A phase III, randomized, double-blind study of ianalumab (VAY736) versus placebo in addition to first-line corticosteroids in primary immune thrombocytopenia

JRCT ID: jRCT2061220095

Registered date:09/02/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Primary immune thrombocytopenia
• Date of first enrollment: 07/06/2023
• Target sample size: 18
• Countries of recruitment: Argentina,Japan,Australia,Japan,Austria,Japan,Belgium,Japan,Brazil,Japan,Bulgaria,Japan,China,Japan,Czech Republic,Japan,France,Japan,Germany,Japan,Hong Kong,Japan,Hungary,Japan,India,Japan,Italy,Japan,Malaysia,Japan,Mexico,Japan,Norway,Japan,Romania,Japan,Singapore,Japan,Spain,Japan,Thailand,Japan,Turkey,Japan,United Kingdom,Japan,United States,Japan,Vietnam,Japan
• Study type: Interventional

Intervention(s)

Arm A: Ianalumab 3 mg/kg intravenously every 4 weeks plus corticosteroid administered orally or parenterally if clinically justified
Arm B: Ianalumab 9 mg/kg intravenously every 4 weeks plus corticosteroid administered orally or parenterally if clinically justified
Arm C: Placebo intravenously every 4 weeks plus corticosteroid administered orally or parenterally if clinically justified

Outcome(s)

• Primary Outcome: Time from randomization to Treatment Failure (TTF) defined as platelet count below 30 G/L later than 8 weeks from randomization, need for a rescue treatment later than 8 weeks from randomization, start of a second-line therapy or death (whatever the cause).

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include 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.

Exclude criteria

- Evans syndrome or any other cytopenia
- Current life-threatening bleeding
- Previous ITP treatment, including splenectomy, except for corticosteroids and/or IVIG for up to 28 days before randomization.
- 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

Related Information

• Primary Sponsor: Yamauchi Kyosuke
• Secondary ID(s): NCT05653349