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

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).

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.

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

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.