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).
This is a multicenter, Phase 2 exploratory study to assess the efficacy and safety of a second course of ianalumab in adults with ITP and wAIHA who have previously benefited from ianalumab. All participants will receive ianalumab at the dose of study treatment from which they previously derived benefit. After completion of the screening period, participants who do not meet treatment failure criteria during treatment period will enter the safety and efficacy follow-up, whereas participants who reported a treatment failure during the treatment period will enter the safety follow-up only. The study will end once all participants have completed safety and/or efficacy follow-up since their last dose of ianalumab or discontinued the study earlier.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
60
Concentrate for solution for infusion for intravenous use
Novartis Investigative Site
Ostrava, Poruba, Czechia
RECRUITINGTreatment 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.
Time frame: From treatment start date until the events indicative of treatment failure (up to 2 years after last 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.
Time frame: At least 8 consecutive weeks, between week 9 and week 25
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.
Time frame: from treatment start to end of study (up to 2 years after last dose)
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.
Time frame: from treatment start to end of study (up to 2 years after last dose)
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.
Time frame: from treatment start to end of study (up to 2 years after last dose)
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.
Time frame: from treatment start to end of study (up to 2 years after last dose)
Number and percentage of participants receiving new wAIHA therapy only
Number and percentage of participants who need new wAIHA therapy among all the participants who received a second course of ianalumab, and among cohorts and/or group of cohorts.
Time frame: from treatment start to end of study (up to 2 years after last dose)
Number and percentage of participants receiving rescue treatment
Number and percentage of participants who need rescue treatment among all the participants who received a second course of ianalumab, and among cohorts and/or group of cohorts.
Time frame: from treatment start to end of study (up to 2 years after last dose)
Number of severe infections and percentage of participants with severe infection
This is to assess the safety profile of ianalumab.
Time frame: from treatment start to end of study (up to 2 years after last dose)
Ianalumab concentration in serum
Assess the pharmacokinetics of a second course of ianalumab treatment.
Time frame: after first dose (pre dose 0 and 672 hours) and at study end (up to 2 years after last dose of Ianalumab)
Incidence of anti-ianalumab antibodies in serum (ADA assay) over time
Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants to assess the immunogenicity of Ianalumab.
Time frame: up to end of treatment (Week 16), end of study (up to 2 years after last dose of Ianalumab)
Titer of anti-ianalumab antibodies in serum (ADA assay) over time
Assess the immunogenicity of second course of ianalumab treatment.
Time frame: up to end of treatment (Week 16), end of study (up to 2 years after last dose of Ianalumab)
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