Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05653349 | Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP) | PHASE3 | ACTIVE NOT_RECRUITING | 226 | — | — | Feb 3, 2023 | Dec 6, 2028 | Apr 22, 2026 | 114 | United States, Argentina +23 |
| NCT07421167 | A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP) | PHASE2 | RECRUITING | 164 | — | — | Nov 11, 2026 | Mar 31, 2027 | May 8, 2026 | 2 | United States |
| NCT05885555 | A Study of Ianalumab (VAY736) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least Two Lines of Therapies | PHASE2 | ACTIVE NOT_RECRUITING | 41 | — | — | Aug 16, 2023 | Mar 19, 2029 | Apr 13, 2026 | 24 | United States, Argentina +11 |
Time from randomization until platelet count below 30 G/L, need for a rescue treatment or start of a second-line therapy or death.
The proportion of participants who tolerate ianalumab (9 mg/kg) is defined as those who do not experience any of the following events during the combination treatment period (up to Week 16): * Discontinuation due to an adverse event (AE) unrelated to efficacy * Adverse events leading to dose reduction or dose rate reduction * Adverse events resulting in study drug interruption.
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.
| Arm | Type | Description |
|---|---|---|
| Ianalumab Lower dose | EXPERIMENTAL | Lower dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified) |
| Ianalumab Higher dose | EXPERIMENTAL | Higher dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified) |
| Placebo | PLACEBO_COMPARATOR | Placebo administered intravenously with corticosteroids oral or parental (if clinically justified) |
| Main cohort: Primary immune thrombocytopenia (ITP) | EXPERIMENTAL | All participants will be assigned to ianalumab 9 mg/kg plus investigator's choice thrombopoietin receptor agonist (IC TPO-RA). |
| Exploratory cohort: Primary Evans syndrome (ES) | EXPERIMENTAL | All participants will be assigned to ianalumab 9 mg/kg plus investigator's choice thrombopoietin receptor agonist (IC TPO-RA). |
| Single-arm | EXPERIMENTAL | All eligible participants will receive ianalumab at the same dose. |
| Name | Type | Description |
|---|---|---|
| Ianalumab | BIOLOGICAL | Intravenous infusion, prepared from concentrate solution |
| Placebo | DRUG | Intravenous infusion, prepared from matching placebo |
| Corticosteroids | DRUG | Oral or parental (if clinically justified) |
| thrombopoietin receptor agonist (TPO-RA) | DRUG | IC TPO-RAs will be administered according to the respective United States Prescribing Information (USPIs) |
Inclusion 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 b...