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Ianalumab

Phase 3

Primary Immune Thrombocytopenia (ITP) | Monoclonal antibody | Hematology |Novartis AG|Last Updated: Jul 17, 2026

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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials4
Total Enrollment467
FDA Designations
No designations recorded
Clinical trial landscape

Ianalumab · 15 trials · 11 indications

Phase 3 8Phase 2 6Phase 1 1
NCT06711887Phase III Extension Study of Efficacy and Safety of Ianalumab With or Without Study Treatment Withdrawal in Participants With Lupus Nephritis (SIRIUS-LN Extension)Lupus Nephritis
RECRUITING348 Analytics
NCT06133972Phase 3 Extension Study to Evaluate Long-term Safety of Ianalumab in Participants With Systemic Lupus Erythematosus (SIRIUS-SLE Extension).Systemic Lupus Erythematosus
RECRUITING550 Analytics
NCT05985915A Randomized, Double-blind 2-arm, Followed by an Open-label 1-arm, NEPTUNUS Extension Study to Assess the Long-term Safety and Efficacy of Ianalumab in Patients With Sjogrens Syndrome.Sjogrens Syndrome
ACTIVE NOT_RECRUITING612 Analytics
NCT05624749Phase 3 Study to Evaluate Ianalumab on Top of Standard-of-care Therapy in Patients With Systemic Lupus Erythematosus (SIRIUS-SLE 2)Systemic Lupus Erythematosus
ACTIVE NOT_RECRUITING288 Analytics
NCT05639114Phase 3 Study to Evaluate Two Regimens of Ianalumab on Top of Standard-of-care Therapy in Patients With Systemic Lupus Erythematosus (SIRIUS-SLE 1)Systemic Lupus Erythematosus
ACTIVE NOT_RECRUITING436 Analytics
NCT05653349Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP)Primary Immune Thrombocytopenia (ITP)
ACTIVE NOT_RECRUITING226 Analytics
NCT05653219A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed SteroidsPrimary Immune Thrombocytopenia
ACTIVE NOT_RECRUITING152 Analytics
NCT05648968A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune Hemolytic AnemiaWarm Autoimmune Hemolytic Anemia (wAIHA)
ACTIVE NOT_RECRUITING90 Analytics
PHASE3RECRUITING
Phase III Extension Study of Efficacy and Safety of Ianalumab With or Without Study Treatment Withdrawal in Participants With Lupus Nephritis (SIRIUS-LN Extension)
Lupus NephritisUnlock trial analytics
PHASE3RECRUITING
Phase 3 Extension Study to Evaluate Long-term Safety of Ianalumab in Participants With Systemic Lupus Erythematosus (SIRIUS-SLE Extension).
Systemic Lupus ErythematosusUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Randomized, Double-blind 2-arm, Followed by an Open-label 1-arm, NEPTUNUS Extension Study to Assess the Long-term Safety and Efficacy of Ianalumab in Patients With Sjogrens Syndrome.
Sjogrens SyndromeUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
Phase 3 Study to Evaluate Ianalumab on Top of Standard-of-care Therapy in Patients With Systemic Lupus Erythematosus (SIRIUS-SLE 2)
Systemic Lupus ErythematosusUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
Phase 3 Study to Evaluate Two Regimens of Ianalumab on Top of Standard-of-care Therapy in Patients With Systemic Lupus Erythematosus (SIRIUS-SLE 1)
Systemic Lupus ErythematosusUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP)Unlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids
Primary Immune ThrombocytopeniaUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune Hemolytic Anemia
Warm Autoimmune Hemolytic Anemia (wAIHA)Unlock trial analytics
Study Endpoints
Primary Endpoints
Cohort 1: Time to renal flare, increase in immunosuppressive medication, or death
Between Week 144E1 and EOS (up to Week 456)

Cohort 1: To estimate the time to renal flare, increase in immunosuppressive medication, or death following study treatment withdrawal in participants who completed the SIRIUS-LN core study on double-blind treatment and achieved CRR or PRR at Week 140 of the core study

Cohort 2: Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
From the start of treatment in the SIRIUS-LN core study to EOS (up to week 560) of the extension study

Number of participants with AEs and SAEs, including changes in vital signs and clinical laboratory measurements qualifying and reported as AEs.

Number of treatment-emergent Adverse events/Serious Adverse events
through study completion, up to approximately 91 months

Assessment of long-term safety and tolerability of ianalumab

Number of Treatment-emergent AEs (TEAEs)/SAEs
Week 52 to Week 464

Assessment of safety and tolerability of ianalumab (VAY736) in patients with active Sjogrens syndrome

Proportion of participants achieving Systemic Lupus Erythematosus Responder Index -4 (SRI-4)
Week 60

SRI-4 response is defined as: * Systemic Lupus Erythematosus Disease Activity Index - 2000 (SLEDAI-2K) reduction from baseline of ≥ 4 points * No British Isles Lupus Assessment Group-2004 (BILAG-2004) worsening, defined as ≥ 1 new A or ≥ 2 new B items compared to baseline * No worsening in Physician Global Assessment of Disease Activity (PhGA), defined as an increase of ≥ 0.3 from baseline on a 0 to 3 visual analog scale

Proportion of participants on monthly ianalumab achieving Systemic Lupus Erythematosus Responder Index -4 (SRI-4)
Week 60

SRI-4 response is defined as: * Systemic Lupus Erythematosus Disease Activity Index - 2000 (SLEDAI-2K) reduction from baseline of ≥ 4 points * No British Isles Lupus Assessment Group-2004 (BILAG-2004) worsening, defined as ≥ 1 new A or ≥ 2 new B items compared to baseline * No worsening in Physician Global Assessment of Disease Activity (PhGA), defined as an increase of ≥ 0.3 from baseline on a 0 to 3 visual analog scale

Rate of Stable Response off treatment at 12 months (SROT-12)
12 months after last patient completed treatment

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 from randomization until treatment failure
Randomization to until end of study (up to 39 months after randomization of last participant)

Time from randomization until treatment failure is defined as the time from randomization date until the first of the following events indicative of treatment failure: * platelet count below 30 G/L * start of a new ITP treatment * need for a rescue treatment * ineligibility to taper or inability to discontinue eltrombopag * death

Binary variable indicating whether a patient achieves a durable response
Randomization to Week 25

Durable response: hemoglobin level ≥10 g/dL and ≥2 g/dL increase from baseline, for a period of at least eight consecutive weeks between W9 and W25, in the absence of rescue medication or prohibited treatment

Time to treatment failure (TTF)
enrolled until end of study (up to 39 months from the last patient enrolled)

TTF is defined as the time from start of treatment until any of a list of specific events is met.

(Main cohort: Primary immune thrombocytopenia (ITP)): Percentages of participants who are tolerable to ianalumab (9 mg/kg)
Up to Week 16

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.

Treatment failure free (yes, no) for participants with Immune Thrombocytopenia (ITP)
From treatment start date until the events indicative of treatment failure (up to 2 years after last dose)

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)
At least 8 consecutive weeks, between week 9 and week 25

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.

3/5 rCRISS25 response
Week 52

To demonstrate the superiority of ianalumab, compared to placebo, in achieving 3/5 Revised Composite Response Index in Systemic Sclerosis 25 (rCRISS25) response at Week 52

Confirmed response
Between Week 1 Day 1 and Week 25 Day 1

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.

Change in logarithm of salivary gland B/B+T cell ratio
Week 25

Change from baseline in logarithm of salivary gland B/B+T cell ratio at Week 25 (EOT)

Safety and tolerability as measured by the number of patients with adverse events
Week 0 - 112

The number of patients with adverse events after repeated subcutaneous (s.c) injections of a fixed dose of ianalumab

Pharmacokinetic comparability at steady state - AUCtau
Week 8 - 12

The area under the serum ianalumab concentration-time curve from time zero to the end of the dosing interval (AUCtau)

Pharmacokinetic comparability at steady state - Cmax
Week 8 - 12

Observed maximum serum concentration of ianalumab following drug administration (Cmax)

Secondary Endpoints
Cohort 2: Incidence and titer of anti-ianalumab antibodies in serum (ADA assay)
from Week 144E1 up to Week 456
Cohort 2: Ianalumab concentration in serum using a validated ELISA
from Week 144E1 up to Week 456
Cohort 1: Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
From the start of treatment in the SIRIUS-LN core study to EOS (up to week 560) of the extension study
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Study Treatment Withdrawal (cohort 1)NO_INTERVENTION(Cohort 1): Study Treatment-Withdrawal group. Participants who received blinded study treatment in the core study and achieve CRR or PRR at Week 140 in the core study will enter the extension study and discontinue study treatment while maintaining standard of care (SoC) medication, as required. If renal flare criteria are met, participants will be eligible to receive open-label ianalumab.
Open-Label Ianalumab (cohort 2)EXPERIMENTAL(Cohort 2): Open-Label Ianalumab group: Participants who received blinded study treatment throughout the core study and did not achieve CRR or PRR at Week 140 of the core study, or who had switched to open-label ianalumab during the core study, will be eligible or will continue to receive open-label ianalumab in the extension study.
Ianalumab monthlyEXPERIMENTALIanalumab s.c. monthly
Ianalumab quarterlyEXPERIMENTALIanalumab s.c. quarterly
Placebo monthlyPLACEBO_COMPARATORPlacebo s.c. monthly
Ianalumab 3 MonthlyEXPERIMENTALIanalumab 300 mg s.c. every three months and placebo once monthly between doses during initial double-blind treatment period. Open-label treatment and receive ianalumab 300 mg monthly.
ianalumab s.c. monthlyEXPERIMENTALianalumab s.c. monthly
placebo s.c. monthlyPLACEBO_COMPARATORplacebo s.c. monthly
Ianalumab s.c. quarterlyEXPERIMENTALIanalumab s.c. quarterly
Ianalumab Lower doseEXPERIMENTALLower dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified)
Ianalumab Higher doseEXPERIMENTALHigher dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified)
PlaceboPLACEBO_COMPARATORPlacebo administered intravenously with corticosteroids oral or parental (if clinically justified)
Treatment arm 1 - Lower doseEXPERIMENTALParticipants will receive eltrombopag and ianalumab lower dose
Treatment arm 2 - Higher doseEXPERIMENTALParticipants will receive eltrombopag and ianalumab higher dose
Treatment arm 3 - PlaceboPLACEBO_COMPARATORParticipants will receive eltrombopag and placebo
Ianalumab low doseEXPERIMENTALParticipants will receive low dose ianalumab intravenously
Ianalumab high doseEXPERIMENTALParticipants will receive high dose ianalumab intravenously
Ianalumab + eltrombopagEXPERIMENTALParticipants will receive ianalumab in addition to eltrombopag.
Main cohort: Primary immune thrombocytopenia (ITP)EXPERIMENTALAll 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)EXPERIMENTALAll participants will be assigned to ianalumab 9 mg/kg plus investigator's choice thrombopoietin receptor agonist (IC TPO-RA).
Treatment arm 1EXPERIMENTALParticipants will receive ianalumab lower dose
Treatment arm 2EXPERIMENTALParticipants will receive ianalumab higher dose
VAY736 (Ianalumab)EXPERIMENTALTreatment Period 1: Ianalumab subcutaneous (s.c.) injection as defined in the protocol Treatment Period 2: Open-label (OL) Ianalumab subcutaneous (s.c.) injection as defined in the protocol
Single-armEXPERIMENTALAll eligible participants will receive ianalumab at the same dose.
Treatment ArmEXPERIMENTALIanalumab 300 mg subcutaneous monthly
Reference VAY736 Drug ProductACTIVE_COMPARATORPowder for solution for injection / infusion
Test VAY736 Drug ProductEXPERIMENTALSolution for injection
Interventions
NameTypeDescription
IanalumabDRUGIanalumab (VAY736) is a human monoclonal antibody (mAb) of the IgG1/κ-class, directed against B cells and binding to BAFF receptor (BAFF-R).
PlaceboDRUGPlacebo s.c. monthly
Ianalumab (VAY736)DRUGIanalumab 300 mg / 2 mL, PFS or AI, monthly or every 3 months, solution for injection for subcutaneous use
CorticosteroidsDRUGOral or parental (if clinically justified)
EltrombopagDRUGFilm-coated tablet for oral use
thrombopoietin receptor agonist (TPO-RA)DRUGIC TPO-RAs will be administered according to the respective United States Prescribing Information (USPIs)
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Eligibility Criteria
Age Range18 Years to 100 Years
SexALL
Healthy VolunteersNo
Study Sites41

Inclusion Criteria: 1. Signed informed consent prior to participation in the extension study. 2. Participants must have participated in the SIRIUS-LN core study and must have completed the entire treatment up to Week 144 on double-blind or open label study treatment. Exclusion Criteria: 1. Use of...

Countries:United StatesBrazilChinaColombiaCzechiaGuatemalaHong KongHungaryMalaysiaMexicoRomaniaSingaporeSouth KoreaSpainTaiwanThailandArgentinaAustraliaBulgariaCanadaChileFranceGermanyIndiaIsraelItalyJapanPolandPortugalSlovakiaSouth AfricaAustriaBelgiumGreeceLithuaniaSwedenTurkey (Türkiye)United KingdomNorwayVietnamNetherlandsPhilippinesJordan
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Recent Changes (Last 90 Days)
LOWJul 17, 2026NCT07039422lastUpdatePostDate: changed
LOWJul 17, 2026NCT07039422lastUpdatePostDate: changed
LOWJul 14, 2026NCT07421167Completion: 2030-08-08 → 2030-08-10
LOWJul 14, 2026NCT07421167Completion: 2030-08-08 → 2030-08-10
LOWJul 10, 2026NCT06470048lastUpdatePostDate: changed
MEDIUMJul 10, 2026NCT06711887Enrollment: 315 → 348
MEDIUMJul 10, 2026NCT06711887Enrollment: 315 → 348
LOWJul 10, 2026NCT06470048lastUpdatePostDate: changed
LOWJul 9, 2026NCT07421167lastUpdatePostDate: changed
LOWJul 9, 2026NCT07421167lastUpdatePostDate: changed
LOWJul 8, 2026NCT06133972lastUpdatePostDate: changed
LOWJul 8, 2026NCT05985915lastUpdatePostDate: changed
LOWJul 8, 2026NCT06133972lastUpdatePostDate: changed
LOWJul 8, 2026NCT05985915lastUpdatePostDate: changed
LOWJul 2, 2026NCT07421167primaryCompletionDate: changed
LOWJul 2, 2026NCT07421167primaryCompletionDate: changed
LOWJul 2, 2026NCT07421167primaryCompletionDate: changed
LOWJun 26, 2026NCT07421167lastUpdatePostDate: changed
LOWJun 26, 2026NCT07421167lastUpdatePostDate: changed
LOWJun 25, 2026NCT07660172lastUpdatePostDate: changed