Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP) (VAYHIT1)

Novartis

Status and phase: Active, not recruiting; Phase 3

Conditions: Primary Immune Thrombocytopenia (ITP)

Treatments: Biological: Ianalumab; Drug: Placebo; Drug: Corticosteroids

Study type: Interventional

Funder types: Industry

Identifiers

NCT05653349

2024-511704-17-00 (Registry Identifier)

CVAY736I12301

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 226 patients

Sex: All

Ages: 18 to 100 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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226 participants in 3 patient groups, including a placebo group

Ianalumab Lower dose

Experimental group

Description:

Lower dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified)

Treatment:

Drug: Corticosteroids

Biological: Ianalumab

Ianalumab Higher dose

Experimental group

Description:

Higher dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified)

Treatment:

Drug: Corticosteroids

Biological: Ianalumab

Placebo

Placebo Comparator group

Description:

Placebo administered intravenously with corticosteroids oral or parental (if clinically justified)

Treatment:

Drug: Corticosteroids

Drug: Placebo