Randomization to end of study (up to 39 months after randomization of last patient)

    Time from randomization to date of first complete response.

  5. Duration of response in each treatment group

    Time frame: Randomization to end of study (up to 39 months after randomization of last patient)

    Time from achievement of complete response to loss of complete response

  6. Stable response at 6 months

    Time frame: At 6 months

    Percentage of participants with at least 2 platelet count collected at month 6 (between study dates 107 and 183) and at least 66% of platelet counts qualified as a response

  7. Stable response at 1 year

    Time frame: At 1 year

    Percentage of participants with at least 2 platelet counts collected at year 1 (between study days 296 and 379) and at least 66% of platelet counts qualified as a response

  8. Percentage of participants with bleeding events overall and by World Health Organization (WHO) bleeding scale severity

    Time frame: Randomization to end of study (up to 39 months after randomization of last patient)

    This is to assess the incidence and severity of bleeding in each treatment arm

  9. Number of participants with bleeding events overall and by World Health Organization (WHO) bleeding scale severity

    Time frame: Randomization to end of study (up to 39 months after randomization of last patient)

    This is to assess the number and severity of bleeding in each treatment arm

  10. Number of participants receiving rescue treatment (cummulative dose/duration of steroids exposure)

    Time frame: Randomization to end of study (up to 39 months after randomization of last patient)

    This is to assess the number of participants receiving rescue treatment.

  11. Percentage of participants receiving rescue treatment (cummulative dose/duration of steroids exposure)

    Time frame: Randomization to end of study (up to 39 months after randomization of last patient)

    This is to assess the need of rescue treatment in each treatment group by percentage.

  12. Cumulative dose/duration of steroids exposure

    Time frame: From screening to end of study (up to 39 months after randomization of last patient)

    Duration of exposure to corticosteroids calculated from randomization (first dose) to end of study or last last contact date (if the participant is lost to follow-up).

  13. Change from baseline on T scores of the PROMIS SF v1.0 Fatigue 13a

    Time frame: From screening (baseline) till end of study (up to 39 months after randomization of last patient)

    The Patient-Reported Outcomes Measurement Information System (PROMIS) Short Form v1.0 Fatigue 13a includes 13 items that assess fatigue

  14. Change from baseline in ITP-PAQ domain scores

    Time frame: From screening (baseline) till end of study (up to 39 months after randomization of last patient)

    The ITP-PAQ is a 44 item scale for measuring HRQoL in adults with ITP across ten scales: Symptoms, Bother-Physical Health, Fatigue/Sleep, Activity, Fear, Psychological Health, Work, Social Activity, Women's Reproductive Health, and Overall QoL. Each item is rated on a Likert type scale

  15. Change from baseline in frequency of CD19+ B cell counts

    Time frame: Randomization to end of study (up to 39 months after randomization of last patient)

    Post baseline frequency (%within the CD45) of CD19+ B cell counts compare to baseline.

  16. Change from baseline in absolute number of CD19+ B cell counts

    Time frame: Randomization to end of study (up to 39 months after randomization of last patient)

    Post baseline absolute number of CD19+ B cell counts compare with baseline

  17. Time to first occurrence of B-cell recovery

    Time frame: Randomization to end of study (up to 39 months after randomized of last patient)

    B-cell recovery is defined as ≥80% of baseline and ≥LLOQ, or ≥50 cells/μL

  18. Change from baseline in inmmunoglobulins

    Time frame: Randomization to end of study (up to 39 months after last randomized patients)

    Change from baseline in immunoglobulin levels

  19. PK parameters: AUClast

    Time frame: After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)

    AUClast: area under the curve from time zero till the last measurable concentration sampling time (tlast)

  20. PK parameter: AUCtau

    Time frame: After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)

    Area under the curve calculated to the end of a dosing interval (tau)

  21. PK parameters: Cmax

    Time frame: After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after 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 after single dose administration

  22. PK parameters: Tmax

    Time frame: After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)

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

  23. PK parameters: Accumulation ratio Racc

    Time frame: After last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)

    Accumulation ratio calculated using AUC values obtained between the last and first dose

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

    Time frame: Up to Week 33

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

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

    Time frame: Up to Week 33

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

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

A Phase III, Randomized, Double-blind Study of Ianalumab (VAY736) Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (VAYHIT1)

Acronym: VAYHIT1

Important dates

Study start
2023
Primary completion
2026
Study completion
2029
First posted
Dec 16, 2022
Registry last updated
Jun 15, 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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