ClinicalTrials.gov record NCT07421167

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)

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

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)

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age18 years old and older
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 2, open-label study tests how well adults with primary ITP tolerate ianalumab 9 mg/kg given every 4 weeks together with an investigator's choice thrombopoietin receptor agonist (TPO‑RA). It includes an exploratory cohort for people with primary Evans syndrome. Eligible adults are previously treated for ITP (at least one and up to four prior treatments) and should be on a stable TPO‑RA dose for at least 14 days before starting ianalumab. The study has a 28‑day screening, 16‑week treatment period, a TPO‑RA tapering period, and about 15 months of safety follow‑up.

What it is studying. The study is testing the tolerability of ianalumab (9 mg/kg IV every 4 weeks) when given together with an investigator's choice TPO‑RA in adults with primary ITP; an exploratory group includes people with primary Evans syndrome. Tolerability is measured by whether participants experience adverse events that lead to stopping, interrupting, or reducing the study drug during the combination treatment period.

What the researchers hope to learn. To evaluate tolerability of ianalumab 9 mg/kg given with investigator's choice TPO‑RA. To collect safety data (adverse events), track platelet counts over time, and assess whether participants can successfully taper and discontinue TPO‑RA during the study.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • thrombopoietin receptor agonist (TPO-RA)
Biological
  • ianalumab

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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 description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

After a screening period (about 28 days), participants receive ianalumab infusions (9 mg/kg) every 4 weeks during a 16‑week treatment period while taking an investigator‑chosen TPO‑RA per its prescribing information. There is then a TPO‑RA tapering period with monitoring for 16 weeks, followed by about 60 weeks (15 months) of long‑term safety follow‑up. Participation involves regular clinic visits for the infusions, frequent blood tests including platelet counts, and safety monitoring for adverse events. The study protocol may include temporary interruptions, dose changes, or rescue treatments if clinically required.

How the study is structured. Interventional Phase 2, randomized, parallel-group, open‑label trial. All participants receive ianalumab 9 mg/kg every 4 weeks plus their investigator‑chosen TPO‑RA per its prescribing information. The timeline includes screening, a 16‑week combination treatment period, a TPO‑RA tapering window, and long‑term safety follow‑up of about 60 weeks. There are two cohorts: a main ITP cohort and an exploratory Evans syndrome cohort.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Evans syndrome

Clinical trials7 trials listed for Evans syndromeEvery trial the registry lists for Evans syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Evans syndrome researchLatest monthly update: September 2026. Includes: Genetic immune disorders. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Aug 4, 2026
Primary completion
Jan 18, 2029 (estimated)
Study completion
Aug 31, 2030 (estimated)
Target enrollment
164 participants (estimated)
Last updated on registry
Oct 9, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Novartis Pharmaceuticals

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (16)

  • Highlands Oncology GroupRecruiting

    Fayetteville, Arkansas, 72703 · United States

  • Yale Cancer CenterRecruiting

    New Haven, Connecticut, 06520 · United States

  • Stamford HospitalRecruiting

    Stamford, Connecticut, 06902 · United States

  • Baptist MD Anderson Cancer CenterRecruiting

    Jacksonville, Florida, 32207 · United States

  • DH Cancer Research Center LLCRecruiting

    Margate, Florida, 33063 · United States

  • University Of MiamiRecruiting

    Miami, Florida, 33136 · United States

  • Hope And Healing CareRecruiting

    Hinsdale, Illinois, 60521 · United States

  • Pontchartrain Cancer CenterRecruiting

    Covington, Louisiana, 70433 · United States

  • Mayo Clinic RochesterRecruiting

    Rochester, Minnesota, 55905 · United States

  • University Health Kansas CityRecruiting

    Kansas City, Missouri, 64108 · United States

Terms used on this page · explanations by MedicalUpdates.ai

informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
cohort
A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
corticosteroid
Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
prior treatment
Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
stable dose
A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
screening period
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
relapse
A condition that improved with treatment and has since returned or worsened.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
refractory
A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
immunosuppressant
A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
Key Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Key Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
safety follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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