Overview
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.
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.
Eligibility
Inclusion Criteria:
- 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.
Key exclusion criteria:
- 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.
- 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.