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 increasing the rate of stable response off treatment at 12 months (SROT-12) in adult participants with primary ITP.
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. The efficacy and safety follow-up period will last until treatment failure or up to 39 months after randomization of the last patient, whichever occurs first. Safety follow-up period will be performed for at least 20 weeks and up to 2 years after the last ianalumab/placebo dose. The primary endpoint was updated in May 2026 to stable response off treatment at 12 months (SROT-12). The previous primary endpoint, time to treatment failure (TTF), remains in the study as a key secondary objective.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
226
Intravenous infusion, prepared from concentrate solution
Intravenous infusion, prepared from matching placebo
Oral or parental (if clinically justified)
Rate of Stable Response off treatment at 12 months (SROT-12)
A participant will be considered in SROT-12 if at least 75% of the platelet counts collected between visits Week 53 Day 1 and Week 65 Day 1 (Study Days 352 and 463) qualify as Response. Response is any platelet counts of at least 50 G/L in the absence of rescue treatment or new immune thrombocytopenia (ITP) treatment).
Time frame: 12 months after last patient completed treatment
Time from randomization to Treatment Failure (TTF) (Key Secondary Endpoint)
Time to Treatment Failure (TTF) is defined as the time from randomization until the first of the following events/situation indicative of treatment failure: platelet count below 30 G/L later than 8 weeks from randomization, need for a rescue treatment later than 8 weeks from randomization, intake of rescue treatment (e.g., corticosteroids, Intravenous immunoglobulins (IVIG), or platelet transfusion) given later than 8 weeks from randomization, start of new Immune Thrombocytopenia (ITP) treatment whenever it occurs, death (whatever the cause).
Time frame: Randomization to end of study (up to 39 months after randomization of last patient
Complete Response (CR) rate in each treatment group
Complete Response (CR) rate at each timepoint defined as the proportion of participants with any platelet count of at least 100 G/L in the absence of rescue treatment or new ITP treatment.
Time frame: Randomization to end of study (up to 39 months after randomization of last patient)
Response (R) rate in each treatment group
Response (R) rate at each timepoint defined as the proportion of participants with any platelet count of at least 50 G/L in the absence of rescue treatment or new ITP treatment.
Time frame: Randomization to end of study (up to 39 months after randomization of last patient)
Time to complete response in each treatment group
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Time from randomization to date of first complete response.
Time frame: Randomization to end of study (up to 39 months after randomization of last patient)
Duration of response in each treatment group
Time from achievement of complete response to loss of complete response
Time frame: Randomization to end of study (up to 39 months after randomization of last patient)
Stable response at 6 months
Percentage of participants with at least 2 platelet count collected at month 6 (between study dates 107 and 183) and at least 66% of platelet counts qualified as a response
Time frame: At 6 months
Stable response at 1 year
Percentage of participants with at least 2 platelet counts collected at year 1 (between study days 296 and 379) and at least 66% of platelet counts qualified as a response
Time frame: At 1 year
Percentage of participants with bleeding events overall and by World Health Organization (WHO) bleeding scale severity
This is to assess the incidence and severity of bleeding in each treatment arm
Time frame: Randomization to end of study (up to 39 months after randomization of last patient)
Number of participants with bleeding events overall and by World Health Organization (WHO) bleeding scale severity
This is to assess the number and severity of bleeding in each treatment arm
Time frame: Randomization to end of study (up to 39 months after randomization of last patient)
Number of participants receiving rescue treatment (cummulative dose/duration of steroids exposure)
This is to assess the number of participants receiving rescue treatment.
Time frame: Randomization to end of study (up to 39 months after randomization of last patient)
Percentage of participants receiving rescue treatment (cummulative dose/duration of steroids exposure)
This is to assess the need of rescue treatment in each treatment group by percentage.
Time frame: Randomization to end of study (up to 39 months after randomization of last patient)
Cumulative dose/duration of steroids exposure
Duration of exposure to corticosteroids calculated from randomization (first dose) to end of study or last last contact date (if the participant is lost to follow-up).
Time frame: From screening to end of study (up to 39 months after randomization of last patient)
Change from baseline on T scores of the PROMIS SF v1.0 Fatigue 13a
The Patient-Reported Outcomes Measurement Information System (PROMIS) Short Form v1.0 Fatigue 13a includes 13 items that assess fatigue
Time frame: From screening (baseline) till end of study (up to 39 months after randomization of last patient)
Change from baseline in ITP-PAQ domain scores
The ITP-PAQ is a 44 item scale for measuring HRQoL in adults with ITP across ten scales: Symptoms, Bother-Physical Health, Fatigue/Sleep, Activity, Fear, Psychological Health, Work, Social Activity, Women's Reproductive Health, and Overall QoL. Each item is rated on a Likert type scale
Time frame: From screening (baseline) till end of study (up to 39 months after randomization of last patient)
Change from baseline in frequency of CD19+ B cell counts
Post baseline frequency (%within the CD45) of CD19+ B cell counts compare to baseline.
Time frame: Randomization to end of study (up to 39 months after randomization of last patient)
Change from baseline in absolute number of CD19+ B cell counts
Post baseline absolute number of CD19+ B cell counts compare with baseline
Time frame: Randomization to end of study (up to 39 months after randomization of last patient)
Time to first occurrence of B-cell recovery
B-cell recovery is defined as ≥80% of baseline and ≥LLOQ, or ≥50 cells/μL
Time frame: Randomization to end of study (up to 39 months after randomized of last patient)
Change from baseline in inmmunoglobulins
Change from baseline in immunoglobulin levels
Time frame: Randomization to end of study (up to 39 months after last randomized patients)
PK parameters: AUClast
AUClast: area under the curve from time zero till the last measurable concentration sampling time (tlast)
Time frame: After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
PK parameter: AUCtau
Area under the curve calculated to the end of a dosing interval (tau)
Time frame: After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
PK parameters: Cmax
Maximum (peak) observed plasma, blood, serum, or other body fluid drug concentration after single dose administration
Time frame: After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
PK parameters: Tmax
Time to reach maximum (peak) plasma, blood, serum, or other body fluid drug concentration
Time frame: After first dose (pre-dose, 2, 168, 336 and 504 hours post dose) and after last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
PK parameters: Accumulation ratio Racc
Accumulation ratio calculated using AUC values obtained between the last and first dose
Time frame: After last dose (pre-dose, 2, 336, 672, 1344, 2016, 3360 hours post dose)
Incidence of anti-ianalumab antibodies in serum (ADA assay) over time
Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants assess the immunogenicity of ianalumab
Time frame: Up to Week 33
Titer of anti-ianalumab antibodies in serum (ADA assay) over time
Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants assess the immunogenicity of ianalumab
Time frame: Up to Week 33