Primary Immune Thrombocytopenia (ITP)Phase 2NCT05885555
A Study of Ianalumab (VAY736) in Patients With Primary Immune Thrombocytopenia (ITP) Previously Treated With at Least Two Lines of Therapies
A Phase 2 study testing Ianalumab in people with Primary Immune Thrombocytopenia (ITP), run by Novartis Pharmaceuticals at 24 sites in 13 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Primary Immune Thrombocytopenia (ITP)
- What is being tested?
- Ianalumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 41 participants
- Where
- 24 sitesin 13 countries
- Study dates
- 16 Aug 2023to 19 Mar 2029
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
24 sites in 13 countries.
24 sites in 13 countries
Turkey (Türkiye)5 sites
Novartis Investigative Site
Aydin, Efeler, 09100
Novartis Investigative Site
Edirne, Merkez, 22030
Novartis Investigative Site
Istanbul, Fatih, 34098
Novartis Investigative Site
Izmir, 35100
Novartis Investigative Site
Kocaeli, 41380
China3 sites
Novartis Investigative Site
Beijing, 100730
Novartis Investigative Site
Jinan, 250012
Novartis Investigative Site
Wuhan, Hubei, 430022
Germany3 sites
Novartis Investigative Site
Dresden, Saxony, 01307
Novartis Investigative Site
Giessen, 35392
Novartis Investigative Site
Jena, Thuringia, 07740
United States2 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
Beth Israel Deaconess Med Center
Boston, Massachusetts, 02215
Australia2 sites
Novartis Investigative Site
Garran, Australian Capital Territory, 2605
Novartis Investigative Site
Melbourne, Victoria, 3004
United Kingdom2 sites
Novartis Investigative Site
Glasgow, G31 2ER
Novartis Investigative Site
London, W12 0HS
About this study
Official 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)
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.
Read the detailed description
This is a phase 2, open-label, single-arm study to evaluate the efficacy, safety and tolerability of ianalumab in participants with primary ITP (platelet count <30 G/L at screening) previously treated with at least a corticosteroid and a TPO-RA.
The study consists of the screening period, the primary endpoint assessment period, the follow-up period. The screening period will last for up to 14 days prior to the first dose of ianalumab. All eligible participants will be treated with the same dose of ianalumab and will complete the primary endpoint assessment period. After completion of the primary endpoint assessment period, all participants will continue in safety monitoring and those with a platelet count ≥30 G/L in absence of a new line of ITP therapy and rescue therapy will also continue in efficacy monitoring. The trial includes an option to offer a second course of ianalumab treatment to participants who achieved confirmed response during the initial course of ianalumab and later lost response to explore the benefit of the second course of treatment. The study will end once all participants have completed 24 months of safety follow-up since their last dose of ianalumab (including the optional second course of ianalumab treatment),or discontinued the study earlier.
- Conditions
- Primary Immune Thrombocytopenia (ITP)
- Being tested
- Ianalumab
- Keywords
- Primary immune thrombocytopenia (ITP), ianalumab, VAY736, B-cell depletion, B-cell Activating Factor Receptor (BAFF-R) blockade
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IanalumabBiological
Also called VAY736
Intravenous infusion, prepared from concentrate solution
| Study group | Role | What they receive |
|---|---|---|
| Single-arm | Experimental | All eligible participants will receive ianalumab at the same dose. Receives: Biological: Ianalumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Confirmed response
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.
Measured: Between Week 1 Day 1 and Week 25 Day 1
Secondary outcomes (35)
Time to confirmed response
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.
Measured: From Week 1 Day 1 to Week 25 Day 1
Duration of confirmed response
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).
Measured: 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)
Complete Response rate at each timepoint
Percentage of participants with a platelet count of at least 100 G/L in the absence of rescue treatment/new ITP treatment.
Measured: 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)
Response rate at each timepoint
Percentage of participants with a platelet count of at least 50 G/L in the absence of rescue treatment/new ITP treatment.
Measured: 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)
Stable response at 6 months
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.
Measured: At 6 months
Stable response at 1 year
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.
Measured: At 1 year
Bleeding events according to the Modified World Health Organization (WHO) bleeding scale
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.
Measured: 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)
Percentage of participants with bleeding events according to the Modified World Health Organization (WHO) bleeding scale
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.
Measured: 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)
Number of participants who received rescue treatment
Number of participants who required rescue treatment
Measured: 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)
Percentage of participants who received rescue treatment
Percentage of participants who required rescue treatment
Measured: 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)
Change from baseline in the frequency of CD19+ B-cell counts
Post-baseline frequency of CD19+ B-cell counts compared to baseline
Measured: 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)
Change from baseline in the absolute number of CD19+ B-cell counts
Post-baseline absolute number of CD19+ B-cell counts compared to baseline
Measured: 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)
Time to first occurrence of B-cell recovery defined as ≥80% of baseline ≥50 cells/µL
Time to B-cell recovery defined as ≥80% of baseline or ≥50 cells/µL
Measured: 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)
Change from baseline in immunoglobulins
Post-baseline immunoglobulin levels (change in titers of Total Ig, IgG, IgM, IgA) compared to baseline
Measured: 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)
Incidence of anti-ianalumab antibodies in serum (ADA assay) over time
Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants to assess the immunogenicity of ianalumab
Measured: Up to 20 weeks after last dose of ianalumab
Titer of anti-ianalumab antibodies in serum (ADA assay) over time
Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants to assess the immunogenicity of ianalumab
Measured: Up to 20 weeks after last dose of ianalumab
Ianalumab serum concentrations over time
Ianalumab concentration in serum over time, including end of infusion and concentration at trough.
Measured: 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)
Ianalumab serum PK: AUC concentrations over time
Ianalumab concentration in serum over time, including end of infusion and concentration at trough.
Measured: 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)
Ianalumab serum PK parameters: Cmax
Maximum (peak) observed plasma, blood, serum or other body fluid drug concentration
Measured: 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)
Ianalumab serum PK parameters: Tmax
Time to reach maximum (peak) observed plasma, blood, serum or other body fluid drug concentration
Measured: 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)
Confirmed response (CR) in the second course
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.
Measured: Second course Week 1 Day1 to second course Week 25 Day1
Time to confirmed response in the second course
two (or more) platelet assessments meeting the criteria of a confirmed response.
Measured: Second course Week 1 Day 1 to second course Week 25 Day 1
Duration of confirmed response in the second course
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).
Measured: Second course Week 1 Day 1 to second course Week 25 Day 1
Response in the second course
Percentage of participants with a platelet count of at least 50 G/L in the absence of rescue treatment/new ITP treatment.
Measured: Second course Week 1 Day 1 to second course Week 25 Day 1
Complete Response in the second course
Percentage of participants with a platelet count of at least 100 G/L in the absence of rescue treatment/new ITP treatment.
Measured: Second course Week 1 Day 1 to second course Week 25 Day 1
Bleeding events in the second course according to the Modified World Health Organization (WHO) bleeding scale
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.
Measured: Second course Week 1 Day 1 to second course Week 25 Day 1
Percentage of participants with bleeding events in the retreatment/second courseaccording to the Modified World Health Organization (WHO) bleeding scale
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.
Measured: Second course Week 1 Day 1 to second course Week 25 Day 1
Number of Participants who received rescue treatment after second course
Number of participants who required rescue treatment
Measured: Second course Week 1 Day 1 to second course Week 25 Day 1
Percentage of participants who received rescue treatment after receiving second course
Percentage of participants who required rescue treatment
Measured: Second course Week 1 Day 1 to second course Week 25 Day 1
Titer of anti-ianalumab antibodies in serum (ADA assay) over time for second course
Anti-drug antibodies (ADA) will be evaluated in samples collected from all participants to assess the immunogenicity of ianalumab
Measured: Second course Week 1 Day 1 until 20 weeks after last dose of ianalumab
Change from start of second course in immunoglobulins
Post-baseline immunoglobulin levels (change in titers of Total Ig, IgG, IgM, IgA) compared to retreatment baseline
Measured: 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)
Change from start of second course in the absolute number of CD19+ B-cell counts
Post-baseline absolute number of CD19+ B-cell counts compared to baseline
Measured: 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)
Change from start of second course to end of study in the absolute number of CD19+ B-cell counts
Post-baseline absolute number of CD19+ B-cell counts compared to baseline
Measured: 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)
Time to first occurrence of B-cell recovery defined as ≥80% of baseline ≥50 cells/µL
Time to B-cell recovery defined as ≥80% of baseline or ≥50 cells/µL
Measured: 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)
Ianalumab serum concentrations over time in the second course
Ianalumab concentration in serum over time, including end of infusion and concentration at trough.
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jun 2023
- Study start16 Aug 2023
- Primary completion5 Feb 2025
- Study completion19 Mar 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immune Thrombocytopenia (ITP)Studies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05885555
- Sponsor's protocol ID
- CVAY736Q12201
- Other IDs
- 2022-503041-21-00
- Status last verified
- Oct 2026
- First submitted
- 15 May 2023
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
- Expanded access
- Available