Primary Immune thrombocytopenia (ITP)
Conditions
Sponsors
Novartis Pharma AG
Eligibility
Sex/Gender
All
Inclusion criteria
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. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 169 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 56
Exclusion criteria
Exclusion criteria: -Evans syndrome or any other cytopenia -Current life-threatening bleeding -Previous ITP treatment, including splenectomy, except for corticosteroids and/or IVIG initiated as first-line therapy for up 28 days before randomization/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 Other protocol-defined Inclusion/Exclusion may apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate that the addition of ianalumab (either dose) to standard first-line corticosteroids prolongs Time to Treatment Failure (TTF) compared to corticosteroids alone in participants with primary ITP who responded to corticosteroids (+/-IVIG) prior to randomization ;Secondary Objective: - To assess quality of response, time to and duration of complete response in each treatment group - To assess the incidence and severity of bleeding in each treatment arm - To assess the need of rescue treatment in each treatment group - To evaluate treatment effects on ITP related symptoms, functioning, and health-related quality of life (HRQoL) - To assess B-cell and immunoglobulin levels in each treatment group - To assess ianalumab pharmacokinetics (PK) - To assess the immunogenicity of ianalumab ;Primary end point(s): Time from randomization to treatment failure (TTF) defined as platelet count below 30 G/L, need for a rescue treatment, start of a second-line therapy or death. ;Timepoint(s) of evaluation of this end point: Randomization to end of study (up to 39 months after randomization of last patient) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Complete Response (CR) rate in each treatment group 2. Response (R) rate in each treatment group 3. Time to response / complete response in each treatment group 4. Duration of response in each treatment group 5. Stable response in each treatment group 6. Proportion of participants with bleeding events overall and by WHO bleeding scale severity 7. Number and proportion of participants receiving rescue treatment 8. Cumulative dose/duration of steroids exposure 9. Change from baseline on total scores of the PROMIS SF v1.0 Fatigue 13a 10. Change from baseline in ITP-PAQ domain scores of Symptoms, Fatigue, Bother, Activity 11. Change from baseline in frequency and absolute number of CD19+ B cell counts 12. Time to first occurrence of B-cell recovery 13. Change from baseline in inmmunoglobulins 14. PK parameters: AUClast, AUCtau - PK parameters: Cmax - PK parameters: Tmax - PK parameters: Accumulation ratio Racc 15. Incidence and titer of anti-ianalumab antibodies in serum (ADA assay) over time ;Timepoint(s) of evaluation of this end point: 1-4, 6-7, 10-12 Randomization to end of study (up to 39 months after randomization of last patient) 5: At 6 months and 1 year 7-9: From screening (baseline) till end of study (up to 39 months after randomization of last patient) 13: After the first and last dose of study medication 14: Up to end of study (up to 39 months after randomization of last patient) | — |
Countries
Argentina, Australia, Austria, Belgium, Bulgaria, China, Czechia, Czech Republic, France, Germany, Hong Kong, Hungary, India, Italy, Japan, Malaysia, Mexico, Norway, Romania, Singapore, Spain, Thailand, Turkey, United Kingdom, United States, Viet Nam
Contacts
Public ContactPublic Information Desk
Novartis Hungary Kft.
Outcome results
None listed