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

NCT ID: NCT05653219

Last Updated: 2026-01-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

152 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-02-02

Study Completion Date

2028-04-08

Brief Summary

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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

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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

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Primary Immune Thrombocytopenia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Treatment arm 1

Participants will receive eltrombopag and ianalumab lower dose

Group Type EXPERIMENTAL

Ianalumab

Intervention Type BIOLOGICAL

Concentrate for solution for infusion for intravenous use

Eltrombopag

Intervention Type DRUG

Film-coated tablet for oral use

Treatment arm 2

Participants will receive eltrombopag and ianalumab higher dose

Group Type EXPERIMENTAL

Ianalumab

Intervention Type BIOLOGICAL

Concentrate for solution for infusion for intravenous use

Eltrombopag

Intervention Type DRUG

Film-coated tablet for oral use

Treatment arm 3

Participants will receive eltrombopag and placebo

Group Type PLACEBO_COMPARATOR

Eltrombopag

Intervention Type DRUG

Film-coated tablet for oral use

Placebo

Intervention Type DRUG

Concentrate for solution for infusion for intravenous use.

Interventions

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Ianalumab

Concentrate for solution for infusion for intravenous use

Intervention Type BIOLOGICAL

Eltrombopag

Film-coated tablet for oral use

Intervention Type DRUG

Placebo

Concentrate for solution for infusion for intravenous use.

Intervention Type DRUG

Other Intervention Names

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VAY736 ETB115

Eligibility Criteria

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Inclusion Criteria

1. Male or female patients aged 18 years and older on the day of signing the informed consent.
2. A signed informed consent must be obtained prior to participation in the study.
3. A diagnosis of primary ITP, with insufficient response to, or relapse after a first-line corticosteroid therapy ± IVIG.
4. Patient with platelet count \<30G/L (whom eltrombopag is clinically indicated as per physician's discretion) and with no contraindication to receive eltrombopag

Exclusion Criteria

1. 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.
2. 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).
3. Patients with history of clinically significant hematological disorders, or with marked altered hematologic parameters
4. Patients with current or history of life-threatening bleeding
5. 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
6. Patients with known active or uncontrolled infection requiring systemic treatment during screening period
7. Patients with hepatic impairment
8. Patients with concurrent coagulation disorders and/or receiving antiplatelet or anticoagulant medication with an exemption of low dose of acetylsalicylic acid (≤150 mg daily)
9. Nursing (breast feeding) or pregnant women
Minimum Eligible Age

18 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Novartis Investigative Site

Chiang Mai, , Thailand

Site Status

Yuma Regional Medical Center

Yuma, Arizona, United States

Site Status

University of Colorado Anschutz

Aurora, Colorado, United States

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NorthShore University Health System

Evanston, Illinois, United States

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Boston Medical Center

Boston, Massachusetts, United States

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UMASS Memorial Medical Center

Worcester, Massachusetts, United States

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Michigan Center of Medical Research

Farmington Hills, Michigan, United States

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St Vincent Frontier Cancer Center

Billings, Montana, United States

Site Status

Hematology Oncology Association of Rockland

Nyack, New York, United States

Site Status

Montefiore Medical Center

The Bronx, New York, United States

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Novartis Investigative Site

CABA, , Argentina

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Novartis Investigative Site

Vienna, , Austria

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Novartis Investigative Site

Yvoir, Namur, Belgium

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Roeselare, West-Vlaanderen, Belgium

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Guangzhou, Guangdong, China

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Wuhan, Hubei, China

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Binzhou, Shandong, China

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Hangzhou, Zhejiang, China

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Beijing, , China

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Jinan, , China

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Tianjin, , China

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Brno, , Czechia

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Prague, , Czechia

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Prague, , Czechia

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Blois, , France

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Le Mans, , France

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Cologne, North Rhine-Westphalia, Germany

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Dresden, Saxony, Germany

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Jena, Thuringia, Germany

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Dresden, , Germany

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Debrecen, Hajdu Bihar Megye, Hungary

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Budapest, , Hungary

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Rishikesh, Uttarakhand, India

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Kolkata, West Bengal, India

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Bologna, BO, Italy

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Roma, RM, Italy

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Roma, RM, Italy

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Trieste, TS, Italy

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Nagoya, Aichi-ken, Japan

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Osaka, Osaka, Japan

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Suita, Osaka, Japan

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Kofu, Yamanashi, Japan

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Kumamoto, , Japan

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George Town, Pulau Pinang, Malaysia

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Kota Kinabalu, Sabah, Malaysia

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Subang Jaya, Selangor, Malaysia

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George Town, , Malaysia

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Johor Bahru, , Malaysia

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Kuala Selangor, , Malaysia

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Saltillo, Coahuila, Mexico

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Morelia, Michoacán, Mexico

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Monterrey, Nuevo León, Mexico

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Leiden, South Holland, Netherlands

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Utrecht, , Netherlands

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Makati City, , Philippines

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Quezon, , Philippines

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Craiova, Dolj, Romania

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Timișoara, , Romania

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Singapore, , Singapore

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Singapore, , Singapore

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Jeollanam, , South Korea

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Seoul, , South Korea

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Santiago Compostela, A Coruna, Spain

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Salamanca, , Spain

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Taoyuan District, , Taiwan

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Bangkok, , Thailand

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Samsun, Atakum, Turkey (Türkiye)

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Ankara, Bilkent Cankaya, Turkey (Türkiye)

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Aydin, Efeler, Turkey (Türkiye)

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Istanbul, Fatih, Turkey (Türkiye)

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Izmir, , Turkey (Türkiye)

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London, , United Kingdom

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London, , United Kingdom

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Novartis Investigative Site

Oxford, , United Kingdom

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Countries

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United States Argentina Austria Belgium China Czechia France Germany Hungary India Italy Japan Malaysia Mexico Netherlands Philippines Romania Singapore South Korea Spain Taiwan Thailand Turkey (Türkiye) United Kingdom

References

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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.

Reference Type DERIVED
PMID: 41363800 (View on PubMed)

Other Identifiers

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2024-512890-28-00

Identifier Type: OTHER

Identifier Source: secondary_id

CVAY736Q12301

Identifier Type: -

Identifier Source: org_study_id

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