A phase 3, randomized, double-blind study of ianalumab (VAY736) versus placebo in addition to first-line corticosteroids in primary immune thrombocytopenia (VAYHIT1)
Study of a study medicine added to steroids for primary ITP
Brief description of study
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 increasing the rate of stable response off treatment at 12 months (SROT-12) in adult participants with primary ITP.
Detailed description of study
This is a multi-center, randomized, double-blind Phase 3 study to assess the efficacy and safety of two different doses of ianalumab compared to placebo in adults with primary ITP (platelets count <30 G/L) who require first-line standard-of-care corticosteroids.
After completion of the screening period, the participants will enter the randomized treatment period (ianalumab/placebo with standard of care corticosteroids).
After the treatment period, all participants will enter the follow-up period to be monitored for efficacy and safety or safety only depending on how they respond to the study treatment.
The efficacy and safety follow-up period will last until treatment failure or up to 39 months after randomization of the last patient, whichever occurs first. Safety follow-up period will be performed for at least 20 weeks and up to 2 years after the last ianalumab/placebo dose.
The primary endpoint was updated in May 2026 to stable response off treatment at 12 months (SROT-12). The previous primary endpoint, time to treatment failure (TTF), remains in the study as a key secondary objective.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Primary Immune Thrombocytopenia (ITP)
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Age: 18 years - 100 years
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Gender: All
Inclusion Criteria:
- 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.
Key Exclusion Criteria:
- 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
Other protocol-defined Inclusion/Exclusion may apply.
The purpose of this research study is to learn about the effects and safety of adding a study medicine to first-line corticosteroids (steroids) for adults with primary immune thrombocytopenia (primary ITP). Primary ITP is a condition where the immune system attacks platelets, which can lead to low platelet counts and bleeding risk. The study is focused on adults who were diagnosed recently and who need first-line steroid treatment.
Participants will receive first-line corticosteroids and will also receive either the study medicine (at 1 of 2 dose levels) or a placebo. A placebo looks like the study medicine but does not contain any medicine. After the treatment period, participants will continue into a follow-up period. During follow-up, the study team will monitor participants for effects and safety, or for safety only, depending on how they respond to the study treatment. Follow-up for effects and safety may continue until treatment failure or for a longer period set by the study. Safety follow-up will continue for at least 20 weeks and may continue for up to 2 years after the last dose of study medicine or placebo.
Who can participate:
Adults with primary ITP may be able to participate if they:
- Are age 18 or older
- Were diagnosed with primary ITP within the past 3 months
- Had a very low platelet count before starting any first-line ITP treatment
- Have had a platelet response to corticosteroids (with or without IVIG) at some point before joining the study
Study timeline and visits:
After a screening period, participants will enter a treatment period and then a follow-up period. Follow-up for effects and safety may continue until treatment failure or up to about 39 months after the last person is assigned to treatment (whichever happens first). Safety follow-up will last at least 20 weeks and may last up to 2 years after the last dose.
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