A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)

NCT07421167 | Recruiting Phase 2 FDA Drug

• 1 other identifier: CVAY736Q1US01
• Study Type: interventional
• Target: 164
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to investigate the tolerability of ianalumab (9 mg/kg) with investigator's choice thrombopoietin receptor agonist (IC TPO-RA) in participants diagnosed with primary immune thrombocytopenia (ITP) who have been treated with at least one but no more than four prior treatments, and with no change in IC TPO-RA dose in at least the last 14 days prior to the start of ianalumab.

Conditions

Primary Evans Syndrome (ES); Primary Immune Thrombocytopenia (ITP)

Keywords

Immune thrombocytopenia (ITP); Evans syndrome (ES); Ianalumab; VAY736; B-cell depletion; B-cell Activating Factor Receptor (BAFF-R)

Outcome Measures

Primary Outcomes (1)

• (Main cohort: Primary immune thrombocytopenia (ITP)): Percentages of participants who are tolerable to ianalumab (9 mg/kg)

  The proportion of participants who tolerate ianalumab (9 mg/kg) is defined as those who do not experience any of the following events during the combination treatment period (up to Week 16): * Discontinuation due to an adverse event (AE) unrelated to efficacy * Adverse events leading to dose reduction or dose rate reduction * Adverse events resulting in study drug interruption.

  Up to Week 16

Secondary Outcomes (4)

• (Main cohort: Primary immune thrombocytopenia (ITP)): Incidence rate of Adverse Events

  From Week 1 Day 1 (first dose of ianalumab) to the end of study (EOS), an average of 4 years

• (Main cohort: Primary immune thrombocytopenia (ITP)): Percentage of participants with platelet count ≥ 30 G/L, ≥ 50 G/L, ≥ 100 G/L

  Baseline, and at each scheduled study visit until the end of study (EOS), an average of 2 years

• (Main cohort: Primary immune thrombocytopenia (ITP)): Change from baseline in platelet count for prespecified subgroups (<30, 30 to 50, 50 to <100 G/L)

  Baseline, and at each scheduled study visit until the end of study (EOS), an average of 2 years

• (Main cohort: Primary immune thrombocytopenia (ITP)): Percentage of participants with successful IC TPO-RA tapering

  From Week 17 Day 1 (W17D1) through Week 33 Day 1 (W33D1).

Study Arms (2)

Main cohort: Primary immune thrombocytopenia (ITP)

EXPERIMENTAL

All participants will be assigned to ianalumab 9 mg/kg plus investigator's choice thrombopoietin receptor agonist (IC TPO-RA).

Biological: ianalumab; Drug: thrombopoietin receptor agonist (TPO-RA)

Exploratory cohort: Primary Evans syndrome (ES)

EXPERIMENTAL

All participants will be assigned to ianalumab 9 mg/kg plus investigator's choice thrombopoietin receptor agonist (IC TPO-RA).

Biological: ianalumab; Drug: thrombopoietin receptor agonist (TPO-RA)

Interventions

ianalumab BIOLOGICAL

9 mg per kilogram infusion every 4 weeks (Q4W) for 16 weeks

Also known as: VAY736

Exploratory cohort: Primary Evans syndrome (ES); Main cohort: Primary immune thrombocytopenia (ITP)

thrombopoietin receptor agonist (TPO-RA) DRUG

IC TPO-RAs will be administered according to the respective United States Prescribing Information (USPIs)

Also known as: eltrombopag, avatrombopag, romiplostim

Exploratory cohort: Primary Evans syndrome (ES); Main cohort: Primary immune thrombocytopenia (ITP)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed informed consent must be obtained prior to participation in the study.
• Patients aged 18 years and older on the day of signing the informed consent.
• ITP cohort only: Confirmed diagnosis of primary ITP that has previously responded to corticosteroid treatment or IVIG treatment but the response was not sustained (response is defined as a platelet count ≥ 50 G/L).
• ITP cohort only: Received at least one prior treatment for ITP.
• ITP cohort only: Patients with a platelet count \< 100 G/L who are receiving a TPO-RA. Patients may already be receiving a TPO-RA or may start a TPO-RA at the time of screening. All patients should be on a stable dose of TPO-RA for at least 14 days prior to first dose of ianalumab. Note: during the screening period, a documented assessment of platelets \< 100 G/L is mandatory for enrollment. For patients who received rescue medication before screening, platelet count results obtained prior to the start of the rescue therapy should be used to assess eligibility if collected within 14 days prior to screening.
• ES cohort only: Patients with clinical diagnosis of primary ES with active thrombocytopenia (\< 100 G/L) with warm autoimmune hemolytic anemia (wAIHA) for whom a TPO-RA is appropriate per Investigator.
• ES cohort only: Inadequate response to or relapse after treatment with corticosteroid therapy.
• ES cohort only: Diagnosis confirmed by current or past positive direct antiglobulin test (DAT) (IgG+, with or without C3+) and evidence of hemolysis.
• ES cohort only: any supportive care treatment administered for wAIHA must be stable for at least 4 weeks prior to enrollment.

You may not qualify if:

• Patients being treated with TPO-RA for \> 6 months.
• Current life-threatening bleeding (related to thrombocytopenia).
• Prior splenectomy within 6 months of first administration of ianalumab.
• Patients with the following laboratory abnormalities:
• Neutrophils: \< 1000/mm3
• Serum creatinine \> 1.5 × upper limit of normal (ULN)
• Aspartate aminotransferase (AST) \> 3.0 × ULN
• Alanine aminotransferase (ALT) \> 3.0 × ULN
• Immunoglobulin G (IgG) \< 5 g/L
• ITP cohort only: hemoglobin \< 10 g/L, total bilirubin \> 1.5 × ULN
• Patients with significantly compromised liver disease (Child-Pugh 7 to 9) and decompensated liver disease (Child-Pugh 10 to 15).
• Treatment with a B-cell depleting therapy (e.g. rituximab or anti-B cell Activating Factor (e.g. belimumab) within 12 weeks prior to the first administration of ianalumab. Patients who are refractory to rituximab will be excluded from this trial, where refractory is defined as:
• \~ Patients who have not achieved a response (defined as platelet count ≥ 30 G/L and at least doubling from baseline within 12 weeks in the absence of rescue therapy) following completion of a standard course of rituximab
• History of hypersensitivity to any of the study treatments or its excipients or to drugs of similar chemical classes.
• Known history of primary or secondary immunodeficiency, or a positive human immunodeficiency virus (HIV) enzyme-linked immunosorbent assay (ELISA) and Western blot) test result.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Hope And Healing Care

Hinsdale, Illinois, 60521, United States

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Evans Syndrome

Interventions

ianalumab; eltrombopag; avatrombopag; romiplostim

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

novartis.email@novartis.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 12, 2026
• First Posted: February 19, 2026
• Study Start (Estimated): June 29, 2026
• Primary Completion (Estimated): December 26, 2028
• Study Completion (Estimated): September 5, 2030
• Last Updated: May 5, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will share

Locations

US (1)