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Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP)

PHASE3RECRUITING

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 maintaining platelet count ≥30 G/L in adult participants with primary ITP.

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Study details:

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.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

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 below 30 G/L before starting any first-line ITP therapy (corticosteroids, IVIG)

Response (platelet count >=50 G/L) to corticosteroids (+/- IVIG) at any time prior to randomization. Note: Platelet count measured within 7 days of platelet transfusion will not be considered as response.

Exclusion criteria

Evans syndrome or any other cytopenia (patients with anemia related to bleeding or iron deficiency are eligible)

Current life-threatening bleeding

Previous ITP treatment, including splenectomy, except for corticosteroids and/or IVIG initiated as first-line therapy for up to 28 days before randomization and rescue corticosteroids and/or IVIG given prior to confirmed diagnosis of primary ITP.

Prior use of B-cell depleting therapy (e.g., rituximab).

Absolute neutrophil count below 1.0 G/L at randomization

Participants with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication with an exemption of low dose of acetylsalicylic acid

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Eligibility

Age eligible for study : 18 and older

Healthy volunteers accepted : No

Gender eligible for study: All

Things to know

Study dates

Study start: 2023-03-06

Primary completion: 2026-04-24

Study completion finish: 2028-12-18

Study type

TREATMENT

Phase

PHASE3

Trial ID

NCT05653349

Intervention or treatment

BIOLOGICAL: Ianalumab

DRUG: Placebo

DRUG: Corticosteroids

Conditions

• Primary Immune Thrombocytopenia (ITP)

Image related to Primary Immune Thrombocytopenia (ITP)

Condition: Primary Immune Thrombocytopenia (ITP)
BIOLOGICAL: Ianalumab and other drugs
Canberra, Australian Capital Territory, Australia and more
Sponsor: Novartis Pharmaceuticals

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Find a site

Closest Location:

Novartis Investigative Site

Research sites nearby

Select from list below to view details:

Novartis Investigative Site
Canberra, Australian Capital Territory, Australia
Novartis Investigative Site
Clayton, Victoria, Australia
Novartis Investigative Site
Melbourne, Victoria, Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: Ianalumab Lower dose Lower dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified)	BIOLOGICAL: Ianalumab Intravenous infusion, prepared from concentrate solution
EXPERIMENTAL: Ianalumab Higher dose Higher dose of ianalumab administered intravenously with corticosteroids oral or parental (if clinically justified)	BIOLOGICAL: Ianalumab Intravenous infusion, prepared from concentrate solution
PLACEBO_COMPARATOR: Placebo Placebo administered intravenously with corticosteroids oral or parental (if clinically justified)	DRUG: Placebo Intravenous infusion, prepared from matching placebo

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
Time from randomization to treatment failure (TTF)	Time from randomization until platelet count below 30 G/L, need for a rescue treatment or start of a second-line therapy or death.	Randomization to end of study (up to 39 months after randomization of last patient)

Secondary outcome

Secondary Outcome Measure	Secondary Outcome Description	Secondary Outcome Time Frame
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.	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.	Randomization to end of study (up to 39 months after randomization of last patient)
Time to complete response in each treatment group	Time from randomization to date of first complete response.	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	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	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	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	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	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.	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.	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).	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	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	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.	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	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, defined as ≥80% of baseline or ≥50 cells/μL	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	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)	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)	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	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	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	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	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	Up to Week 33

Frequently Asked Questions

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References

Clinical Trials Gov: Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP)