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NCT Number: NCT05653219

A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids

The purpose of this study is to evaluate the effect of two different doses of ianalumab added to eltrombopag to prolong Time to Treatment Failure (TTF) in adults with primary ITP who failed previous first-line treatment with steroids.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Primary Immune Thrombocytopenia 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, Argentina

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About this study

This is a multicenter, randomized, double-blinded phase 3 study to assess efficacy and safety of two different doses of ianalumab versus placebo in addition to eltrombopag in adults with primary ITP (platelet count <30 G/L) who failed previous first-line treatment with corticosteroids.

After completion of the screening period, the participants will enter the randomized treatment period (ianalumab/placebo with eltrombopag) followed by the eltrombopag tapering period. Afterwards, all participants will enter the follow-up period to be monitored for efficacy and safety or safety only depending on how the participants responded to the study treatment.

Who can participate

Healthy volunteers accepted: No

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

Key Inclusion criteria

  • Male or female patients aged 18 years and older on the day of signing the informed consent.
  • A signed informed consent must be obtained prior to participation in the study.
  • A diagnosis of primary ITP, with insufficient response to, or relapse after a first-line corticosteroid therapy ± IVIG.
  • Patient with platelet count <30G/L (whom eltrombopag is clinically indicated as per physician's discretion) and with no contraindication to receive eltrombopag

Key Exclusion criteria

  • ITP patients who received second-line ITP treatments (other than steroid therapy± IVIG) including splenectomy. However, patients exposed to thrombopoietin receptor agonists (TPO-RAs) for a limited time (max one week) before screening are eligible.
  • Patients with key lab abnormalities and patients with Evans syndrome or any other cytopenia, (patients with low grade anemia related to bleeding or iron deficiency are eligible).
  • Patients with history of clinically significant hematological disorders, or with marked altered hematologic parameters
  • Patients with current or history of life-threatening bleeding
  • Patient that are Human Immunodeficiency Virus (HIV), Hepatitis C Virus (HCV), Hepatitis B surface Antigen (HBsAg)/ Hepatitis B core antibody (HBcAb)-positive. HBcAb-positive patients can be enrolled if HBsAg negative, HBV DNA negative, no pre-existing liver fibrosis is present and antiviral prophylaxis is given
  • Patients with known active or uncontrolled infection requiring systemic treatment during screening period
  • Patients with hepatic impairment
  • Patients with concurrent coagulation disorders and/or receiving antiplatelet or anticoagulant medication with an exemption of low dose of acetylsalicylic acid (≤150 mg daily)
  • Nursing (breast feeding) or pregnant women

Other protocol-defined inclusion/exclusion criteria may apply.

Treatment and study plan

Ianalumab

Biological

Concentrate for solution for infusion for intravenous use

Other names: VAY736

eltrombopag

Drug

Film-coated tablet for oral use

Other names: ETB115

Placebo

Drug

Concentrate for solution for infusion for intravenous use.

Primary outcomes

  1. Time from randomization until treatment failure

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

    Time from randomization until treatment failure is defined as the time from randomization date until the first of the following events indicative of treatment failure:

    • platelet count below 30 G/L
    • start of a new ITP treatment
    • need for a rescue treatment
    • ineligibility to taper or inability to discontinue eltrombopag
    • death

Secondary outcomes

  1. Stable response at 6 months (Key Secondary Endpoint)

    Time frame: At 6 months

    Percentage of participants with at least 3 platelet count collected at month 6 (between study days 121 and 183 and at least 75% of platelet counts qualified as a response).

  2. Complete Response rate at each timepoint

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

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

  3. Response rate at each timepoint

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

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

  4. Best response rate across all timepoints

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

    Percentage of participants with a best response rate of either response or complete response

  5. Time to first response/time to first complete response

    Time frame: Time from randomization up to the longest observed treatment period duration

    Time from randomization to date of first response and time from randomization to date of first complete response

  6. Duration of response

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

    Time from achievement of response to treatment failure.

  7. Stable response at 1 year

    Time frame: At 1 year

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

  8. Duration of complete response

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

    Time from achievement of complete response to loss of complete response stable response at 1 year period

  9. Rate of participants who successfully taper and discontinue eltrombopag in each treatment arm

    Time frame: up to week 24

    Probability to be treatment failure-free (as defined for the primary efficacy endpoint)

  10. Percentage of participants with bleeding events according to World Health Organization (WHO)

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

    Percentage of participants reporting bleeding events according to WHO bleeding scale

  11. Number of participants receiving rescue treatment

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

    Number of participants who are in need of rescue treatment in each treatment arm

  12. Percentage of participants receiving rescue treatment

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

    Percentage of participants who are in need of rescue treatment

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

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

    Post-baseline frequency of CD19+ B-cell counts (percentage within CD45) compared to baseline

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

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

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

  15. Change from baseline on T-score of the PROMIS SF v1.0 Fatigue 13a

    Time frame: From screening (baseline) until end of study (up 39 months after randomization of last participant)

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

  16. Change from baseline in ITP PAQ domain scores of symptoms, fatigue, bother (uncomfortable), activity

    Time frame: From screening (baseline) until end of study (up 39 months after randomization of last participant)

    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, overall QoL. Each item is rated on a Likert type scale. Each scale is scored from 0 to 100. Higher scores represent better HRQoL.

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

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

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

  18. Change from baseline in immunoglobulins

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

    Change from baseline in immunoglobulin levels

  19. PK parameters: AUClast

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

    AUClast: Area under the curve from time zero to the last measurable concentration sampling time (tlast)

  20. PK parameters: AUCtau

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

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

  21. PK parameters: Cmax

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

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

  22. PK parameters: Tmax

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

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

  23. PK parameters: Accumulation ratio Racc

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

    Accumulation ratio calculated using AUC values obtained after 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 assessing 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 assessing the immunogenicity of ianalumab

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

A Phase 3 Randomized, Double-blind Study of Ianalumab (VAY736) Versus Placebo in Addition to Eltrombopag in Patients With Primary Immune Thrombocytopenia (ITP) Who Had an Insufficient Response or Relapsed After First Line Steroid Treatment (VAYHIT2)

Acronym: VAYHIT2

Important dates

Study start
2023
Primary completion
2025
Study completion
2028
First posted
Dec 16, 2022
Registry last updated
Jun 16, 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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