Last Update: Dec 12, 2024
A Phase 3 Randomized, Double-blind Study of Ianalumab (VAY736) Versus Placebo in Addition to Eltrombopag in Patients With Primary Immune Thrombocytopenia (ITP) Who Had an Insufficient Response or Relapsed After First Line Steroid Treatment (VAYHIT2)
ClinicalTrials.gov Identifier:
Novartis Reference Number:CVAY736Q12301
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

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. 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.

Primary Immune Thrombocytopenia
Phase3
Recruiting
150
Feb 02, 2023
May 19, 2028
All
18 Years - 100 Years (Adult, Older Adult)

Interventions

Drug

Eltrombopag

Film-coated tablet for oral use
Biological

Ianalumab

Concentrate for solution for infusion for intravenous use
Drug

Placebo

Concentrate for solution for infusion for intravenous use.

Eligibility Criteria

Key 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

Key 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

Other protocol-defined inclusion/exclusion criteria may apply.

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Caba,Buenos Aires,C1181ach,Argentina

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Buenos Aires,C1425aum,Argentina

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Clayton,Victoria,3168,Australia

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Parkville,Victoria,3050,Australia

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Vienna,A 1090,Austria

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Wels,A 4600,Austria

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Linz,4010,Austria

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Brugge,8000,Belgium

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Leuven,3000,Belgium

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Roeselare,8800,Belgium

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Guangzhou,Guangdong,510515,China

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Tianjin,300020,China

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Wuhan,Hubei,430022,China

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Beijing,Beijing,100044,China

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Brno Bohunice,625 00,Czechia

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Praha 10,100 34,Czechia

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Praha,12808,Czechia

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Blois Cedex,41000,France

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Le Mans,72000,France

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Vandoeuvre Les Nancy,54511,France

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Koeln,50937,Germany

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Dresden,01307,Germany

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Giessen,35392,Germany

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Hannover,30161,Germany

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Jena,07740,Germany

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Budapest,H-1083,Hungary

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Debrecen,4032,Hungary

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Rishikesh,Uttarakhand,249203,India

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Kolkata,West Bengal,700014,India

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Chandigarh,160 012,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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Torino,TO,10126,Italy

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Trieste,TS,34129,Italy

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Vicenza,VI,36100,Italy

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Bunkyo-ku,Tokyo,113-8603,Japan

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Narita,Chiba,286-8523,Japan

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Chiyoda-ku,Tokyo,101-8643,Japan

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Kumamoto-city,Kumamoto,860-0008,Japan

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Chuo ku,Tokyo,104 8560,Japan

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Omura,Nagasaki,856-8562,Japan

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Kofu-city,Yamanashi,400-8506,Japan

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Hirakata-city,Osaka,573-1191,Japan

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Aomori,030 8553,Japan

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Osaka-city,Osaka,540-0006,Japan

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Hiroshima,730-8619,Japan

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Suita,Osaka,565 0871,Japan

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Nagoya,Aichi,453-8511,Japan

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Seoul,06351,Korea, Republic of

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Pulau Pinang,10990,Malaysia

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Kota Kinabalu,Sabah,88586,Malaysia

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Selangor,68000,Malaysia

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Kuching,Sarawak,93586,Malaysia

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Subang Jaya,Selangor,47500,Malaysia

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Johor Bahru,80100,Malaysia

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Kuala Lumpur,56000,Malaysia

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Penang,10050,Malaysia

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Ciudad De Mexico,Mexico CP,14080,Mexico

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Morelia,Michoacan,58260,Mexico

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Monterrey,Nuevo Leon,64460,Mexico

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Saltillo,Coahuila,25230,Mexico

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Utrecht,3584cx,Netherlands

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Leiden,Zuid Holland,2333 za,Netherlands

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Gralum,1714,Norway

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Makati City,1229,Philippines

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Quezon,1102,Philippines

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Sibiu,550245,Romania

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Timisoara,300079,Romania

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Bucharest,District 2,022328,Romania

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Bucharest,030 171,Romania

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Craiova,200136,Romania

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Singapore,169608,Singapore

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Singapore,S308433,Singapore

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Barcelona,Catalunya,08003,Spain

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Santiago De Compostela,Galicia,15706,Spain

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Madrid,28041,Spain

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Madrid,28046,Spain

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Murcia,30008,Spain

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Salamanca,Castilla Y Leon,37007,Spain

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London,Sw17 0qt,United Kingdom

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Lake Success,New York,11042,United States

Jennifer Valentine
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Nyack,New York,10960,United States

Marion VanWoudenberg
Sung Ho Lee

Washington U Sch Of Med Ctr For CS

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Saint Louis,Missouri,63110,United States

Morey A. Blinder

Boston Medical Center

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Boston,Massachusetts,02118,United States

J Mark Sloan

Community Cancer Trials of Utah

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Ogden,Utah,84405,United States

Shirley Waters
Carl Gray

University of Colorado Anschutz

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Aurora,Colorado,80045,United States

George Gemlyn

STAT Research Inc

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Dayton,Ohio,45402,United States

Charles Bane

Yuma Regional Medical Center

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Yuma,Arizona,85349,United States

Abhinav Chandra
Amy Olmeda

St Vincent Frontier Cancer Center

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Billings,Montana,59102,United States

Heather Duyck
Patrick Cobb

UMASS Memorial Medical Center

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Worcester,Massachusetts,01665,United States

Daniel Winokur
Lindsey Allen

INTEGRIS Cancer Institute of Oklahoma

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Oklahoma City,Oklahoma,73142,United States

Basgar Alasad
Kellie Larsen-Dyer

University of Pennsylvania

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Philadelphia,Pennsylvania,19104,United States

Adam Cuker
Emmanuel Ndukwudimma

Napa Research

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Margate,Florida,33063,United States

Emilio Araujo-Mino

Community Cancer Institute

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Clovis,California,93611,United States

Mohammed Bukari

Montefiore Medical Center

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Bronx,New York,10461,United States

Irina Murakhovskaya
Noelle Townsend

Michigan Center of Medical Research

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Farmington Hills,Michigan,48334,United States

Faisal Musa

NorthShore University Health System

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Evanston,Illinois,60201,United States

Amy Wang
Michele Britto

Uni of Chi Medi Ctr Hema and Onco

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Chicago,Illinois,60637,United States

Michael Drazer
Sara Moellering

Texas Oncology

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Dallas,Texas,75251,United States

Racha Halawi

Center of Clnl Rsch and Innovation

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Houston,Texas,77008,United States

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John Knecht

Summit Health

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Florham Park,New Jersey,07932,United States

David Gallinson

Compassionate Care Res Group Inc

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Fountain Valley,California,92708,United States

Eric Lee

Parkview Research Center

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Fort Wayne,Indiana,46845,United States

Jeffrey Letzer

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