Last Update: Nov 25, 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 - (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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Buenos Aires,C1425aum,Argentina

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Wels,A 4600,Austria

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Vienna,A 1090,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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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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Le Mans,72000,France

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Chandigarh,160 012,India

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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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Suita,Osaka,565 0871,Japan

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Nagoya,Aichi,453-8511,Japan

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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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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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Seoul,Seocho Gu,06591,Korea, Republic of

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Seoul,06351,Korea, Republic of

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Seoul,158-710,Korea, Republic of

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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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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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Ciudad De Mexico,Mexico CP,14080,Mexico

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Makati City,1229,Philippines

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Ogden,Utah,84405,United States

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Aurora,Colorado,80045,United States

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Worcester,Massachusetts,01665,United States

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Michael Drazer
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Dallas,Texas,75251,United States

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University of Pennsylvania

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Philadelphia,Pennsylvania,19104,United States

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Napa Research

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Margate,Florida,33063,United States

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Clovis,California,93611,United States

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Lake Success,New York,11042,United States

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NorthShore University Health System

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