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NCT Number: NCT07660172

A Study of Ianalumab in Addition to Eltrombopag in Pediatric Patients With Primary ITP Who Failed Corticosteroids.

The purpose of this study is to assess the efficacy, safety and pharmacokinetics (PK) of ianalumab (VAY736) in addition to eltrombopag treatment; and to inform the dose of ianalumab in pediatric patients (5 to <18 years of age) with primary ITP who have had an insufficient response to or relapsed after first-line treatment with corticosteroids.

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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

5 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

About this study

The study will consist of 3 phases:

  • Screening phase
  • Treatment phase
  • Follow-up phase consisting of Efficacy follow-up phase and Safety follow-up phase

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Signed informed consent and/or assent must be obtained from the participant and/or their parent(s)/legal guardian(s) before any study-related activity or assessment is performed. Informed consent must be obtained from participants once they reach the local legal age of adulthood during the study.
  • Male or female patients aged 5 to <18 years on the day of signing informed assent/consent (as appropriate for age).
  • A confirmed diagnosis of primary ITP, with insufficient response to, or relapse after a firstline corticosteroid therapy with or without IVIG.
  • Patients with platelet count <30 G/L for whom eltrombopag is clinically indicated (per physician's discretion) and with no contraindications to receive eltrombopag.
  • Patients who are up-to-date on childhood vaccinations as per the local recommended vaccination schedule.

Exclusion criteria

  • Patients with ITP who received previous second-line ITP treatments (other than corticosteroid therapy with or without IVIG) including splenectomy. However, patients exposed to thrombopoietin receptor agonists (TPO-RAs) for a limited time (maximum one week) before or during screening are eligible.
  • Patients with key renal or hepatic laboratory abnormalities.
  • Patients with other hematologic diagnosis associated with cytopenias, including Evans Syndrome.
  • Patients with current or history of life-threatening bleeding due to thrombocytopenia.
  • Patients who are Human Immunodeficiency Virus (HIV), hepatitis C virus (HCV), hepatitis B surface antigen (HBsAg), or hepatitis B core antibody (HBcAb) positive.
  • Patients with known active or uncontrolled infections requiring systemic treatment during the screening period or history of recurrent clinically significant infection.
  • Patients with hepatic impairment (Child-Pugh score >5, or equivalent).
  • Patients with concurrent bleeding disorders or coagulation disorders and/or receiving antiplatelet or anticoagulant medication.
  • Female patients who are nursing or pregnant.

Other protocol-defined inclusion/exclusion may apply.

Treatment and study plan

Ianalumab

Biological

Liquid in a vial Concentrate for solution for infusion

Other names: VAY736

eltrombopag

Drug

Film-coated tablet and also Powder for oral suspension

Other names: ETB115

Primary outcomes

  1. Time to treatment failure (TTF)

    Time frame: enrolled until end of study (up to 39 months from the last patient enrolled)

    TTF is defined as the time from start of treatment until any of a list of specific events is met.

Secondary outcomes

  1. Stable response rate at 6 months (SR-6)

    Time frame: 6 months

    SR-6 is defined as the percentage of participants with a platelet count of at least 50 G/L at 75% of the measures taken between Study Day 121 and 183, in the absence of rescue or new Immune Thrombocytopenia (ITP) therapy.

  2. Stable response rate at 12 months (SR-12)

    Time frame: 12 months

    SR-12 is defined as the percentage of participants with a platelet count of at least 50 G/L at 66% of the measures taken between Study Day 296 and 379, in the absence of rescue or new ITP therapy.

  3. Complete Response (CR) rate

    Time frame: from enrollment until end of study (up to 39 months from the last patient enrolled)

    CR is defined as the percentage of participants with any platelet count of at least 100 G/L in the absence of rescue treatment or new ITP treatment.

  4. Response Rate (RR)

    Time frame: from enrollment until end of study (up to 39 months from the last patient enrolled)

    RR is defined as the percentage of participants with any platelet count of at least 50 G/L in the absence of rescue treatment or new ITP treatment.

  5. Time to response

    Time frame: from enrollment until end of study (up to 39 months from the last patient enrolled)

    Time to response is defined as the time from the start of treatment to the date of the first response.

  6. Time to complete response

    Time frame: from enrollment until end of study (up to 39 months from the last patient enrolled)

    Time to complete response is defined as the time from the start of treatment to the date of complete response.

  7. Duration of response

    Time frame: from enrollment until end of study (up to 39 months from the last patient enrolled)

    Duration of response is defined as the time from achievement of response (platelet count ≥ 50 G/L) to treatment failure.

  8. Duration of complete response

    Time frame: from enrollment until end of study (up to 39 months from the last patient enrolled)

    Duration of complete response is defined as the time from achievement of response to loss of complete response.

  9. Bleeding Events

    Time frame: from enrollment until end of study (up to 39 months from the last patient enrolled)

    Percentage of participants with bleeding events according to WHO Bleeding Scale

  10. Rescue medication

    Time frame: from enrollment until end of study (up to 39 months from the last patient enrolled)

    Number and percentage of participants receiving rescue treatment

  11. Rate of participants who successfully taper and discontinue eltrombopag - being treatment failure-free

    Time frame: End of Week 24

    Treatment failure-free (as defined for the primary efficacy endpoint) at the end of the planned Treatment period.

  12. Ianalumab serum concentration (Cmax)

    Time frame: at pre-dose of each of the 4 dosing events and end-of-infusion and first and last dose

    Cmax is the maximum (or peak) serum concentration that a drug achieves in a specified compartment or test area of the body.

  13. B cell levels: Change from baseline in the frequency (% within CD45+ cells) and absolute number of CD19+ B cells

    Time frame: 12 months after the first treatment of the last enrolled participant, final dbl: until end of study (up to 39 months from the last patient enrolled)

    Number and percentage of B-cell levels in the blood, and comparison with baseline levels of B-cells at specific timepoints.

  14. B cell levels: Time to first occurrence of B cell recovery

    Time frame: 12 months after the first treatment of the last enrolled participant, final dbl: until end of study (up to 39 months from the last patient enrolled)

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

  15. Immunoglobulins: Change from baseline in immunoglobulin levels

    Time frame: 12 months after the first treatment of the last enrolled participant, final dbl: until end of study (up to 39 months from the last patient enrolled)

    Change from baseline levels of serum immunoglobulins at specific timepoints.

  16. Platelet counts: Change from baseline in platelet count

    Time frame: 12 months after the first treatment of the last enrolled participant, final dbl: until end of study (up to 39 months from the last patient enrolled)

    Change from baseline levels of platelets at specific timepoints.

  17. Incidence and titer of anti-ianalumab antibodies in serum (anti-drug-antibody (ADA) assay)

    Time frame: over time (up to 39 months from the last patient enrolled)

    Anti-drug antibodies (ADAs) are immune responses developed by a participant's body against biologic or gene therapies.

Study contacts

Contact information is provided by the study sponsor or research team.

Novartis Pharmaceuticals

CONTACT

[email protected]

1-888-669-6682

Novartis Pharmaceuticals

CONTACT

+41613241111

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

A Phase 2, Open-label, Single-arm Study of Ianalumab in Addition to Eltrombopag in Pediatric Patients With Primary Immune Thrombocytopenia Who Had an Insufficient Response to or Relapsed After First-line Corticosteroid Treatment (VAYHIT-P).

Acronym: VAYHIT-P

Important dates

Study start
2027
Primary completion
2033
Study completion
2033
First posted
Jun 22, 2026
Registry last updated
Jun 25, 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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