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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT05885555
Registration number
NCT05885555
Ethics application status
Date submitted
15/05/2023
Date registered
2/06/2023
Titles & IDs
Public title
A Study of Ianalumab (VAY736) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least Two Lines of Therapies
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Scientific title
A Phase 2 Study to Evaluate the Efficacy and Safety of Ianalumab (VAY736) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least a Corticosteroid and a Thrombopoietin Receptor Agonist (TPO-RA)
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Secondary ID [1]
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2022-503041-21
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Secondary ID [2]
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CVAY736Q12201
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Primary Immune Thrombocytopenia (ITP)
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Condition category
Condition code
Blood
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Haematological diseases
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Blood
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0
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Other blood disorders
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Inflammatory and Immune System
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0
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Autoimmune diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Other - Ianalumab
Experimental: Single-arm - All eligible participants will receive ianalumab at the same dose.
Treatment: Other: Ianalumab
Intravenous infusion, prepared from concentrate solution
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Intervention code [1]
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Treatment: Other
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Confirmed response
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Assessment method [1]
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Confirmed response is defined as a platelet count of equal or above 50 G/L at two (or more) consecutive assessments at least 7 days apart, in the absence of:
* Rescue treatment for =4 weeks prior to the assessment of the platelet count, and
* New immune thrombocytopenia (ITP) treatment before reaching a confirmed response.
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Timepoint [1]
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Between Week 1 Day 1 and Week 25 Day 1
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Secondary outcome [1]
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Time to confirmed response
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Assessment method [1]
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Time from the first administration of ianalumab to the first assessment in the first sequence of two (or more) platelet assessments meeting the criteria of a confirmed response as defined by the primary endpoint.
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Timepoint [1]
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From Week 1 Day 1 to Week 25 Day 1
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Secondary outcome [2]
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Duration of confirmed response
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Assessment method [2]
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Time from the first assessment in the first sequence of two (or more) platelet assessments meeting the criteria of a confirmed response to loss of response; with loss of response defined as the first of the following events:
* platelet count \<30 G/L,
* start of any rescue or new ITP treatment,
* death (whatever the cause).
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Timepoint [2]
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From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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Secondary outcome [3]
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Complete Response rate at each timepoint
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Assessment method [3]
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Percentage of participants with a platelet count of at least 100 G/L in the absence of rescue treatment/new ITP treatment.
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Timepoint [3]
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From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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Secondary outcome [4]
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Response rate at each timepoint
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Assessment method [4]
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Percentage of participants with a platelet count of at least 50 G/L in the absence of rescue treatment/new ITP treatment.
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Timepoint [4]
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From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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Secondary outcome [5]
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Stable response at 6 months
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Assessment method [5]
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Percentage of participants with at least 75% of platelet counts collected at month 6 (between study days 121 and 183) equal to or above 50 G/L in the absence of rescue treatment/new ITP treatment.
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Timepoint [5]
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At 6 months
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Secondary outcome [6]
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Stable response at 1 year
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Assessment method [6]
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Percentage of participants with at least 66% of platelet counts collected at year 1 (between study days 296 and 379) equal to or above 50 G/L in the absence of rescue treatment/new ITP treatment.
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Timepoint [6]
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At 1 year
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Secondary outcome [7]
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Bleeding events according to the Modified World Health Organization (WHO) bleeding scale
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Assessment method [7]
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Number of participants reporting bleeding events for each grade of the World Health Organization (WHO) bleeding scale at each time point. Severity is graded from 0 to 4, with 0 = no bleeding and 4 = severe hemodynamic instability/central nervous system (CNS) bleeding/fatal.
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Timepoint [7]
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0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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Secondary outcome [8]
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0
Percentage of participants with bleeding events according to the Modified World Health Organization (WHO) bleeding scale
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Assessment method [8]
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Percentage of participants reporting bleeding events for each grade of the WHO bleeding scale at each time point. Severity is graded from 0 to 4, with 0 = no bleeding and 4 = severe hemodynamic instability/central nervous system (CNS) bleeding/fatal.
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Timepoint [8]
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0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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Secondary outcome [9]
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0
Number of participants who received rescue treatment
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Assessment method [9]
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Number of participants who required rescue treatment
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Timepoint [9]
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0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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Secondary outcome [10]
0
0
Percentage of participants who received rescue treatment
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Assessment method [10]
0
0
Percentage of participants who required rescue treatment
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Timepoint [10]
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0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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Secondary outcome [11]
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0
Change from baseline in the frequency of CD19+ B-cell counts
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Assessment method [11]
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Post-baseline frequency of CD19+ B-cell counts compared to baseline
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Timepoint [11]
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From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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Secondary outcome [12]
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0
Change from baseline in the absolute number of CD19+ B-cell counts
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Assessment method [12]
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0
Post-baseline absolute number of CD19+ B-cell counts compared to baseline
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Timepoint [12]
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0
From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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Secondary outcome [13]
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0
Time to first occurrence of B-cell recovery defined as =80% of baseline =50 cells/µL
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Assessment method [13]
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Time to B-cell recovery defined as =80% of baseline or =50 cells/µL
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Timepoint [13]
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From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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Secondary outcome [14]
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0
Change from baseline in immunoglobulins
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Assessment method [14]
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0
Post-baseline immunoglobulin levels (change in titers of Total Ig, IgG, IgM, IgA) compared to baseline
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Timepoint [14]
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From Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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Secondary outcome [15]
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0
Incidence of anti-ianalumab antibodies in serum (ADA assay) over time
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Assessment method [15]
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0
Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants to assess the immunogenicity of ianalumab
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Timepoint [15]
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Up to 20 weeks after last dose of ianalumab
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Secondary outcome [16]
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Titer of anti-ianalumab antibodies in serum (ADA assay) over time
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Assessment method [16]
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Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants to assess the immunogenicity of ianalumab
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Timepoint [16]
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Up to 20 weeks after last dose of ianalumab
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Secondary outcome [17]
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Ianalumab serum concentrations over time
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Assessment method [17]
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Ianalumab concentration in serum over time, including end of infusion and concentration at trough.
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Timepoint [17]
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First dose (pre-dose, 2, 168, 336, 504, 672 hours post-dose); Subsequent doses (pre-dose and 2 hours post-dose); Last dose (pre-dose, 2 336, 672, 1344, 2016, 3360 hours post-dose)
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Secondary outcome [18]
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Confirmed response (CR) in the second course
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Assessment method [18]
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Confirmed response is defined as a platelet count of equal or above 50 G/L at two (or more) consecutive assessments at least 7 days apart, in the absence of:
* Rescue treatment for =4 weeks prior to the assessment of the platelet count, and
* New ITP treatment before reaching a confirmed response.
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Timepoint [18]
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Second course Week 1 Day1 to second course Week 25 Day1
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Secondary outcome [19]
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Time to confirmed response in the second course
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Assessment method [19]
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0
two (or more) platelet assessments meeting the criteria of a confirmed response.
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Timepoint [19]
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Second course Week 1 Day 1 to second course Week 25 Day 1
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Secondary outcome [20]
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Duration of confirmed response in the second course
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Assessment method [20]
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Time from the first assessment, in the second course of two (or more) platelet assessments meeting the criteria of a confirmed response to loss of response; with loss of response defined as the first of the following events:
* platelet count \<30 G/L,
* start of any rescue or new ITP treatment,
* death (whatever the cause).
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Timepoint [20]
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0
Second course Week 1 Day 1 to second course Week 25 Day 1
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Secondary outcome [21]
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Response in the second course
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Assessment method [21]
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0
Percentage of participants with a platelet count of at least 50 G/L in the absence of rescue treatment/new ITP treatment.
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Timepoint [21]
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Second course Week 1 Day 1 to second course Week 25 Day 1
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Secondary outcome [22]
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Complete Response in the second course
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Assessment method [22]
0
0
Percentage of participants with a platelet count of at least 100 G/L in the absence of rescue treatment/new ITP treatment.
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Timepoint [22]
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0
Second course Week 1 Day 1 to second course Week 25 Day 1
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Secondary outcome [23]
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Bleeding events in the second course according to the Modified World Health Organization (WHO) bleeding scale
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Assessment method [23]
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Number of participants reporting bleeding events for each grade of the World Health Organization (WHO) bleeding scale at each time point. Severity is graded from 0 to 4, with 0 = no bleeding and 4 = severe hemodynamic instability/central nervous system (CNS) bleeding/fatal.
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Timepoint [23]
0
0
Second course Week 1 Day 1 to second course Week 25 Day 1
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Secondary outcome [24]
0
0
Percentage of participants with bleeding events in the retreatment/second courseaccording to the Modified World Health Organization (WHO) bleeding scale
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Assessment method [24]
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Percentage of participants reporting bleeding events for each grade of the WHO bleeding scale at each time point. Severity is graded from 0 to 4, with 0 = no bleeding and 4 = severe hemodynamic instability/central nervous system (CNS) bleeding/fatal.
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Timepoint [24]
0
0
Second course Week 1 Day 1 to second course Week 25 Day 1
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Secondary outcome [25]
0
0
Number of Participants who received rescue treatment after second course
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Assessment method [25]
0
0
Number of participants who required rescue treatment
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Timepoint [25]
0
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Second course Week 1 Day 1 to second course Week 25 Day 1
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Secondary outcome [26]
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Percentage of participants who received rescue treatment after receiving second course
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Assessment method [26]
0
0
Percentage of participants who required rescue treatment
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Timepoint [26]
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Second course Week 1 Day 1 to second course Week 25 Day 1
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Secondary outcome [27]
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0
Titer of anti-ianalumab antibodies in serum (ADA assay) over time for second course
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Assessment method [27]
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0
Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants to assess the immunogenicity of ianalumab
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Timepoint [27]
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Second course Week 1 Day 1 until 20 weeks after last dose of ianalumab
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Secondary outcome [28]
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0
Change from start of second course in immunoglobulins
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Assessment method [28]
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Post-baseline immunoglobulin levels (change in titers of Total Ig, IgG, IgM, IgA) compared to retreatment baseline
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Timepoint [28]
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0
From second course Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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Secondary outcome [29]
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0
Change from start of second course in the absolute number of CD19+ B-cell counts
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Assessment method [29]
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0
Post-baseline absolute number of CD19+ B-cell counts compared to baseline
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Timepoint [29]
0
0
From second course Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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Secondary outcome [30]
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0
Change from start of second course to end of study in the absolute number of CD19+ B-cell counts
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Assessment method [30]
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0
Post-baseline absolute number of CD19+ B-cell counts compared to baseline
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Timepoint [30]
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From second course Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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Secondary outcome [31]
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0
Time to first occurrence of B-cell recovery defined as =80% of baseline =50 cells/µL
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Assessment method [31]
0
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Time to B-cell recovery defined as =80% of baseline or =50 cells/µL
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Timepoint [31]
0
0
From second course Week 1 Day 1 to end of study (until all participants have completed 24 months of safety follow-up since their last dose of ianalumab or discontinued the study earlier)
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Secondary outcome [32]
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Ianalumab serum concentrations over time in the second course
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Assessment method [32]
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Ianalumab concentration in serum over time, including end of infusion and concentration at trough.
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Timepoint [32]
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First dose (pre-dose, 2, 168, 336, 504, 672 hours post-dose); Subsequent doses (pre-dose and 2 hours post-dose); Last dose (pre-dose, 2 336, 672, 1344, 2016, 3360 hours post-dose) in the second course
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Eligibility
Key inclusion criteria
* 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.
* Confirmed diagnosis of primary ITP.
* Prior treatment with at least a corticosteroid (±IVIG) and a TPO-RA:
* Prior additional therapies are allowed; the corticosteroid or the TPO-RA do not need to be the last treatment.
* Prior response to IVIG/anti-D or a corticosteroid (platelet count =50 G/L) that was not maintained.
* At last ITP treatment, loss of response, insufficient response, no response or intolerance.
* Platelet count <30 G/L and assessed as needing treatment (per physician's discretion) at screening. If concomitant ITP medication is clinically indicated, the platelet assessment showing a value <30 G/L must be performed after at least 14 days on a stable dose of a corticosteroid or/and a TPO-RA (less than 10% variation from current dose) and continue stable thereafter.
Key exclusion criteria:
* Diagnosis of secondary thrombocytopenia.
* Platelet or whole blood transfusion, plasmapheresis, or use of any other rescue medications within 14 days before first ianalumab infusion.
* Participants with the following conditions at screening:
* Neutrophils <1000/mm3.
* Immunoglobulin G (IgG) <5 g/L
* Treatment with a B-cell depleting therapy (e.g., rituximab) or anti-B-cell Activating Factor of the TNF Family (BAFF) (e.g., belimumab) within 12 weeks prior to the first administration of ianalumab.
* Immunosuppressant drugs other than corticosteroids within 5 times the elimination half-life of the drug or 14 days before first ianalumab infusion, whichever is longer.
* Prior splenectomy.
Other protocol-defined inclusion/exclusion criteria may apply.
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
16/08/2023
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
19/03/2029
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Actual
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Sample size
Target
40
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
ACT,VIC
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Recruitment hospital [1]
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0
Novartis Investigative Site - Canberra
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Recruitment hospital [2]
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0
Novartis Investigative Site - Prahran
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Recruitment postcode(s) [1]
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0
2605 - Canberra
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Recruitment postcode(s) [2]
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0
3181 - Prahran
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Recruitment outside Australia
Country [1]
0
0
United States of America
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State/province [1]
0
0
District of Columbia
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Country [2]
0
0
United States of America
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State/province [2]
0
0
Massachusetts
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Country [3]
0
0
United States of America
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State/province [3]
0
0
New York
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Country [4]
0
0
United States of America
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State/province [4]
0
0
Pennsylvania
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Country [5]
0
0
United States of America
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State/province [5]
0
0
Virginia
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Country [6]
0
0
Argentina
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State/province [6]
0
0
Buenos Aires
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Country [7]
0
0
China
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State/province [7]
0
0
Hubei
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Country [8]
0
0
China
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State/province [8]
0
0
Beijing
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Country [9]
0
0
China
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State/province [9]
0
0
Jinan
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Country [10]
0
0
Czechia
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State/province [10]
0
0
Czech Republic
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Country [11]
0
0
France
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State/province [11]
0
0
Dijon
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Country [12]
0
0
France
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State/province [12]
0
0
Toulouse
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Country [13]
0
0
Germany
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State/province [13]
0
0
Dresden
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Country [14]
0
0
Germany
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State/province [14]
0
0
Giessen
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Country [15]
0
0
Germany
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State/province [15]
0
0
Jena
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Country [16]
0
0
Italy
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State/province [16]
0
0
FI
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Country [17]
0
0
Italy
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State/province [17]
0
0
TO
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Country [18]
0
0
Italy
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State/province [18]
0
0
TS
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Country [19]
0
0
Korea, Republic of
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State/province [19]
0
0
Seocho Gu
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Country [20]
0
0
Korea, Republic of
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State/province [20]
0
0
Seoul
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Country [21]
0
0
Malaysia
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State/province [21]
0
0
Sabah
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Country [22]
0
0
Malaysia
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State/province [22]
0
0
Sarawak
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Country [23]
0
0
Malaysia
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State/province [23]
0
0
Johor Bahru
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Country [24]
0
0
Poland
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State/province [24]
0
0
Katowice
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Country [25]
0
0
Poland
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State/province [25]
0
0
Krakow
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Country [26]
0
0
Spain
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State/province [26]
0
0
Andalucia
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Country [27]
0
0
Spain
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State/province [27]
0
0
Catalunya
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Country [28]
0
0
Spain
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State/province [28]
0
0
Madrid
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Country [29]
0
0
Turkey
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State/province [29]
0
0
TUR
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Country [30]
0
0
Turkey
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State/province [30]
0
0
Aydin
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Country [31]
0
0
Turkey
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State/province [31]
0
0
Edirne
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Country [32]
0
0
Turkey
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State/province [32]
0
0
Izmir
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Country [33]
0
0
Turkey
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State/province [33]
0
0
Kocaeli
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Country [34]
0
0
United Kingdom
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State/province [34]
0
0
Glasgow
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Country [35]
0
0
United Kingdom
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State/province [35]
0
0
London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Novartis Pharmaceuticals
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
The purpose of this study is to evaluate the therapeutic efficacy, safety and tolerability of ianalumab in adult patients with primary ITP previously treated with at least one corticosteroid and one TPO-RA.
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Trial website
https://clinicaltrials.gov/study/NCT05885555
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
0
0
Novartis Pharmaceuticals
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Address
0
0
Novartis Pharmaceuticals
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Country
0
0
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Phone
0
0
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Fax
0
0
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Email
0
0
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Contact person for public queries
Name
0
0
Novartis Pharmaceuticals
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Address
0
0
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Country
0
0
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Phone
0
0
1-888-669-6682
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Fax
0
0
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Email
0
0
[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
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What data in particular will be shared?
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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When will data be available (start and end dates)?
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Available to whom?
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Available for what types of analyses?
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How or where can data be obtained?
IPD available at link: https://www.clinicalstudydatarequest.com
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT05885555