This study is intended to explore the efficacy and safety of a second course of ianalumab after experiencing treatment failure in the pivotal Primary Immune Thrombocytopenia (ITP) trials (CVAY736I12301, CVAY736Q12301) and after loss of durable response in the pivotal Warm Autoimmune Hemolytic Anemia (wAIHA) trial (CVAY736O12301).
Signed informed consent obtained prior to participation in the study.
Male or female participants aged 18 years and older on the day of signing informed consent
Previously enrolled and treated either with ianalumab/placebo in addition to first-line corticosteroids on protocol CVAY736I12301 or with ianalumab/placebo in addition to eltrombopag in the second line on protocol CVAY736Q12301, and who experienced treatment failure (TF) by parent trial definition ≥ 2 years after the last infusion of ianalumab/placebo
Rescue medication and/or bridging therapy are allowed to be started within the 28 days prior to screening; platelet count results obtained prior to the start of the therapy must be used to assess eligibility and have to be collected within 30 days prior to screening
Evans syndrome or any cytopenia other than thrombocytopenia (for ITP participants) or anemia (for wAIHA), except for grade 1 anemia due to blood loss or iron deficiency.
Secondary wAIHA with BM involvement for wAIHA patients
Current life-threatening bleeding or history of life-threatening bleeding due to thrombocytopenia
Therapy for ITP or wAIHA other than ianalumab/placebo, bridging/rescue therapies and supportive care prior to the beginning of the screening window
Parallel
Ianalumab
Biological/VaccineConcentrate for solution for infusion for intravenous use
Treatment failure free (yes, no) for participants with Immune Thrombocytopenia (ITP)
Treatment failure free (yes, no) by 12 months after start of second course of ianalumab at assigned dose.
Durable response for participants with Warm Autoimmune Hemolytic Anemia (wAIHA)
Durable response (hemoglobin (Hb) ≥10 g/dL and ≥2 g/dL increase from baseline) for a period of at least 8 consecutive weeks, between W9 and W25 in the absence of rescue or prohibited treatment prior to that durable response achievement.
Response rate in participants with ITP only
Percentage of participants with ITP achieving a response among all participants who received a second course of ianalumab, and among cohorts and/or group of cohorts.
Complete response rate in participants with ITP only
Percentage of participants with ITP achieving a complete response among all participants who received a second course of ianalumab, and among cohorts and/or group of cohorts.
Number and percentage of participants receiving new ITP therapy only
Number and percentage of participants who need new ITP therapy among all the participants who received a second course of ianalumab, and among cohorts and/or group of cohorts.
Response and complete response in participants with wAIHA only
Percentage of participants with wAIHA achieving a response and complete response among all participants who received a second course of ianalumab and among cohorts and/or group of cohorts.
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