Primary Immune Thrombocytopenia (ITP)Phase 2VAY2EXPLORENCT07421167
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 study testing ianalumab and thrombopoietin receptor agonist (TPO-RA) in people with Primary Immune Thrombocytopenia (ITP) and Primary Evans Syndrome (ES), run by Novartis Pharmaceuticals at 19 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
- What is being tested?
- ianalumab and thrombopoietin receptor agonist (TPO-RA)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 164 participants
- Where
- 19 sitesUnited States
- Study dates
- 4 Aug 2026to 31 Aug 2030
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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)
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- 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
Trial locations and contacts
19 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
19 sites in 1 country
United States19 sites, 19 recruiting
- Recruiting
Intermountain Healthcare
Billings, Montana, 59102
Cyndi Bowman, cyndi.bowman@imail.org
Patrick Cobb
- Recruiting
Ironwood Cancer and Research Centers
Chandler, Arizona, 85224
Ashley Mercer, 480-821-2838, Ashley.mercer@ironwoodcrc.com
Anu Batra
- Recruiting
Pontchartrain Cancer Center
Covington, Louisiana, 70433
McKenna Chaney, mckenna@pcclouisiana.com
David Oubre
- Recruiting
Astera Cancer Center
East Brunswick, New Jersey, 08816
Carina Cedeno, Carina.cedeno@asterahealthcare.org
Anem Waheed
- Recruiting
Highlands Oncology Group
Fayetteville, Arkansas, 72703
Connor Ray, +1 479 587 1700, JRay@hogonc.com
Joseph Thaddeus Beck
- Recruiting
Hope And Healing Care
Hinsdale, Illinois, 60521
Aparanjitha Eluri, aeluri@hopenheal.care
Srilata Gundala
- Recruiting
Baptist MD Anderson Cancer Center
Jacksonville, Florida, 32207
Seth Waller, 614-657-1463, seth.waller@bmcjax.com
Jorge Antunez de Mayolo
- Recruiting
University Health Kansas City
Kansas City, Missouri, 64108
Richard Dugan-Starr, Richard.Dugan-Starr@uhkc.org
Anuj Shrestha
- Recruiting
DH Cancer Research Center LLC
Margate, Florida, 33063
Maria Pena, mpena@nrcinc.com
Mazyar Rouhani
- Recruiting
University Of Miami
Miami, Florida, 33136
Avinash Gogineni, 305-243-1169, axg1630@med.miami.edu
Gerald Soff
- Recruiting
Yale Cancer Center
New Haven, Connecticut, 06520
Ria Syam, ria.syam@yale.edu
Yevgeniya Foster
- Recruiting
Western Pennsylvania Hospital
Pittsburgh, Pennsylvania, 15224
Brandy Ince, 412-578-5000, brandy.ince@ahn.org
Deep Shah
- Recruiting
Mayo Clinic Rochester
Rochester, Minnesota, 55905
Suzanne Hogue, +1 507 284 2467, Eastlund.Suzanne@mayo.edu
Alexandra Paula Wolanskyj-Spinner
- Recruiting
Utah Cancer Specialists
Salt Lake City, Utah, 84106
Emra Kazic, Ekazic@utahcancer.com
DiSean Kendall
- Recruiting
Avera Cancer Institute
Sioux Falls, South Dakota, 57106
Sydney Schultz, 605-322-3000, sydney.schultz@avera.org
Waqas Jehangir
- Recruiting
Stamford Hospital
Stamford, Connecticut, 06902
Tiffany Mariscal, tmariscal@stamhealth.org
Joshua Dower
- Recruiting
Summit Medical Group
Summit, New Jersey, 07901
Michelle Mackenzie, 973-436-1755, mmackenzie@summithealth.com
Dipen Patel
- Recruiting
Montefiore Medical Center
The Bronx, New York, 10467
Akhil Aakunuri, 718-405-8427, aaakunuri@montefiore.org
Irina Murakhovskaya
- Recruiting
Harbor-UCLA Medical Center
Torrance, California, 90502
Abisay Ortega, 310-974-9334, abisay.ortega@lundquist.org
Sarah Tomassetti
About this study
Official title: 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.
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.
- Being tested
- ianalumab, thrombopoietin receptor agonist (TPO-RA)
- Keywords
- Immune thrombocytopenia (ITP), Evans syndrome (ES), Ianalumab, VAY736, B-cell depletion, B-cell Activating Factor Receptor (BAFF-R)
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic, Evans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ianalumabBiological
Also called VAY736
9 mg per kilogram infusion every 4 weeks (Q4W) for 16 weeks
thrombopoietin receptor agonist (TPO-RA)Drug
Also called eltrombopag, avatrombopag, romiplostim
IC TPO-RAs will be administered according to the respective United States Prescribing Information (USPIs)
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: 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). Receives: Biological: ianalumab; Drug: thrombopoietin receptor agonist (TPO-RA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
(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.
Measured: Up to Week 16
Secondary outcomes (4)
(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.
Measured: 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
Platelet count will be measured as part of routine hematology safety assessments
Measured: 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)
Platelet count will be measured as part of routine hematology safety assessments
Measured: 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
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.
Measured: From Week 17 Day 1 (W17D1) through Week 33 Day 1 (W33D1).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Feb 2026
- Study start4 Aug 2026
- Primary completion18 Jan 2029 (estimated)
- Study completion31 Aug 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immune Thrombocytopenia (ITP)Studies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 164 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07421167
- Sponsor's protocol ID
- CVAY736Q1US01
- Acronym
- VAY2EXPLORE
- Status last verified
- Oct 2026
- First submitted
- 12 Feb 2026
- Last updated
- 22 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- 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.