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) (VAY2EXPLORE)

May 7, 2026 updated by: Novartis Pharmaceuticals

A Phase 2 Open-label Study to Evaluate the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least One Treatment (VAY2EXPLORE)

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

The study will include an exploratory cohort of participants with primary Evans syndrome (ES) for whom IC TPO-RA therapy is appropriate per investigator's assessment.

The study will consist of a 28-day screening period; a 16-week treatment period; an IC TPO-RA tapering period during which all participants will be monitored for 16 weeks. All participants will then continue to be followed for another 60-weeks (15 months) of long-term safety follow-up period.

Study Type

Interventional

Enrollment (Estimated)

164

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Florida
      • Margate, Florida, United States, 33063
        • Recruiting
        • DH Cancer Research Center LLC
        • Principal Investigator:
          • Mazyar Rouhani
        • Contact:
    • Illinois
      • Hinsdale, Illinois, United States, 60521
        • Recruiting
        • Hope and Healing Care
        • Principal Investigator:
          • Srilata Gundala
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • 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.

Key Exclusion Criteria:

  • 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.
  • Patients exposed to more than 4 prior treatments for ITP.
  • ITP cohort only: Diagnosis of secondary thrombocytopenia.
  • ITP cohort: Use of immunosuppressant drugs other than corticosteroids or rituximab.
  • ES cohort only: Diagnosis of secondary ES.
  • ES cohort only: Life-threatening hemolysis.
  • ES cohort only: patients with autoimmune hemolytic anemia other than wAIHA

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Main cohort: Primary immune thrombocytopenia (ITP)
All participants will be assigned to ianalumab 9 mg/kg plus investigator's choice thrombopoietin receptor agonist (IC TPO-RA).
Biological: ianalumab
9 mg per kilogram infusion every 4 weeks (Q4W) for 16 weeks
Other Names:
  • VAY736
Drug: thrombopoietin receptor agonist (TPO-RA)
IC TPO-RAs will be administered according to the respective United States Prescribing Information (USPIs)
Other Names:
  • romiplostim
  • eltrombopag
  • avatrombopag
Experimental: Exploratory cohort: Primary Evans syndrome (ES)
All participants will be assigned to ianalumab 9 mg/kg plus investigator's choice thrombopoietin receptor agonist (IC TPO-RA).
Biological: ianalumab
9 mg per kilogram infusion every 4 weeks (Q4W) for 16 weeks
Other Names:
  • VAY736
Drug: thrombopoietin receptor agonist (TPO-RA)
IC TPO-RAs will be administered according to the respective United States Prescribing Information (USPIs)
Other Names:
  • romiplostim
  • eltrombopag
  • avatrombopag

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
(Main cohort: Primary immune thrombocytopenia (ITP)): Percentages of participants who are tolerable to ianalumab (9 mg/kg)
Time Frame: Up to Week 16

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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
(Main cohort: Primary immune thrombocytopenia (ITP)): Incidence rate of Adverse Events
Time Frame: From Week 1 Day 1 (first dose of ianalumab) to the end of study (EOS), an average of 4 years
The distribution of adverse events will be conducted through the analysis of frequencies for treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs), based on the monitoring of relevant clinical and laboratory safety parameters.
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
Time Frame: Baseline, and at each scheduled study visit until the end of study (EOS), an average of 2 years
Platelet count will be measured as part of routine hematology safety assessments
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)
Time Frame: Baseline, and at each scheduled study visit until the end of study (EOS), an average of 2 years
Platelet count will be measured as part of routine hematology safety assessments
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
Time Frame: From Week 17 Day 1 (W17D1) through Week 33 Day 1 (W33D1).

Tapering of investigator's choice thrombopoietin receptor agonist (IC TPO-RA) will be evaluated based on the proportion of participants who successfully discontinue IC TPO-RA without requiring rescue therapy or new immune thrombocytopenia (ITP) treatments by the end of the tapering period.

A participant is considered successfully tapered if all of the following criteria are met:

  • Discontinuation of IC TPO-RA before Week 33 Day 1 (W33D1) with at least two consecutive platelet counts ≥ 30 G/L from separate visits.
  • No use of rescue treatment or initiation of new ITP therapy in the 4 weeks prior to W33D1.
From Week 17 Day 1 (W17D1) through Week 33 Day 1 (W33D1).

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Investigators

  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

November 11, 2026

Primary Completion (Estimated)

March 3, 2027

Study Completion (Estimated)

March 31, 2027

Study Registration Dates

First Submitted

February 12, 2026

First Submitted That Met QC Criteria

February 12, 2026

First Posted (Actual)

February 19, 2026

Study Record Updates

Last Update Posted (Actual)

May 8, 2026

Last Update Submitted That Met QC Criteria

May 7, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CVAY736Q1US01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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