Primary Immune Thrombocytopenia (ITP)Phase 3VAYHIT1NCT05653349
Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP)
A Phase 3 study testing Ianalumab and Corticosteroids in people with Primary Immune Thrombocytopenia (ITP), run by Novartis Pharmaceuticals at 111 sites in 25 countries.
Trial summary
The purpose of this study is to evaluate the effect of two different doses of ianalumab versus placebo in addition to first-line corticosteroids in increasing the rate of stable response off treatment at 12 months (SROT-12) in adult participants with primary ITP. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Primary Immune Thrombocytopenia (ITP)
- What is being tested?
- Ianalumab and Corticosteroids
- 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
- 226 participants
- Where
- 111 sitesin 25 countries
- Study dates
- 3 Feb 2023to 5 Jan 2029
- Phase
- Phase 3
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
- Signed informed consent prior to participation in the study.
- Male or female participants aged 18 years and older on the day of signing informed consent
- Primary ITP diagnosed within 3 months before initiating first-line ITP therapy (corticosteroids, IVIG)
- Platelet count below 30 G/L before starting any first-line ITP therapy (corticosteroids, IVIG)
- Response (platelet count >=50 G/L) to corticosteroids (+/- IVIG) at any time prior to randomization. Note: Platelet count measured within 7 days of platelet transfusion will not be considered as response.
Who cannot join
- Evans syndrome or any other cytopenia (patients with low grade anemia related to bleeding or iron deficiency are eligible)
- Current life-threatening bleeding
- Previous ITP treatment, including splenectomy, except for corticosteroids and/or IVIG initiated as first-line therapy for up to 28 days before randomization and rescue corticosteroids and/or IVIG given prior to confirmed diagnosis of primary ITP .
- Prior use of B-cell depleting therapy (e.g., rituximab).
- Absolute neutrophil count below 1.0 G/L at randomization
- Participants with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication with an exemption of low dose of acetylsalicylic acid
- Other protocol-defined Inclusion/Exclusion may apply.
Trial locations and contacts
111 sites in 25 countries.
111 sites in 25 countries
United States20 sites
University of Colorado Anschutz
Aurora, Colorado, 80045
Oncology Care Associates
Bethesda, Maryland, 20817
St Vincent Frontier Cancer Center
Billings, Montana, 59102
Massachusetts General Hospital
Boston, Massachusetts, 02114
Beth Israel Deaconess Med Center
Boston, Massachusetts, 02215
Uni of Chi Medi Ctr Hema and Onco
Chicago, Illinois, 60637
Community Cancer Institute
Clovis, California, 93611
STAT Research Inc
Dayton, Ohio, 45402
Michigan Center of Medical Research
Farmington Hills, Michigan, 48334
Parkview Research Center
Fort Wayne, Indiana, 46845
Compassionate Care Res Group Inc
Fountain Valley, California, 92708
Optum Health
Lake Success, New York, 11042
DH Cancer Research Center LLC
Margate, Florida, 33063
Hematology Oncology Association of Rockland
Nyack, New York, 10960
Community Cancer Trials of Utah
Ogden, Utah, 84405
Rhode Island Hospital
Providence, Rhode Island, 02903
Mayo Clinic Rochester
Rochester, Minnesota, 55905
Metro Minnesota CCOP
Saint Louis Park, Minnesota, 55416
Mays Cancer Ctr Uthsa Mdacc
San Antonio, Texas, 78229
Yuma Regional Medical Center
Yuma, Arizona, 85349
China13 sites
Novartis Investigative Site
Beijing, Beijing Municipality, 100044
Novartis Investigative Site
Bengbu, 233004
Novartis Investigative Site
Changzhou, Jiangsu, 213004
Novartis Investigative Site
Dalian, 116000
Novartis Investigative Site
Guangzhou, Guangdong, 510515
Novartis Investigative Site
Hangzhou, Zhejiang, 310006
Novartis Investigative Site
Kunming, Yunnan, 650101
Novartis Investigative Site
Luoyang, Henan, 471003
Novartis Investigative Site
Nanchang, Jiangxi, 330006
Novartis Investigative Site
Shenzhen, Guangdong, 518037
Novartis Investigative Site
Shijiazhuang, Hebei, 050000
Novartis Investigative Site
Tianjin, 300020
Novartis Investigative Site
Zhengzhou, Henan, 450003
Japan9 sites
Novartis Investigative Site
Chiba, 260-0852
Novartis Investigative Site
Fukuoka, 815-8555
Novartis Investigative Site
Gifu, 5008513
Novartis Investigative Site
Iruma-gun, Saitama, 3500495
Novartis Investigative Site
Nagakute, Aichi-ken, 480-1195
Novartis Investigative Site
Okayama, Okayama-ken, 7011192
Novartis Investigative Site
Osaka, 543-8555
Novartis Investigative Site
Sapporo, Hokkaido, 0608604
Novartis Investigative Site
Shibukawa, Gunma, 377-0280
Germany6 sites
Novartis Investigative Site
Dresden, Saxony, 01307
Novartis Investigative Site
Dresden, 01307
Novartis Investigative Site
Essen, 45147
Novartis Investigative Site
Frankfurt am Main, Hesse, 60590
Novartis Investigative Site
Giessen, 35392
Novartis Investigative Site
Jena, Thuringia, 07740
Spain6 sites
Novartis Investigative Site
Barcelona, 08035
Novartis Investigative Site
Madrid, 28009
Novartis Investigative Site
Madrid, 28046
Novartis Investigative Site
Murcia, 30008
Novartis Investigative Site
Salamanca, 37007
Novartis Investigative Site
Seville, 41013
Argentina5 sites
Novartis Investigative Site
Caba, C1039AAC
Novartis Investigative Site
CABA, Buenos Aires, C1280AEB
Novartis Investigative Site
CABA, Buenos Aires, C1414DRK
Novartis Investigative Site
Córdoba, 5000
Novartis Investigative Site
San Miguel Tucuman, Tucumán Province, T4000DPK
France5 sites
Novartis Investigative Site
Caen, 14033
Novartis Investigative Site
Chambéry, 73000
Novartis Investigative Site
Le Mans, 72000
Novartis Investigative Site
Lille, 59037
Novartis Investigative Site
Rennes, 35033
Italy5 sites
Novartis Investigative Site
Bologna, BO, 40138
Novartis Investigative Site
Florence, FI, 50134
Novartis Investigative Site
Roma, RM, 00168
Novartis Investigative Site
Trieste, TS, 34129
Novartis Investigative Site
Vicenza, VI, 36100
Australia4 sites
Novartis Investigative Site
Clayton, Victoria, 3168
Novartis Investigative Site
Garran, Australian Capital Territory, 2605
Novartis Investigative Site
Melbourne, Victoria, 3004
Novartis Investigative Site
Wooloongabba, Queensland, 4102
Belgium4 sites
Novartis Investigative Site
Brasschaat, 2930
Novartis Investigative Site
Bruges, 8000
Novartis Investigative Site
Leuven, 3000
Novartis Investigative Site
Roeselare, West-Vlaanderen, 8800
Malaysia4 sites
Novartis Investigative Site
George Town, 10050
Novartis Investigative Site
Johor Bahru, 80100
Novartis Investigative Site
Kuala Selangor, 68000
Novartis Investigative Site
Kuching, Sarawak, 93586
Czechia3 sites
Novartis Investigative Site
Ostrava, Poruba, 708 52
Novartis Investigative Site
Prague, 100 34
Novartis Investigative Site
Prague, 128 08
Thailand3 sites
Novartis Investigative Site
Bangkok, 10330
Novartis Investigative Site
Bangkok, 10700
Novartis Investigative Site
Chiang Mai, 50200
Turkey (Türkiye)3 sites
Novartis Investigative Site
Aydin, Efeler, 09100
Novartis Investigative Site
Istanbul, Fatih, 34098
Novartis Investigative Site
Samsun, Atakum, 55200
United Kingdom3 sites
Novartis Investigative Site
Birmingham, West Midlands, B15 2TH
Novartis Investigative Site
London, W12 0HS
Novartis Investigative Site
Nottingham, NG5 1PB
Austria2 sites
Novartis Investigative Site
Innsbruck, Tyrol, 6020
Novartis Investigative Site
Salzburg, 5020
Bulgaria2 sites
Novartis Investigative Site
Plovdiv, 4002
Novartis Investigative Site
Sofia, 1431
Hong Kong2 sites
Novartis Investigative Site
Hong Kong, 999077
Novartis Investigative Site
New Territories, Hong Kong, 999077
Hungary2 sites
Novartis Investigative Site
Budapest, H-1083
Novartis Investigative Site
Debrecen, Hajdu Bihar Megye, 4032
India2 sites
Novartis Investigative Site
Ahmedabad, Gujarat, 380013
Novartis Investigative Site
Rishikesh, Uttarakhand, 249203
Mexico2 sites
Novartis Investigative Site
Mexico City, Mexico City, 06720
Novartis Investigative Site
Monterrey, Nuevo León, 64460
Singapore2 sites
Novartis Investigative Site
Singapore, 119074
Novartis Investigative Site
Singapore, 169608
Vietnam2 sites
Novartis Investigative Site
Hanoi, 100000
Novartis Investigative Site
Ho Chi Minh City, 70000
About this study
Official title: A Phase III, Randomized, Double-blind Study of Ianalumab (VAY736) Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (VAYHIT1)
The purpose of this study is to evaluate the effect of two different doses of ianalumab versus placebo in addition to first-line corticosteroids in increasing the rate of stable response off treatment at 12 months (SROT-12) in adult participants with primary ITP.
Read the detailed description
This is a multi-center, randomized, double-blind Phase 3 study to assess the efficacy and safety of two different doses of ianalumab compared to placebo in adults with primary ITP (platelets count <30 G/L) who require first-line standard-of-care corticosteroids.
After completion of the screening period, the participants will enter the randomized treatment period (ianalumab/placebo with standard of care corticosteroids).
After the treatment period, all participants will enter the follow-up period to be monitored for efficacy and safety or safety only depending on how they respond to the study treatment.
The efficacy and safety follow-up period will last until treatment failure or up to 39 months after randomization of the last patient, whichever occurs first. Safety follow-up period will be performed for at least 20 weeks and up to 2 years after the last ianalumab/placebo dose.
The primary endpoint was updated in May 2026 to stable response off treatment at 12 months (SROT-12). The previous primary endpoint, time to treatment failure (TTF), remains in the study as a key secondary objective.
- Conditions
- Primary Immune Thrombocytopenia (ITP)
- Being tested
- Ianalumab, Placebo, Corticosteroids
- Keywords
- Primary immune thrombocytopenia (ITP), ianalumab, VAY736, B-cell depletion, B-cell Activating Factor Receptor (BAFF-R) blockade
- 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
Intravenous infusion, prepared from concentrate solution
PlaceboDrug
Intravenous infusion, prepared from matching placebo
CorticosteroidsDrug
Oral or parental (if clinically justified)
| Study group | Role | What they receive |
|---|---|---|
| Ianalumab Lower dose | Experimental | Lower dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified) Receives: Biological: Ianalumab; Drug: Corticosteroids |
| Ianalumab Higher dose | Experimental | Higher dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified) Receives: Biological: Ianalumab; Drug: Corticosteroids |
| Placebo | Placebo comparator | Placebo administered intravenously with corticosteroids oral or parental (if clinically justified) Receives: Drug: Placebo; Drug: Corticosteroids |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of Stable Response off treatment at 12 months (SROT-12)
A participant will be considered in SROT-12 if at least 75% of the platelet counts collected between visits Week 53 Day 1 and Week 65 Day 1 (Study Days 352 and 463) qualify as Response. Response is any platelet counts of at least 50 G/L in the absence of rescue treatment or new immune thrombocytopenia (ITP) treatment).
Measured: 12 months after last patient completed treatment
Secondary outcomes (25)
Time from randomization to Treatment Failure (TTF) (Key Secondary Endpoint)
Time to Treatment Failure (TTF) is defined as the time from randomization until the first of the following events/situation indicative of treatment failure: platelet count below 30 G/L later than 8 weeks from randomization, need for a rescue treatment later than 8 weeks from randomization, intake of rescue treatment (e.g., corticosteroids, Intravenous immunoglobulins (IVIG), or platelet transfusion) given later than 8 weeks from randomization, start of new Immune Thrombocytopenia (ITP) treatment whenever it occurs, death (whatever the cause).
Measured: Randomization to end of study (up to 39 months after randomization of last patient
Complete Response (CR) rate in each treatment group
Complete Response (CR) rate at each timepoint defined as the proportion 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 end of study (up to 39 months after randomization of last patient)
Response (R) rate in each treatment group
Response (R) rate at each timepoint defined as the proportion 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 end of study (up to 39 months after randomization of last patient)
Time to complete response in each treatment group
Time from randomization to date of first complete response.
Measured: Randomization to end of study (up to 39 months after randomization of last patient)
Duration of response in each treatment group
Time from achievement of complete response to loss of complete response
Measured: Randomization to end of study (up to 39 months after randomization of last patient)
Stable response at 6 months
Percentage of participants with at least 2 platelet count collected at month 6 (between study dates 107 and 183) and at least 66% of platelet counts qualified as a response
Measured: At 6 months
Stable response at 1 year
Percentage of participants with at least 2 platelet counts 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
Percentage of participants with bleeding events overall and by World Health Organization (WHO) bleeding scale severity
This is to assess the incidence and severity of bleeding in each treatment arm
Measured: Randomization to end of study (up to 39 months after randomization of last patient)
Number of participants with bleeding events overall and by World Health Organization (WHO) bleeding scale severity
This is to assess the number and severity of bleeding in each treatment arm
Measured: Randomization to end of study (up to 39 months after randomization of last patient)
Number of participants receiving rescue treatment (cummulative dose/duration of steroids exposure)
This is to assess the number of participants receiving rescue treatment.
Measured: Randomization to end of study (up to 39 months after randomization of last patient)
Percentage of participants receiving rescue treatment (cummulative dose/duration of steroids exposure)
This is to assess the need of rescue treatment in each treatment group by percentage.
Measured: Randomization to end of study (up to 39 months after randomization of last patient)
Cumulative dose/duration of steroids exposure
Duration of exposure to corticosteroids calculated from randomization (first dose) to end of study or last last contact date (if the participant is lost to follow-up).
Measured: From screening to end of study (up to 39 months after randomization of last patient)
Change from baseline on T scores 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
Measured: From screening (baseline) till end of study (up to 39 months after randomization of last patient)
Change from baseline in ITP-PAQ domain scores
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, and Overall QoL. Each item is rated on a Likert type scale
Measured: From screening (baseline) till end of study (up to 39 months after randomization of last patient)
Change from baseline in frequency of CD19+ B cell counts
Post baseline frequency (%within the CD45) of CD19+ B cell counts compare to baseline.
Measured: Randomization to end of study (up to 39 months after randomization of last patient)
Change from baseline in absolute number of CD19+ B cell counts
Post baseline absolute number of CD19+ B cell counts compare with baseline
Measured: Randomization to end of study (up to 39 months after randomization of last patient)
Time to first occurrence of B-cell recovery
B-cell recovery is defined as ≥80% of baseline and ≥LLOQ, or ≥50 cells/μL
Measured: Randomization to end of study (up to 39 months after randomized of last patient)
Change from baseline in inmmunoglobulins
Change from baseline in immunoglobulin levels
Measured: Randomization to end of study (up to 39 months after last randomized patients)
PK parameters: AUClast
AUClast: area under the curve from time zero till the last measurable concentration sampling time (tlast)
Measured: After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
PK parameter: AUCtau
Area under the curve calculated to the end of a dosing interval (tau)
Measured: After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
PK parameters: Cmax
Maximum (peak) observed plasma, blood, serum, or other body fluid drug concentration after single dose administration
Measured: After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
PK parameters: Tmax
Time to reach maximum (peak) plasma, blood, serum, or other body fluid drug concentration
Measured: After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
PK parameters: Accumulation ratio Racc
Accumulation ratio calculated using AUC values obtained between the last and first dose
Measured: After last dose (pre-dose, 2, 336, 672, 1344, 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 assess 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 assess 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 start3 Feb 2023
- Primary completion29 Oct 2026 (estimated)
- Study completion5 Jan 2029 (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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 226 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
- NCT05653349
- Sponsor's protocol ID
- CVAY736I12301
- Other IDs
- 2024-511704-17-00
- Acronym
- VAYHIT1
- Status last verified
- Jun 2026
- First submitted
- 23 Nov 2022
- Last updated
- 15 Jun 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