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

NCT07660172 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: CVAY736T122012026-525995-25
• Study Type: interventional
• Target: 36
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Primary Immune Thrombocytopenia (ITP)

Keywords

Primary Immune Thrombocytopenia; ITP; VAY736; ianalumab; first-line corticosteroid treatment; eltrombopag; Pediatric patients

Outcome Measures

Primary Outcomes (1)

• Time to treatment failure (TTF)

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

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

Secondary Outcomes (17)

• Stable response rate at 6 months (SR-6)

  6 months

• Stable response rate at 12 months (SR-12)

  12 months

• Complete Response (CR) rate

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

• Response Rate (RR)

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

• Time to response

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

Study Arms (1)

Ianalumab + eltrombopag

EXPERIMENTAL

Participants will receive ianalumab in addition to eltrombopag.

Biological: Ianalumab; Drug: Eltrombopag

Interventions

Ianalumab BIOLOGICAL

Liquid in a vial Concentrate for solution for infusion

Also known as: VAY736

Ianalumab + eltrombopag

Eltrombopag DRUG

Film-coated tablet and also Powder for oral suspension

Also known as: ETB115

Ianalumab + eltrombopag

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

ianalumab; eltrombopag

Condition Hierarchy (Ancestors)

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

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

1-888-669-6682; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+41613241111

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: June 15, 2026
• First Posted: June 22, 2026
• Study Start (Estimated): February 8, 2027
• Primary Completion (Estimated): April 22, 2033
• Study Completion (Estimated): April 22, 2033
• Last Updated: June 25, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will share