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: * Screening phase * Treatment phase * Follow-up phase consisting of Efficacy follow-up phase and Safety follow-up phase
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
36
Liquid in a vial Concentrate for solution for infusion
Film-coated tablet and also Powder for oral suspension
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.
Time frame: enrolled until end of study (up to 39 months from the last patient enrolled)
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.
Time frame: 6 months
Stable response rate at 12 months (SR-12)
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: from enrollment until end of study (up to 39 months from the last patient enrolled)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Time to complete response
Time to complete response is defined as the time from the start of treatment to the date of complete response.
Time frame: 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.
Time frame: 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.
Time frame: 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
Time frame: 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
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: over time (up to 39 months from the last patient enrolled)