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| ID | Type | Description | Link |
|---|---|---|---|
| 2026-525995-25 | Registry Identifier | EU CTIS |
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The purpose of this study is to assess the efficacy, safety and pharmacokinetics (PK) of ianalumab (VAY736) in addition to eltrombopag treatment; and to inform the dose of ianalumab in pediatric patients (5 to <18 years of age) with primary ITP who have had an insufficient response to or relapsed after first-line treatment with corticosteroids.
The study will consist of 3 phases:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ianalumab + eltrombopag | Experimental | Participants will receive ianalumab in addition to eltrombopag. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ianalumab | Biological | Liquid in a vial Concentrate for solution for infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| Time to treatment failure (TTF) | TTF is defined as the time from start of treatment until any of a list of specific events is met. | enrolled until end of study (up to 39 months from the last patient enrolled) |
| Measure | Description | Time Frame |
|---|---|---|
| Stable response rate at 6 months (SR-6) | SR-6 is defined as the percentage of participants with a platelet count of at least 50 G/L at 75% of the measures taken between Study Day 121 and 183, in the absence of rescue or new Immune Thrombocytopenia (ITP) therapy. | 6 months |
| Stable response rate at 12 months (SR-12) |
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Inclusion Criteria:
Exclusion Criteria:
Other protocol-defined inclusion/exclusion may apply.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Novartis Pharmaceuticals | Contact | 1-888-669-6682 | novartis.email@novartis.com | |
| Novartis Pharmaceuticals | Contact | +41613241111 |
| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
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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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| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| C000656267 | ianalumab |
| C520809 | eltrombopag |
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| Eltrombopag | Drug | Film-coated tablet and also Powder for oral suspension |
|
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SR-12 is defined as the percentage of participants with a platelet count of at least 50 G/L at 66% of the measures taken between Study Day 296 and 379, in the absence of rescue or new ITP therapy. |
| 12 months |
| Complete Response (CR) rate | CR is defined as the percentage of participants with any platelet count of at least 100 G/L in the absence of rescue treatment or new ITP treatment. | from enrollment until end of study (up to 39 months from the last patient enrolled) |
| Response Rate (RR) | RR is defined as the percentage of participants with any platelet count of at least 50 G/L in the absence of rescue treatment or new ITP treatment. | from enrollment until end of study (up to 39 months from the last patient enrolled) |
| Time to response | Time to response is defined as the time from the start of treatment to the date of the first response. | from enrollment until end of study (up to 39 months from the last patient enrolled) |
| Time to complete response | Time to complete response is defined as the time from the start of treatment to the date of complete response. | from enrollment until end of study (up to 39 months from the last patient enrolled) |
| Duration of response | Duration of response is defined as the time from achievement of response (platelet count ≥ 50 G/L) to treatment failure. | from enrollment until end of study (up to 39 months from the last patient enrolled) |
| Duration of complete response | Duration of complete response is defined as the time from achievement of response to loss of complete response. | from enrollment until end of study (up to 39 months from the last patient enrolled) |
| Bleeding Events | Percentage of participants with bleeding events according to WHO Bleeding Scale | from enrollment until end of study (up to 39 months from the last patient enrolled) |
| Rescue medication | Number and percentage of participants receiving rescue treatment | from enrollment until end of study (up to 39 months from the last patient enrolled) |
| Rate of participants who successfully taper and discontinue eltrombopag - being treatment failure-free | Treatment failure-free (as defined for the primary efficacy endpoint) at the end of the planned Treatment period. | End of Week 24 |
| Ianalumab serum concentration (Cmax) | Cmax is the maximum (or peak) serum concentration that a drug achieves in a specified compartment or test area of the body. | at pre-dose of each of the 4 dosing events and end-of-infusion and first and last dose |
| B cell levels: Change from baseline in the frequency (% within CD45+ cells) and absolute number of CD19+ B cells | Number and percentage of B-cell levels in the blood, and comparison with baseline levels of B-cells at specific timepoints. | 12 months after the first treatment of the last enrolled participant, final dbl: until end of study (up to 39 months from the last patient enrolled) |
| B cell levels: Time to first occurrence of B cell recovery | Time to first occurrence of B cell recovery is defined as ≥80% of baseline or ≥50 cells/μL. | 12 months after the first treatment of the last enrolled participant, final dbl: until end of study (up to 39 months from the last patient enrolled) |
| Immunoglobulins: Change from baseline in immunoglobulin levels | Change from baseline levels of serum immunoglobulins at specific timepoints. | 12 months after the first treatment of the last enrolled participant, final dbl: until end of study (up to 39 months from the last patient enrolled) |
| Platelet counts: Change from baseline in platelet count | Change from baseline levels of platelets at specific timepoints. | 12 months after the first treatment of the last enrolled participant, final dbl: until end of study (up to 39 months from the last patient enrolled) |
| Incidence and titer of anti-ianalumab antibodies in serum (anti-drug-antibody (ADA) assay) | Anti-drug antibodies (ADAs) are immune responses developed by a participant's body against biologic or gene therapies. | over time (up to 39 months from the last patient enrolled) |
| D006425 |
| Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |