ClinicalTrials.gg
RecruitingNCT07421167VAY2EXPLOREUpdated Nov 6, 2026

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)

A Phase 2 interventional study of ianalumab and thrombopoietin receptor agonist (TPO-RA) in Primary Immune Thrombocytopenia (ITP) and Primary Evans Syndrome (ES), sponsored by Novartis Pharmaceuticals. Recruiting at 23 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-11-06.

Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 2, Interventional, and Treatment

From the registry’s dates

  • Started Aug 2026; still recruiting 3 months later.
Updated Nov 6, 202610 sites addedGo to Updates ↓
Phase
Phase 2
Study type
Interventional
Enrollment
164
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study 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.

Read the 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.

02

Conditions studied

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

Keywords

  • Immune thrombocytopenia (ITP)
  • Evans syndrome (ES)
  • Ianalumab
  • VAY736
  • B-cell depletion
  • B-cell Activating Factor Receptor (BAFF-R)
03

In context

Purpura, Thrombocytopenic, Idiopathic

503 studies on the registry are indexed under Purpura, Thrombocytopenic, Idiopathic; 158 are open to participants now.

This study's planned enrollment of 164 is above the median of 58 across 376 interventional studies indexed under Purpura, Thrombocytopenic, Idiopathic.

Browse Purpura, Thrombocytopenic, Idiopathic studies →

Lead sponsor

Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.

Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Key Inclusion Criteria:

  • Signed 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 treatment or IVIG treatment but the response was not sustained (response is defined as a 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 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 × (ULN)
    • (AST) > 3.0 × ULN
    • Alanine aminotransferase (ALT) > 3.0 × ULN
    • Immunoglobulin G (IgG) \< 5 g/L
    • ITP cohort only: \< 10 g/L, total > 1.5 × ULN
  • Patients with significantly compromised liver disease ( 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 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 to any of the study treatments or its or to drugs of similar chemical classes.
  • Known history of primary or secondary immunodeficiency, or a positive (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 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
Terms in these criteria, in plain language(14)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

Autoimmune disease

A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.

Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

Hemoglobin (Hb)

The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).

Bilirubin

A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.

Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.

Child-Pugh class

A score of how well the liver works in people with liver disease such as cirrhosis: class A is mild, B moderate and C severe.

Relapsed and refractory

Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

Excipients

The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Immunosuppression

A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.

Read whether the line is about a condition or about medicines, and which medicines and doses it means.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
164 participants (estimated)

Study arms

  • 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 · Drug: thrombopoietin receptor agonist (TPO-RA)

  • 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 · Drug: thrombopoietin receptor agonist (TPO-RA)

Interventions

  • Biologicalianalumab

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

    Also known as: VAY736

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

    Also known as: eltrombopag, avatrombopag, romiplostim

06

What researchers measure

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.

    Time frame: Up to Week 16

Secondary outcomes

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

    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.

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

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

    Platelet count will be measured as part of routine hematology safety assessments

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

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

    Platelet count will be measured as part of routine hematology safety assessments

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

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

    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.

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

07

Study locations

23 of 23 sites recruiting
08

References and documents

Individual participant data

Plan to share: Yes — 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.

No publications or documents are linked to this record.

09

Timeline

8 registry updates since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Started Aug 2026 (actual). Primary completion is estimated for Jan 2029, 29 months after the start.

Of 66 phase 2 interventional studies under Purpura, Thrombocytopenic, Idiopathic, half reached primary completion within 34 months of starting (the median), counting those still running; a quarter within 17 and three quarters within 41. This study's planned 29 months is below that.

How these figures are worked out, and which studies are left out of them.

Registered
Feb 12, 2026
First posted
Feb 19, 2026
StartActual
Aug 4, 2026
Primary completionEst.
Jan 18, 2029
CompletionEst.
Aug 31, 2030
Last update
Nov 6, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Sites
10 sites added
Show 10 added (10 United States)
  • Yale Cancer Center · New Haven, United States
  • Astera Cancer Center · East Brunswick, United States
  • University Health Kansas City · Kansas City, United States
  • Western Pennsylvania Hospital · Pittsburgh, United States
  • Harbor-UCLA Medical Center · Torrance, United States
  • Ironwood Cancer and Research Centers · Chandler, United States
  • Advent Health Orlando · Orlando, United States
  • Duke University Medical Center · Durham, United States
  • Hospital of the University of Pennsylvania Health System · Philadelphia, United States
  • JPS health Network · Fort Worth, United States
across 7 updates, Sep 29, 2026 – Nov 6, 2026
Site contacts
Changed at 7 sites. See the sites
Show 7 sites
  • Summit Medical Group · Summit, United StatesContact person changed (no phone or email listed)
  • Yale Cancer Center · New Haven, United StatesContact r•••@yale.edu→B•••@yale.edu
  • University Of Miami · Miami, United StatesContact a•••@med.miami.edu · •••1169→t•••@med.miami.edu · •••1169
  • Hope And Healing Care · Hinsdale, United StatesContact a•••@hopenheal.care→a•••@hopenheal.care
  • Mayo Clinic Rochester · Rochester, United StatesContact E•••@mayo.edu · •••2467→l•••@mayo.edu · •••2467
  • University Health Kansas City · Kansas City, United StatesContact k•••@uhkc.org→R•••@uhkc.org
  • Avera Cancer Institute · Sioux Falls, United StatesContact c•••@avera.org · •••3000→s•••@avera.org · •••3000
across 8 updates, Sep 29, 2026 – Nov 6, 2026
Show all 8 updates
  1. Nov 6, 2026
    3 sites added
    Show 3 added (3 United States)
    • Duke University Medical Center · Durham, United States
    • Hospital of the University of Pennsylvania Health System · Philadelphia, United States
    • JPS health Network · Fort Worth, United States
    Site contacts Changed at 1 site. See the sites
    Show the site
    • Yale Cancer Center · New Haven, United StatesContact r•••@yale.edu→B•••@yale.edu
  2. Nov 3, 2026
    1 site added
    Show site
    • Advent Health Orlando · Orlando, United States
    Site contacts Changed at 3 sites. See the sites
    Show 3 sites
    • University Of Miami · Miami, United StatesContact a•••@med.miami.edu · •••1169→t•••@med.miami.edu · •••1169
    • Hope And Healing Care · Hinsdale, United StatesContact a•••@hopenheal.care→a•••@hopenheal.care
    • Mayo Clinic Rochester · Rochester, United StatesContact E•••@mayo.edu · •••2467→l•••@mayo.edu · •••2467
    + 1 other change: verification date
  3. Oct 22, 2026
    1 site added
    Show site
    • Ironwood Cancer and Research Centers · Chandler, United States
    Site contacts Changed at 3 sites. See the sites
    Show 3 sites
    • Yale Cancer Center · New Haven, United StatesContact B•••@yale.edu→r•••@yale.edu
    • University Of Miami · Miami, United StatesContact a•••@miami.edu · •••1169→a•••@med.miami.edu · •••1169
    • Mayo Clinic Rochester · Rochester, United StatesContact l•••@mayo.edu · •••2467→E•••@mayo.edu · •••2467
  4. Oct 15, 2026
    1 site added
    Show site
    • Harbor-UCLA Medical Center · Torrance, United States
    Site contacts Changed at 3 sites. See the sites
    Show 3 sites
    • Yale Cancer Center · New Haven, United StatesContact r•••@yale.edu→B•••@yale.edu
    • University Of Miami · Miami, United StatesContact t•••@med.miami.edu · •••1169→a•••@miami.edu · •••1169
    • Hope And Healing Care · Hinsdale, United StatesContact a•••@hopenheal.care→a•••@hopenheal.care
  5. Oct 14, 2026
    1 site added
    Show site
    • Western Pennsylvania Hospital · Pittsburgh, United States
    Site contacts Changed at 4 sites. See the sites
    Show 4 sites
    • University Of Miami · Miami, United StatesContact r•••@miami.edu · •••1169→t•••@med.miami.edu · •••1169
    • Hope And Healing Care · Hinsdale, United StatesContact a•••@hopenheal.care→a•••@hopenheal.care
    • University Health Kansas City · Kansas City, United StatesContact k•••@uhkc.org→R•••@uhkc.org
    • Avera Cancer Institute · Sioux Falls, United StatesContact c•••@avera.org · •••3000→s•••@avera.org · •••3000
  6. Oct 9, 2026
    Site contacts Changed at 5 sites. See the sites
    Show 5 sites
    • Yale Cancer Center · New Haven, United StatesContact l•••@yale.edu→r•••@yale.edu
    • University Of Miami · Miami, United StatesContact a•••@miami.edu · •••1169→r•••@miami.edu · •••1169
    • Mayo Clinic Rochester · Rochester, United StatesContact E•••@mayo.edu · •••2467→l•••@mayo.edu · •••2467
    • University Health Kansas City · Kansas City, United StatesContact R•••@uhkc.org→k•••@uhkc.org
    • Avera Cancer Institute · Sioux Falls, United StatesContact s•••@avera.org · •••3000→c•••@avera.org · •••3000
    + 1 other change: verification date
  7. Oct 2, 2026
    1 site added
    Show site
    • University Health Kansas City · Kansas City, United States
    Site contacts Changed at 4 sites. See the sites
    Show 4 sites
    • Yale Cancer Center · New Haven, United StatesContact r•••@yale.edu→l•••@yale.edu
    • University Of Miami · Miami, United StatesContact t•••@med.miami.edu · •••1169→a•••@miami.edu · •••1169
    • Hope And Healing Care · Hinsdale, United StatesContact a•••@hopenheal.care→a•••@hopenheal.care
    • Mayo Clinic Rochester · Rochester, United StatesContact l•••@mayo.edu · •••2467→E•••@mayo.edu · •••2467
  8. Sep 29, 2026
    2 sites added
    Show 2 added (2 United States)
    • Yale Cancer Center · New Haven, United States
    • Astera Cancer Center · East Brunswick, United States
    Site contacts Changed at 1 site. See the sites
    Show the site
    • Summit Medical Group · Summit, United StatesContact person changed (no phone or email listed)

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT07421167
Responsible party
Sponsor
First posted
Feb 19, 2026
Start date
Aug 4, 2026
Primary completion
Jan 18, 2029 (estimated)
Completion
Aug 31, 2030 (estimated)
Last update
Nov 6, 2026

Study contacts

Novartis Pharmaceuticals
Contact
novartis.email@novartis.com
1-888-669-6682
Novartis Pharmaceuticals
Contact
novartis.email@novartis.com
Novartis Pharmaceuticals
study director · Novartis Pharmaceuticals

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Interested in this study?

Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.

Contact study team

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Studies of the drugs tested here: Ianalumab · Thrombopoietin Receptor Agonist

Start the discussion