Primary Immune ThrombocytopeniaPhase 3VAYHIT2NCT05653219
A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids
A Phase 3 study testing Ianalumab and Eltrombopag in people with Primary Immune Thrombocytopenia, run by Novartis Pharmaceuticals at 74 sites in 24 countries.
Trial summary
The purpose of this study is to evaluate the effect of two different doses of ianalumab added to eltrombopag to prolong Time to Treatment Failure (TTF) in adults with primary ITP who failed previous first-line treatment with steroids. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Primary Immune Thrombocytopenia
- What is being tested?
- Ianalumab and Eltrombopag
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 152 participants
- Where
- 74 sitesin 24 countries
- Study dates
- 2 Feb 2023to 8 Apr 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female patients aged 18 years and older on the day of signing the informed consent.
- A signed informed consent must be obtained prior to participation in the study.
- A diagnosis of primary ITP, with insufficient response to, or relapse after a first-line corticosteroid therapy ± IVIG.
- Patient with platelet count <30G/L (whom eltrombopag is clinically indicated as per physician's discretion) and with no contraindication to receive eltrombopag
Who cannot join
- ITP patients who received second-line ITP treatments (other than steroid therapy± IVIG) including splenectomy. However, patients exposed to thrombopoietin receptor agonists (TPO-RAs) for a limited time (max one week) before screening are eligible.
- Patients with key lab abnormalities and patients with Evans syndrome or any other cytopenia, (patients with low grade anemia related to bleeding or iron deficiency are eligible).
- Patients with history of clinically significant hematological disorders, or with marked altered hematologic parameters
- Patients with current or history of life-threatening bleeding
- Patient that are Human Immunodeficiency Virus (HIV), Hepatitis C Virus (HCV), Hepatitis B surface Antigen (HBsAg)/ Hepatitis B core antibody (HBcAb)-positive. HBcAb-positive patients can be enrolled if HBsAg negative, HBV DNA negative, no pre-existing liver fibrosis is present and antiviral prophylaxis is given
- Patients with known active or uncontrolled infection requiring systemic treatment during screening period
Show all 10Show fewer
- Patients with hepatic impairment
- Patients with concurrent coagulation disorders and/or receiving antiplatelet or anticoagulant medication with an exemption of low dose of acetylsalicylic acid (≤150 mg daily)
- Nursing (breast feeding) or pregnant women
- Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
74 sites in 24 countries.
74 sites in 24 countries
United States9 sites
University of Colorado Anschutz
Aurora, Colorado, 80045
St Vincent Frontier Cancer Center
Billings, Montana, 59102
Boston Medical Center
Boston, Massachusetts, 02118
NorthShore University Health System
Evanston, Illinois, 60201
Michigan Center of Medical Research
Farmington Hills, Michigan, 48334
Hematology Oncology Association of Rockland
Nyack, New York, 10960
Montefiore Medical Center
The Bronx, New York, 10461
UMASS Memorial Medical Center
Worcester, Massachusetts, 01665
Yuma Regional Medical Center
Yuma, Arizona, 85349
China7 sites
Novartis Investigative Site
Beijing, 100730
Novartis Investigative Site
Binzhou, Shandong, 256603
Novartis Investigative Site
Guangzhou, Guangdong, 510515
Novartis Investigative Site
Hangzhou, Zhejiang, 310003
Novartis Investigative Site
Jinan, 250012
Novartis Investigative Site
Tianjin, 300020
Novartis Investigative Site
Wuhan, Hubei, 430022
Malaysia6 sites
Novartis Investigative Site
George Town, Pulau Pinang, 10450
Novartis Investigative Site
George Town, 10050
Novartis Investigative Site
Johor Bahru, 80100
Novartis Investigative Site
Kota Kinabalu, Sabah, 88586
Novartis Investigative Site
Kuala Selangor, 68000
Novartis Investigative Site
Subang Jaya, Selangor, 47500
Japan5 sites
Novartis Investigative Site
Kofu, Yamanashi, 400-8506
Novartis Investigative Site
Kumamoto, 860-0008
Novartis Investigative Site
Nagoya, Aichi-ken, 453-8511
Novartis Investigative Site
Osaka, Osaka, 5400006
Novartis Investigative Site
Suita, Osaka, 5650871
Turkey (Türkiye)5 sites
Novartis Investigative Site
Ankara, Bilkent Cankaya, 06800
Novartis Investigative Site
Aydin, Efeler, 09100
Novartis Investigative Site
Istanbul, Fatih, 34098
Novartis Investigative Site
Izmir, 35100
Novartis Investigative Site
Samsun, Atakum, 55200
Germany4 sites
Novartis Investigative Site
Cologne, North Rhine-Westphalia, 50937
Novartis Investigative Site
Dresden, Saxony, 01307
Novartis Investigative Site
Dresden, 01307
Novartis Investigative Site
Jena, Thuringia, 07740
Italy4 sites
Novartis Investigative Site
Bologna, BO, 40138
Novartis Investigative Site
Roma, RM, 00133
Novartis Investigative Site
Roma, RM, 00168
Novartis Investigative Site
Trieste, TS, 34129
Czechia3 sites
Novartis Investigative Site
Brno, 625 00
Novartis Investigative Site
Prague, 100 34
Novartis Investigative Site
Prague, 128 08
Mexico3 sites
Novartis Investigative Site
Monterrey, Nuevo León, 64460
Novartis Investigative Site
Morelia, Michoacán, 58260
Novartis Investigative Site
Saltillo, Coahuila, 25230
United Kingdom3 sites
Novartis Investigative Site
London, E1 1BB
Novartis Investigative Site
London, W12 0HS
Novartis Investigative Site
Oxford, OX3 7LE
Belgium2 sites
Novartis Investigative Site
Roeselare, West-Vlaanderen, 8800
Novartis Investigative Site
Yvoir, Namur, 5530
France2 sites
Novartis Investigative Site
Blois, 41000
Novartis Investigative Site
Le Mans, 72000
Hungary2 sites
Novartis Investigative Site
Budapest, 1083
Novartis Investigative Site
Debrecen, Hajdu Bihar Megye, 4032
India2 sites
Novartis Investigative Site
Kolkata, West Bengal, 700014
Novartis Investigative Site
Rishikesh, Uttarakhand, 249203
Netherlands2 sites
Novartis Investigative Site
Leiden, South Holland, 2333 ZA
Novartis Investigative Site
Utrecht, 3584 CX
Philippines2 sites
Novartis Investigative Site
Makati City, 1229
Novartis Investigative Site
Quezon, 1102
Romania2 sites
Novartis Investigative Site
Craiova, Dolj, 200136
Novartis Investigative Site
Timișoara, 300079
Singapore2 sites
Novartis Investigative Site
Singapore, 119074
Novartis Investigative Site
Singapore, 169608
South Korea2 sites
Novartis Investigative Site
Jeollanam, 519763
Novartis Investigative Site
Seoul, 06591
Spain2 sites
Novartis Investigative Site
Salamanca, 37007
Novartis Investigative Site
Santiago Compostela, A Coruna, 15706
Thailand2 sites
Novartis Investigative Site
Bangkok, 10700
Novartis Investigative Site
Chiang Mai, 50200
About this study
Official title: A Phase 3 Randomized, Double-blind Study of Ianalumab (VAY736) Versus Placebo in Addition to Eltrombopag in Patients With Primary Immune Thrombocytopenia (ITP) Who Had an Insufficient Response or Relapsed After First Line Steroid Treatment (VAYHIT2)
The purpose of this study is to evaluate the effect of two different doses of ianalumab added to eltrombopag to prolong Time to Treatment Failure (TTF) in adults with primary ITP who failed previous first-line treatment with steroids.
Read the detailed description
This is a multicenter, randomized, double-blinded phase 3 study to assess efficacy and safety of two different doses of ianalumab versus placebo in addition to eltrombopag in adults with primary ITP (platelet count <30 G/L) who failed previous first-line treatment with corticosteroids.
After completion of the screening period, the participants will enter the randomized treatment period (ianalumab/placebo with eltrombopag) followed by the eltrombopag tapering period. Afterwards, all participants will enter the follow-up period to be monitored for efficacy and safety or safety only depending on how the participants responded to the study treatment.
- Conditions
- Primary Immune Thrombocytopenia
- Being tested
- Ianalumab, Eltrombopag, Placebo
- Keywords
- Primary immune thrombocytopenia (ITP),, ianalumab, VAY736, B-cell depletion, B-cell Activating Factor Receptor (BAFF-R) blockade, eltrombopag
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IanalumabBiological
Also called VAY736
Concentrate for solution for infusion for intravenous use
EltrombopagDrug
Also called ETB115
Film-coated tablet for oral use
PlaceboDrug
Concentrate for solution for infusion for intravenous use.
| Study group | Role | What they receive |
|---|---|---|
| Treatment arm 1 - Lower dose | Experimental | Participants will receive eltrombopag and ianalumab lower dose Receives: Biological: Ianalumab; Drug: Eltrombopag |
| Treatment arm 2 - Higher dose | Experimental | Participants will receive eltrombopag and ianalumab higher dose Receives: Biological: Ianalumab; Drug: Eltrombopag |
| Treatment arm 3 - Placebo | Placebo comparator | Participants will receive eltrombopag and placebo Receives: Drug: Eltrombopag; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time from randomization until treatment failure
Time from randomization until treatment failure is defined as the time from randomization date until the first of the following events indicative of treatment failure:
- platelet count below 30 G/L
- start of a new ITP treatment
- need for a rescue treatment
- ineligibility to taper or inability to discontinue eltrombopag
- death
Measured: Randomization to until end of study (up to 39 months after randomization of last participant)
Secondary outcomes (25)
Stable response at 6 months (Key Secondary Endpoint)
Percentage of participants with at least 3 platelet count collected at month 6 (between study days 121 and 183 and at least 75% of platelet counts qualified as a response).
Measured: At 6 months
Complete Response rate at each timepoint
Percentage of participants with any platelet count of at least 100 G/L in the absence of rescue treatment or new ITP treatment
Measured: Randomization to until end of study (up to 39 months after randomization of last participant)
Response rate at each timepoint
Percentage of participants with any platelet count of at least 50 G/L in the absence of rescue treatment or new ITP treatment
Measured: Randomization to until end of study (up to 39 months after randomization of last participant)
Best response rate across all timepoints
Percentage of participants with a best response rate of either response or complete response
Measured: Randomization to until end of study (up to 39 months after randomization of last participant)
Time to first response/time to first complete response
Time from randomization to date of first response and time from randomization to date of first complete response
Measured: Time from randomization up to the longest observed treatment period duration
Duration of response
Time from achievement of response to treatment failure.
Measured: Randomization to until end of study (up to 39 months after randomization of last participant)
Stable response at 1 year
Percentage of participants with at least 2 platelet count collected at year 1 (between study days 296 and 379 and at least 66% of platelet counts qualified as a response).
Measured: At 1 year
Duration of complete response
Time from achievement of complete response to loss of complete response stable response at 1 year period
Measured: Randomization to end of study (up to 39 months after randomization of last participant)
Rate of participants who successfully taper and discontinue eltrombopag in each treatment arm
Probability to be treatment failure-free (as defined for the primary efficacy endpoint)
Measured: up to week 24
Percentage of participants with bleeding events according to World Health Organization (WHO)
Percentage of participants reporting bleeding events according to WHO bleeding scale
Measured: Randomization to until end of study (up to 39 months after randomization of last participant)
Number of participants receiving rescue treatment
Number of participants who are in need of rescue treatment in each treatment arm
Measured: Randomization to until end of study (up to 39 months after randomization of last participant)
Percentage of participants receiving rescue treatment
Percentage of participants who are in need of rescue treatment
Measured: Randomization to until end of study (up to 39 months after randomization of last participant)
Change from baseline in the frequency of CD19+ B-cell counts
Post-baseline frequency of CD19+ B-cell counts (percentage within CD45) compared to baseline
Measured: Randomization to until end of study (up to 39 months after randomization of last participant)
Change from baseline in the absolute number of CD19+ B-cell counts
Post-baseline absolute number of CD19+ B-cell counts compared to baseline
Measured: Randomization to until end of study (up to 39 months after randomization of last participant)
Change from baseline on T-score of the PROMIS SF v1.0 Fatigue 13a
The Patient-Reported Outcomes Measurement Information System (PROMIS) Short Form v1.0 Fatigue 13a includes 13 items that assess fatigue in adults.
Measured: From screening (baseline) until end of study (up 39 months after randomization of last participant)
Change from baseline in ITP PAQ domain scores of symptoms, fatigue, bother (uncomfortable), activity
The ITP-PAQ is a 44 item scale for measuring HRQoL in adults with ITP across ten scales: Symptoms, Bother-Physical Health, Fatigue/Sleep, Activity, Fear, Psychological Health, Work, Social Activity, Women´s Reproductive Health, overall QoL. Each item is rated on a Likert type scale. Each scale is scored from 0 to 100. Higher scores represent better HRQoL.
Measured: From screening (baseline) until end of study (up 39 months after randomization of last participant)
Time to first occurence of B-cell recovery defined as ≥80% of baseline ≥50 cells/µL
Time to B-cell recovery defined as ≥80% of baseline or ≥50 cells/µL
Measured: Randomization to until end of study (up to 39 months after randomization of last participant)
Change from baseline in immunoglobulins
Change from baseline in immunoglobulin levels
Measured: Randomization to until end of study (up to 39 months after randomization of last participant)
PK parameters: AUClast
AUClast: Area under the curve from time zero to the last measurable concentration sampling time (tlast)
Measured: After first dose (pre-dose, EOI, 168, 336 and 504 hours post dose) and after last dose (pre-dose, EOI, 336, 672, 2016, 3360 hours post dose)
PK parameters: AUCtau
AUCtau: Area under the curve calculated to the end of a dosing interval (tau)
Measured: After first dose (pre-dose, EOI, 168, 336 and 504 hours post dose) and after last dose (pre-dose, EOI, 336, 672, 2016, 3360 hours post dose)
PK parameters: Cmax
Maximum (peak) observed plasma, blood, serum or other body fluid drug concentration
Measured: After first dose (pre-dose, EOI, 168, 336 and 504 hours post dose) and after last dose (pre-dose, EOI, 336, 672, 2016, 3360 hours post dose)
PK parameters: Tmax
Time to reach maximum (peak) observed plasma, blood, serum or other body fluid drug concentration
Measured: After first dose (pre-dose, EOI, 168, 336 and 504 hours post dose) and after last dose (pre-dose, EOI, 336, 672, 2016, 3360 hours post dose)
PK parameters: Accumulation ratio Racc
Accumulation ratio calculated using AUC values obtained after the last and first dose
Measured: After last dose (pre-dose, EOI, 336, 672, 2016, 3360 hours post dose)
Incidence of anti-ianalumab antibodies in serum (ADA assay) over time
Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants assessing the immunogenicity of ianalumab
Measured: up to week 33
Titer of anti-ianalumab antibodies in serum (ADA assay) over time
Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants assessing the immunogenicity of ianalumab
Measured: up to week 33
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Dec 2022
- Study start2 Feb 2023
- Primary completion19 Jun 2025
- Study completion8 Apr 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Immune ThrombocytopeniaStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 152 participants (actual)
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
- NCT05653219
- Sponsor's protocol ID
- CVAY736Q12301
- Other IDs
- 2024-512890-28-00
- Acronym
- VAYHIT2
- Status last verified
- Aug 2026
- First submitted
- 23 Nov 2022
- Last updated
- 21 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- 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
Publications
Cuker A, Stauch T, Cooper N, Al-Samkari H, Michel M, Ghanima W, Urban P, Fronczek J, Foster M, Weill M, Zhang L, Hou M, Zander T, Sharif A, Sun J, Nath UK, Schutgens R, Rossi E, Deleu L, Cervinek L, Yoon JH, Chang H, Ruchutrakool T, Iino M, Goto T, Zaja F; VAYHIT2 Investigators. Ianalumab plus Eltrombopag in Immune Thrombocytopenia. N Engl J Med. 2026 Apr 16;394(15):1503-1513. doi: 10.1056/NEJMoa2515168. Epub 2025 Dec 9.
PubMed 41363800
Study at a glance
From the registry record, last updated by the sponsor on 21 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Randomized, Double-blind Study of Ianalumab (VAY736) Versus Placebo in Addition to Eltrombopag in Patients With Primary Immune Thrombocytopenia (ITP) Who Had an Insufficient Response or Relapsed After First Line Steroid Treatment (VAYHIT2)
- ClinicalTrials.gov ID
- NCT05653219
- Acronym / sponsor ID
- VAYHIT2 / CVAY736Q12301
- Conditions
- Primary Immune Thrombocytopenia
- Keywords
- Primary immune thrombocytopenia (ITP),, ianalumab, VAY736, B-cell depletion, B-cell Activating Factor Receptor (BAFF-R) blockade, eltrombopag
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 152 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 2 Feb 2023 (actual)
- Primary completion
- 19 Jun 2025 (actual)
- Study completion
- 8 Apr 2028 (estimated)
- First posted
- 16 Dec 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the effect of two different doses of ianalumab added to eltrombopag to prolong Time to Treatment Failure (TTF) in adults with primary ITP who failed previous first-line treatment with steroids.
Detailed description
This is a multicenter, randomized, double-blinded phase 3 study to assess efficacy and safety of two different doses of ianalumab versus placebo in addition to eltrombopag in adults with primary ITP (platelet count <30 G/L) who failed previous first-line treatment with corticosteroids.
After completion of the screening period, the participants will enter the randomized treatment period (ianalumab/placebo with eltrombopag) followed by the eltrombopag tapering period. Afterwards, all participants will enter the follow-up period to be monitored for efficacy and safety or safety only depending on how the participants responded to the study treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Treatment arm 1 - Lower dose | Experimental | Biological: Ianalumab, Drug: EltrombopagParticipants will receive eltrombopag and ianalumab lower dose |
| Treatment arm 2 - Higher dose | Experimental | Biological: Ianalumab, Drug: EltrombopagParticipants will receive eltrombopag and ianalumab higher dose |
| Treatment arm 3 - Placebo | Placebo comparator | Drug: Eltrombopag, Drug: PlaceboParticipants will receive eltrombopag and placebo |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Male or female patients aged 18 years and older on the day of signing the informed consent.
- A signed informed consent must be obtained prior to participation in the study.
- A diagnosis of primary ITP, with insufficient response to, or relapse after a first-line corticosteroid therapy ± IVIG.
- Patient with platelet count <30G/L (whom eltrombopag is clinically indicated as per physician's discretion) and with no contraindication to receive eltrombopag
Key exclusion criteria (10 listed)
- ITP patients who received second-line ITP treatments (other than steroid therapy± IVIG) including splenectomy. However, patients exposed to thrombopoietin receptor agonists (TPO-RAs) for a limited time (max one week) before screening are eligible.
- Patients with key lab abnormalities and patients with Evans syndrome or any other cytopenia, (patients with low grade anemia related to bleeding or iron deficiency are eligible).
- Patients with history of clinically significant hematological disorders, or with marked altered hematologic parameters
- Patients with current or history of life-threatening bleeding
- Patient that are Human Immunodeficiency Virus (HIV), Hepatitis C Virus (HCV), Hepatitis B surface Antigen (HBsAg)/ Hepatitis B core antibody (HBcAb)-positive. HBcAb-positive patients can be enrolled if HBsAg negative, HBV DNA negative, no pre-existing liver fibrosis is present and antiviral prophylaxis is given
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IanalumabAlso: VAY736Concentrate for solution for infusion for intravenous use | Biological | Treatment arm 1 - Lower dose, Treatment arm 2 - Higher dose |
| EltrombopagAlso: ETB115Film-coated tablet for oral use | Drug | Treatment arm 1 - Lower dose, Treatment arm 2 - Higher dose, Treatment arm 3 - Placebo |
| PlaceboConcentrate for solution for infusion for intravenous use. | Drug | Treatment arm 3 - Placebo |
Primary outcome
Time from randomization until treatment failure
Time frame: Randomization to until end of study (up to 39 months after randomization of last participant)
Time from randomization until treatment failure is defined as the time from randomization date until the first of the following events indicative of treatment failure: * platelet count below 30 G/L * start of a new ITP treatment * need for a rescue treatment * ineligibility to taper or inability to discontinue eltrombopag * death
25 secondary outcomes
Stable response at 6 months (Key Secondary Endpoint)
Time frame: At 6 months
Complete Response rate at each timepoint
Time frame: Randomization to until end of study (up to 39 months after randomization of last participant)
Response rate at each timepoint
Time frame: Randomization to until end of study (up to 39 months after randomization of last participant)
Best response rate across all timepoints
Time frame: Randomization to until end of study (up to 39 months after randomization of last participant)
Time to first response/time to first complete response
Time frame: Time from randomization up to the longest observed treatment period duration
Duration of response
Time frame: Randomization to until end of study (up to 39 months after randomization of last participant)
Stable response at 1 year
Time frame: At 1 year
Duration of complete response
Time frame: Randomization to end of study (up to 39 months after randomization of last participant)
Rate of participants who successfully taper and discontinue eltrombopag in each treatment arm
Time frame: up to week 24
Percentage of participants with bleeding events according to World Health Organization (WHO)
Time frame: Randomization to until end of study (up to 39 months after randomization of last participant)
Number of participants receiving rescue treatment
Time frame: Randomization to until end of study (up to 39 months after randomization of last participant)
Percentage of participants receiving rescue treatment
Time frame: Randomization to until end of study (up to 39 months after randomization of last participant)
Change from baseline in the frequency of CD19+ B-cell counts
Time frame: Randomization to until end of study (up to 39 months after randomization of last participant)
Change from baseline in the absolute number of CD19+ B-cell counts
Time frame: Randomization to until end of study (up to 39 months after randomization of last participant)
Change from baseline on T-score of the PROMIS SF v1.0 Fatigue 13a
Time frame: From screening (baseline) until end of study (up 39 months after randomization of last participant)
Change from baseline in ITP PAQ domain scores of symptoms, fatigue, bother (uncomfortable), activity
Time frame: From screening (baseline) until end of study (up 39 months after randomization of last participant)
Time to first occurence of B-cell recovery defined as ≥80% of baseline ≥50 cells/µL
Time frame: Randomization to until end of study (up to 39 months after randomization of last participant)
Change from baseline in immunoglobulins
Time frame: Randomization to until end of study (up to 39 months after randomization of last participant)
PK parameters: AUClast
Time frame: After first dose (pre-dose, EOI, 168, 336 and 504 hours post dose) and after last dose (pre-dose, EOI, 336, 672, 2016, 3360 hours post dose)
PK parameters: AUCtau
Time frame: After first dose (pre-dose, EOI, 168, 336 and 504 hours post dose) and after last dose (pre-dose, EOI, 336, 672, 2016, 3360 hours post dose)
PK parameters: Cmax
Time frame: After first dose (pre-dose, EOI, 168, 336 and 504 hours post dose) and after last dose (pre-dose, EOI, 336, 672, 2016, 3360 hours post dose)
PK parameters: Tmax
Time frame: After first dose (pre-dose, EOI, 168, 336 and 504 hours post dose) and after last dose (pre-dose, EOI, 336, 672, 2016, 3360 hours post dose)
PK parameters: Accumulation ratio Racc
Time frame: After last dose (pre-dose, EOI, 336, 672, 2016, 3360 hours post dose)
Incidence of anti-ianalumab antibodies in serum (ADA assay) over time
Time frame: up to week 33
Titer of anti-ianalumab antibodies in serum (ADA assay) over time
Time frame: up to week 33
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Aug 2026.
- Derived from this study: PubMed 41363800Cuker A, Stauch T, Cooper N, Al-Samkari H, Michel M, Ghanima W, Urban P, Fronczek J, Foster M, Weill M, Zhang L, Hou M, Zander T, Sharif A, Sun J, Nath UK, Schutgens R, Rossi E, Deleu L, Cervinek L, Yoon JH, Chang H, Ruchutrakool T, Iino M, Goto T, Zaja F; VAYHIT2 Investigators. Ianalumab plus Eltrombopag in Immune Thrombocytopenia. N Engl J Med. 2026 Apr 16;394(15):1503-1513. doi: 10.1056/NEJMoa2515168. Epub 2025 Dec 9.
- Individual participant dataSharing planned: 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
74 sites in 24 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ianalumab and Eltrombopag in people with Primary Immune Thrombocytopenia, run by Novartis Pharmaceuticals at 74 sites in 24 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 21 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 74 sites in United States, China, Malaysia, Japan, Turkey (Türkiye) and 19 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time from randomization until treatment failure (time frame: Randomization to until end of study (up to 39 months after randomization of last participant)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05653219, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Aug 2026.