A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids

NCT05653219 | Active Not Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: CVAY736Q123012024-512890-28-00
• Study Type: interventional
• Target: 152
• Locations: 24 countries
• Sites: 77

Brief 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.

Conditions

Primary Immune Thrombocytopenia

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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

  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)

  At 6 months

• Complete Response rate at each timepoint

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

• Response rate at each timepoint

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

• Best response rate across all timepoints

  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 up to the longest observed treatment period duration

Study Arms (3)

Treatment arm 1

EXPERIMENTAL

Participants will receive eltrombopag and ianalumab lower dose

Biological: Ianalumab; Drug: Eltrombopag

Treatment arm 2

EXPERIMENTAL

Participants will receive eltrombopag and ianalumab higher dose

Biological: Ianalumab; Drug: Eltrombopag

Treatment arm 3

PLACEBO COMPARATOR

Participants will receive eltrombopag and placebo

Drug: Eltrombopag; Drug: Placebo

Interventions

Placebo DRUG

Concentrate for solution for infusion for intravenous use.

Treatment arm 3

Ianalumab BIOLOGICAL

Concentrate for solution for infusion for intravenous use

Also known as: VAY736

Treatment arm 1; Treatment arm 2

Eltrombopag DRUG

Film-coated tablet for oral use

Also known as: ETB115

Treatment arm 1; Treatment arm 2; Treatment arm 3

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (77)

Yuma Regional Medical Center

Yuma, Arizona, 85349, United States

Location

University of Colorado Anschutz

Aurora, Colorado, 80045, United States

Location

NorthShore University Health System

Evanston, Illinois, 60201, United States

Location

Boston Medical Center

Boston, Massachusetts, 02118, United States

Location

UMASS Memorial Medical Center

Worcester, Massachusetts, 01665, United States

Location

Michigan Center of Medical Research

Farmington Hills, Michigan, 48334, United States

Location

St Vincent Frontier Cancer Center

Billings, Montana, 59102, United States

Location

Hematology Oncology Association of Rockland

Nyack, New York, 10960, United States

Location

Montefiore Medical Center

The Bronx, New York, 10461, United States

Location

Novartis Investigative Site

CABA, C1181ACH, Argentina

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Novartis Investigative Site

Vienna, A 1090, Austria

Location

Novartis Investigative Site

Yvoir, Namur, 5530, Belgium

Location

Novartis Investigative Site

Roeselare, West-Vlaanderen, 8800, Belgium

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Guangzhou, Guangdong, 510515, China

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Wuhan, Hubei, 430022, China

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Binzhou, Shandong, 256603, China

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Hangzhou, Zhejiang, 310003, China

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Beijing, 100730, China

Location

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Jinan, 250012, China

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Tianjin, 300020, China

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Brno, 625 00, Czechia

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Prague, 100 34, Czechia

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Prague, 128 08, Czechia

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Blois, 41000, France

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Le Mans, 72000, France

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Cologne, North Rhine-Westphalia, 50937, Germany

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Düsseldorf, North Rhine-Westphalia, 40225, Germany

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Dresden, Saxony, 01307, Germany

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Jena, Thuringia, 07740, Germany

Location

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Aachen, 52074, Germany

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Bonn, 53105, Germany

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Dresden, 01307, Germany

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Debrecen, Hajdu Bihar Megye, 4032, Hungary

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Budapest, H-1083, Hungary

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Rishikesh, Uttarakhand, 249203, India

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Kolkata, West Bengal, 700014, India

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Bologna, BO, 40138, Italy

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Roma, RM, 00133, Italy

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Roma, RM, 00168, Italy

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Trieste, TS, 34129, Italy

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Nagoya, Aichi-ken, 453-8511, Japan

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Osaka, Osaka, 5400006, Japan

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Suita, Osaka, 565-0871, Japan

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Kofu, Yamanashi, 400-8506, Japan

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Kumamoto, 860-0008, Japan

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George Town, Pulau Pinang, 10450, Malaysia

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Kota Kinabalu, Sabah, 88586, Malaysia

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Subang Jaya, Selangor, 47500, 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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Saltillo, Coahuila, 25230, Mexico

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Morelia, Michoacán, 58260, Mexico

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Monterrey, Nuevo León, 64460, Mexico

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Leiden, South Holland, 2333 ZA, Netherlands

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Utrecht, 3584 CX, Netherlands

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Makati City, 1229, Philippines

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Quezon, 1102, Philippines

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Craiova, Dolj, 200136, Romania

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Timișoara, 300079, Romania

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Singapore, 119074, Singapore

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Singapore, 169608, Singapore

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Jeollanam, 519763, South Korea

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Seoul, 06591, South Korea

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Santiago Compostela, A Coruna, 15706, Spain

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Salamanca, 37007, Spain

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Taoyuan, 33305, Taiwan

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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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Ankara, Bilkent Cankaya, 06800, Turkey (Türkiye)

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Aydin, Efeler, 09100, Turkey (Türkiye)

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Istanbul, Fatih, 34098, Turkey (Türkiye)

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Novartis Investigative Site

Izmir, 35100, Turkey (Türkiye)

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Novartis Investigative Site

London, E1 1BB, United Kingdom

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Novartis Investigative Site

London, W12 0HS, United Kingdom

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Novartis Investigative Site

Oxford, OX3 7LE, United Kingdom

Location

Related Publications (1)

• 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. 2025 Dec 9. doi: 10.1056/NEJMoa2515168. Online ahead of print.

  PMID: 41363800 DERIVED

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

ianalumab; eltrombopag

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

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 2, 2023
• Primary Completion: June 19, 2025
• Study Completion (Estimated): April 8, 2028
• Last Updated: May 1, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

IT (4); BE (2); RO (2); JP (5); IN (2); NL (2); AU (1); ME (3); FR (2); AR (1); DE (7); PH (2); KR (2); TW (1); GB (3); HU (2); MY (6); SG (2); US (9); CN (7); ES (2); TH (2); TU (5); CZ (3)