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

Novartis

Status and phase: Enrolling; Phase 3

Conditions: Primary Immune Thrombocytopenia (ITP)

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

Study type: Interventional

Funder types: Industry

Identifiers

NCT05653349

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 maintaining platelet count ≥30 G/L 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.

Enrollment: 225 estimated 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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225 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