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

NCT05653349 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: CVAY736I12301
• Study Type: interventional
• Target: 226
• Locations: 24 countries
• Sites: 110

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

Conditions

Primary Immune Thrombocytopenia (ITP)

Keywords

Primary immune thrombocytopenia (ITP); ianalumab; VAY736; B-cell depletion; B-cell Activating Factor Receptor (BAFF-R) blockade

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (24)

• Complete Response (CR) rate in each treatment group

  Randomization to end of study (up to 39 months after randomization of last patient)

• Response (R) rate in each treatment group

  Randomization to end of study (up to 39 months after randomization of last patient)

• Time to complete response in each treatment group

  Randomization to end of study (up to 39 months after randomization of last patient)

• Duration of response in each treatment group

  Randomization to end of study (up to 39 months after randomization of last patient)

• Stable response at 6 months

  At 6 months

Study Arms (3)

Ianalumab Lower dose

EXPERIMENTAL

Lower dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified)

Biological: Ianalumab; Drug: Corticosteroids

Ianalumab Higher dose

EXPERIMENTAL

Higher dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified)

Biological: Ianalumab; Drug: Corticosteroids

Placebo

PLACEBO COMPARATOR

Placebo administered intravenously with corticosteroids oral or parental (if clinically justified)

Drug: Placebo; Drug: Corticosteroids

Interventions

Placebo DRUG

Intravenous infusion, prepared from matching placebo

Placebo

Corticosteroids DRUG

Oral or parental (if clinically justified)

Ianalumab Higher dose; Ianalumab Lower dose; Placebo

Ianalumab BIOLOGICAL

Intravenous infusion, prepared from concentrate solution

Also known as: VAY736

Ianalumab Higher dose; Ianalumab Lower dose

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (114)

Yuma Regional Medical Center

Yuma, Arizona, 85349, United States

Location

Community Cancer Institute

Clovis, California, 93611, United States

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Compassionate Care Res Group Inc

Fountain Valley, California, 92708, United States

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University of Colorado Anschutz

Aurora, Colorado, 80045, United States

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DH Cancer Research Center LLC

Margate, Florida, 33063, United States

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Uni of Chi Medi Ctr Hema and Onco

Chicago, Illinois, 60637, United States

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Parkview Research Center

Fort Wayne, Indiana, 46845, United States

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Oncology Care Associates

Bethesda, Maryland, 20817, United States

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Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

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Beth Israel Deaconess Med Center

Boston, Massachusetts, 02215, United States

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Michigan Center of Medical Research

Farmington Hills, Michigan, 48334, United States

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Mayo Clinic Rochester

Rochester, Minnesota, 55905, United States

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Metro Minnesota CCOP

Saint Louis Park, Minnesota, 55416, United States

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St Vincent Frontier Cancer Center

Billings, Montana, 59102, United States

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Optum Health

Lake Success, New York, 11042, United States

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Hematology Oncology Association of Rockland

Nyack, New York, 10960, United States

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Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

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STAT Research Inc

Dayton, Ohio, 45402, United States

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Rhode Island Hospital

Providence, Rhode Island, 02903, United States

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Mays Cancer Ctr Uthsa Mdacc

San Antonio, Texas, 78229, United States

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Community Cancer Trials of Utah

Ogden, Utah, 84405, United States

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Novartis Investigative Site

CABA, Buenos Aires, 1280, Argentina

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Novartis Investigative Site

CABA, Buenos Aires, C1414DRK, Argentina

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San Miguel Tucuman, Tucumán Province, T4000DPK, Argentina

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Novartis Investigative Site

Caba, C1015ABO, Argentina

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Caba, C1039AAC, Argentina

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Córdoba, 5000, Argentina

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Garran, Australian Capital Territory, 2605, Australia

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Wooloongabba, Queensland, 4102, Australia

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Clayton, Victoria, 3168, Australia

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Melbourne, Victoria, 3004, Australia

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Innsbruck, Tyrol, 6020, Austria

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Salzburg, 5020, Austria

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Roeselare, West-Vlaanderen, 8800, Belgium

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Brasschaat, 2930, Belgium

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Bruges, 8000, Belgium

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Leuven, 3000, Belgium

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Plovdiv, 4002, Bulgaria

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Sofia, 1431, Bulgaria

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Beijing, Beijing Municipality, 100044, China

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Guangzhou, Guangdong, 510515, China

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Shenzhen, Guangdong, 518037, China

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Shijiazhuang, Hebei, 050000, China

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Luoyang, Henan, 471003, China

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Zhengzhou, Henan, 450003, China

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Changzhou, Jiangsu, 213004, China

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Nanchang, Jiangxi, 330006, China

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Kunming, Yunnan, 650101, China

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Hangzhou, Zhejiang, 310006, China

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Bengbu, 233004, China

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Dalian, 116000, China

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Tianjin, 300020, China

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Ostrava, Poruba, 708 52, Czechia

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Prague, 100 34, Czechia

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Prague, 128 08, Czechia

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Caen, 14033, France

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Chambéry, 73000, France

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Le Mans, 72000, France

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Lille, 59037, France

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Rennes, 35033, France

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Frankfurt am Main, Hesse, 60590, Germany

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Dresden, Saxony, 01307, Germany

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Jena, Thuringia, 07740, Germany

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Dresden, 01307, Germany

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Essen, 45147, Germany

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Giessen, 35392, Germany

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New Territories, Hong Kong, 999077, Hong Kong

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Hong Kong, 999077, Hong Kong

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Debrecen, Hajdu Bihar Megye, 4032, Hungary

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Budapest, H-1083, Hungary

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Ahmedabad, Gujarat, 380013, India

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Rishikesh, Uttarakhand, 249203, India

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Kolkata, West Bengal, 700094, India

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Bologna, BO, 40138, Italy

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Florence, FI, 50134, Italy

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Roma, RM, 00168, Italy

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Trieste, TS, 34129, Italy

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Vicenza, VI, 36100, Italy

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Nagakute, Aichi-ken, 480-1195, Japan

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Shibukawa, Gunma, 377-0280, Japan

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Sapporo, Hokkaido, 0608604, Japan

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Okayama, Okayama-ken, 7011192, Japan

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Iruma-gun, Saitama, 3500495, Japan

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Chiba, 260-0852, Japan

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Fukuoka, 815-8555, Japan

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Gifu, 5008513, Japan

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Osaka, 543-8555, Japan

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Kuching, Sarawak, 93586, Malaysia

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George Town, 10050, Malaysia

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Johor Bahru, 80100, Malaysia

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Kuala Selangor, 68000, Malaysia

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Mexico City, Mexico City, 06720, Mexico

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Monterrey, Nuevo León, 64460, Mexico

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Grålum, 1714, Norway

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Bucharest, 030171, Romania

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Singapore, 119074, Singapore

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Singapore, 169608, Singapore

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Barcelona, 08035, Spain

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Madrid, 28009, Spain

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Madrid, 28046, Spain

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Murcia, 30008, Spain

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Salamanca, 37007, Spain

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Seville, 41013, Spain

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Bangkok, 10330, Thailand

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Bangkok, 10700, Thailand

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Chiang Mai, 50200, Thailand

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Samsun, Atakum, 55200, Turkey (Türkiye)

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Aydin, Efeler, 09100, Turkey (Türkiye)

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Istanbul, Fatih, 34098, Turkey (Türkiye)

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Birmingham, West Midlands, B15 2TH, United Kingdom

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London, W12 0HS, United Kingdom

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Nottingham, NG5 1PB, United Kingdom

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Hanoi, 100000, Vietnam

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Novartis Investigative Site

Ho Chi Minh City, 70000, Vietnam

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MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

ianalumab; Adrenal Cortex Hormones

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 23, 2022
• First Posted: December 16, 2022
• Study Start: February 3, 2023
• Primary Completion (Estimated): October 29, 2026
• Study Completion (Estimated): December 6, 2028
• Last Updated: April 22, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

NO (1); BE (4); BU (2); HU (2); MY (4); IN (3); ME (2); TH (3); CZ (3); US (21); AU (2); ES (6); DE (6); IT (5); CN (13); JP (9); TU (3); GB (3); RO (1); SG (2); VN (2); AR (6); FR (5); HK (2)