Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP) (VAYHIT1)

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 maintaining platelet count ≥30 G/L in adult participants with primary ITP.

Study Overview

• Status: Active, not recruiting
• Conditions: Primary Immune Thrombocytopenia (ITP)
• Intervention / Treatment: Biological: Ianalumab; Drug: Corticosteroids; Drug: Placebo

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.

• Study Type: Interventional
• Enrollment (Actual): 226
• Phase: Phase 3

Contacts and Locations

Study Locations

• Argentina
  • Caba, Argentina, C1015ABO
    • Novartis Investigative Site
  • Caba, Argentina, C1039AAC
    • Novartis Investigative Site
  • Córdoba, Argentina, 5000
    • Novartis Investigative Site
  • Buenos Aires
    • CABA, Buenos Aires, Argentina, C1414DRK
      • Novartis Investigative Site
    • CABA, Buenos Aires, Argentina, 1280
      • Novartis Investigative Site
  • Tucumán Province
    • San Miguel Tucuman, Tucumán Province, Argentina, T4000DPK
      • Novartis Investigative Site
• Australia
  • Australian Capital Territory
    • Garran, Australian Capital Territory, Australia, 2605
      • Novartis Investigative Site
  • Queensland
    • Wooloongabba, Queensland, Australia, 4102
      • Novartis Investigative Site
  • Victoria
    • Clayton, Victoria, Australia, 3168
      • Novartis Investigative Site
    • Melbourne, Victoria, Australia, 3004
      • Novartis Investigative Site
• Austria
  • Salzburg, Austria, 5020
    • Novartis Investigative Site
  • Tyrol
    • Innsbruck, Tyrol, Austria, 6020
      • Novartis Investigative Site
• Belgium
  • Brasschaat, Belgium, 2930
    • Novartis Investigative Site
  • Bruges, Belgium, 8000
    • Novartis Investigative Site
  • Leuven, Belgium, 3000
    • Novartis Investigative Site
  • West-Vlaanderen
    • Roeselare, West-Vlaanderen, Belgium, 8800
      • Novartis Investigative Site
• Bulgaria
  • Plovdiv, Bulgaria, 4002
    • Novartis Investigative Site
  • Sofia, Bulgaria, 1431
    • Novartis Investigative Site
• China
  • Bengbu, China, 233004
    • Novartis Investigative Site
  • Dalian, China, 116000
    • Novartis Investigative Site
  • Tianjin, China, 300020
    • Novartis Investigative Site
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 100044
      • Novartis Investigative Site
  • Guangdong
    • Guangzhou, Guangdong, China, 510515
      • Novartis Investigative Site
    • Shenzhen, Guangdong, China, 518037
      • Novartis Investigative Site
  • Hebei
    • Shijiazhuang, Hebei, China, 050000
      • Novartis Investigative Site
  • Henan
    • Luoyang, Henan, China, 471003
      • Novartis Investigative Site
    • Zhengzhou, Henan, China, 450003
      • Novartis Investigative Site
  • Jiangsu
    • Changzhou, Jiangsu, China, 213004
      • Novartis Investigative Site
  • Jiangxi
    • Nanchang, Jiangxi, China, 330006
      • Novartis Investigative Site
  • Yunnan
    • Kunming, Yunnan, China, 650101
      • Novartis Investigative Site
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310006
      • Novartis Investigative Site
• Czechia
  • Prague, Czechia, 128 08
    • Novartis Investigative Site
  • Prague, Czechia, 100 34
    • Novartis Investigative Site
  • Poruba
    • Ostrava, Poruba, Czechia, 708 52
      • Novartis Investigative Site
• France
  • Caen, France, 14033
    • Novartis Investigative Site
  • Chambéry, France, 73000
    • Novartis Investigative Site
  • Le Mans, France, 72000
    • Novartis Investigative Site
  • Lille, France, 59037
    • Novartis Investigative Site
  • Rennes, France, 35033
    • Novartis Investigative Site
• Germany
  • Dresden, Germany, 01307
    • Novartis Investigative Site
  • Essen, Germany, 45147
    • Novartis Investigative Site
  • Giessen, Germany, 35392
    • Novartis Investigative Site
  • Hesse
    • Frankfurt am Main, Hesse, Germany, 60590
      • Novartis Investigative Site
  • Saxony
    • Dresden, Saxony, Germany, 01307
      • Novartis Investigative Site
  • Thuringia
    • Jena, Thuringia, Germany, 07740
      • Novartis Investigative Site
• Hong Kong
  • Hong Kong, Hong Kong, 999077
    • Novartis Investigative Site
  • Hong Kong
    • New Territories, Hong Kong, Hong Kong, 999077
      • Novartis Investigative Site
• Hungary
  • Budapest, Hungary, H-1083
    • Novartis Investigative Site
  • Hajdu Bihar Megye
    • Debrecen, Hajdu Bihar Megye, Hungary, 4032
      • Novartis Investigative Site
• India
  • Gujarat
    • Ahmedabad, Gujarat, India, 380013
      • Novartis Investigative Site
  • Uttarakhand
    • Rishikesh, Uttarakhand, India, 249203
      • Novartis Investigative Site
  • West Bengal
    • Kolkata, West Bengal, India, 700094
      • Novartis Investigative Site
• Italy
  • BO
    • Bologna, BO, Italy, 40138
      • Novartis Investigative Site
  • FI
    • Florence, FI, Italy, 50134
      • Novartis Investigative Site
  • RM
    • Roma, RM, Italy, 00168
      • Novartis Investigative Site
  • TS
    • Trieste, TS, Italy, 34129
      • Novartis Investigative Site
  • VI
    • Vicenza, VI, Italy, 36100
      • Novartis Investigative Site
• Japan
  • Chiba, Japan, 260-0852
    • Novartis Investigative Site
  • Fukuoka, Japan, 815-8555
    • Novartis Investigative Site
  • Gifu, Japan, 5008513
    • Novartis Investigative Site
  • Osaka, Japan, 543-8555
    • Novartis Investigative Site
  • Aichi-ken
    • Nagakute, Aichi-ken, Japan, 480-1195
      • Novartis Investigative Site
  • Gunma
    • Shibukawa, Gunma, Japan, 377-0280
      • Novartis Investigative Site
  • Hokkaido
    • Sapporo, Hokkaido, Japan, 0608604
      • Novartis Investigative Site
  • Okayama-ken
    • Okayama, Okayama-ken, Japan, 7011192
      • Novartis Investigative Site
  • Saitama
    • Iruma-gun, Saitama, Japan, 3500495
      • Novartis Investigative Site
• Malaysia
  • George Town, Malaysia, 10050
    • Novartis Investigative Site
  • Johor Bahru, Malaysia, 80100
    • Novartis Investigative Site
  • Kuala Selangor, Malaysia, 68000
    • Novartis Investigative Site
  • Sarawak
    • Kuching, Sarawak, Malaysia, 93586
      • Novartis Investigative Site
• Mexico
  • Mexico City
    • Mexico City, Mexico City, Mexico, 06720
      • Novartis Investigative Site
  • Nuevo León
    • Monterrey, Nuevo León, Mexico, 64460
      • Novartis Investigative Site
• Norway
  • Grålum, Norway, 1714
    • Novartis Investigative Site
• Romania
  • Bucharest, Romania, 030171
    • Novartis Investigative Site
• Singapore
  • Singapore, Singapore, 119074
    • Novartis Investigative Site
  • Singapore, Singapore, 169608
    • Novartis Investigative Site
• Spain
  • Barcelona, Spain, 08035
    • Novartis Investigative Site
  • Madrid, Spain, 28046
    • Novartis Investigative Site
  • Madrid, Spain, 28009
    • Novartis Investigative Site
  • Murcia, Spain, 30008
    • Novartis Investigative Site
  • Salamanca, Spain, 37007
    • Novartis Investigative Site
  • Seville, Spain, 41013
    • Novartis Investigative Site
• Thailand
  • Bangkok, Thailand, 10330
    • Novartis Investigative Site
  • Bangkok, Thailand, 10700
    • Novartis Investigative Site
  • Chiang Mai, Thailand, 50200
    • Novartis Investigative Site
• Turkey (Türkiye)
  • Atakum
    • Samsun, Atakum, Turkey (Türkiye), 55200
      • Novartis Investigative Site
  • Efeler
    • Aydin, Efeler, Turkey (Türkiye), 09100
      • Novartis Investigative Site
  • Fatih
    • Istanbul, Fatih, Turkey (Türkiye), 34098
      • Novartis Investigative Site
• United Kingdom
  • London, United Kingdom, W12 0HS
    • Novartis Investigative Site
  • Nottingham, United Kingdom, NG5 1PB
    • Novartis Investigative Site
  • West Midlands
    • Birmingham, West Midlands, United Kingdom, B15 2TH
      • Novartis Investigative Site
• United States
  • Arizona
    • Yuma, Arizona, United States, 85349
      • Yuma Regional Medical Center
  • California
    • Clovis, California, United States, 93611
      • Community Cancer Institute
    • Fountain Valley, California, United States, 92708
      • Compassionate Care Res Group Inc
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado Anschutz
  • Florida
    • Margate, Florida, United States, 33063
      • DH Cancer Research Center LLC
  • Illinois
    • Chicago, Illinois, United States, 60637
      • Uni of Chi Medi Ctr Hema and Onco
  • Indiana
    • Fort Wayne, Indiana, United States, 46845
      • Parkview Research Center
  • Maryland
    • Bethesda, Maryland, United States, 20817
      • Oncology Care Associates
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
    • Boston, Massachusetts, United States, 02215
      • Beth Israel Deaconess Med Center
  • Michigan
    • Farmington Hills, Michigan, United States, 48334
      • Michigan Center of Medical Research
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic Rochester
    • Saint Louis Park, Minnesota, United States, 55416
      • Metro Minnesota CCOP
  • Montana
    • Billings, Montana, United States, 59102
      • St Vincent Frontier Cancer Center
  • New York
    • Lake Success, New York, United States, 11042
      • Optum Health
    • Nyack, New York, United States, 10960
      • Hematology Oncology Association of Rockland
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic Foundation
    • Dayton, Ohio, United States, 45402
      • STAT Research Inc
  • Rhode Island
    • Providence, Rhode Island, United States, 02903
      • Rhode Island Hospital
  • Texas
    • San Antonio, Texas, United States, 78229
      • Mays Cancer Ctr Uthsa Mdacc
  • Utah
    • Ogden, Utah, United States, 84405
      • Community Cancer Trials of Utah
• Vietnam
  • Hanoi, Vietnam, 100000
    • Novartis Investigative Site
  • Ho Chi Minh City, Vietnam, 70000
    • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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.

Key Exclusion Criteria:

• 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ianalumab Lower dose; Lower dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified)	Biological: Ianalumab; Intravenous infusion, prepared from concentrate solution; Other Names: VAY736; Drug: Corticosteroids; Oral or parental (if clinically justified)
Experimental: Ianalumab Higher dose; Higher dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified)	Biological: Ianalumab; Intravenous infusion, prepared from concentrate solution; Other Names: VAY736; Drug: Corticosteroids; Oral or parental (if clinically justified)
Placebo Comparator: Placebo; Placebo administered intravenously with corticosteroids oral or parental (if clinically justified)	Drug: Corticosteroids; Oral or parental (if clinically justified); Drug: Placebo; Intravenous infusion, prepared from matching placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time from randomization to treatment failure (TTF)	Time from randomization until platelet count below 30 G/L, need for a rescue treatment or start of a second-line therapy or death.	Randomization to end of study (up to 39 months after randomization of last patient)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	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.	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	Randomization to end of study (up to 39 months after randomization of last patient)
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	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	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.	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.	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).	From screening to 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	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.	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	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, defined as ≥80% of baseline or ≥50 cells/μL	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	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)	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)	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	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	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	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	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	Up to Week 33
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	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	At 1 year
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	From screening (baseline) till end of study (up to 39 months after randomization of last patient)

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): February 3, 2023
• Primary Completion (Estimated): October 29, 2026
• Study Completion (Estimated): December 6, 2028

Study Registration Dates

• First Submitted: November 23, 2022
• First Submitted That Met QC Criteria: December 14, 2022
• First Posted (Actual): December 16, 2022

Study Record Updates

• Last Update Posted (Actual): April 22, 2026
• Last Update Submitted That Met QC Criteria: April 20, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: VAY736; B-cell depletion; ianalumab; B-cell Activating Factor Receptor (BAFF-R) blockade; Primary immune thrombocytopenia (ITP)
• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Thrombocytopenia; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Hemic and Lymphatic Diseases; Purpura, Thrombocytopenic, Idiopathic; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; ianalumab; Adrenal Cortex Hormones
• Other Study ID Numbers: CVAY736I12301

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

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

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No