Study Description
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. 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.
Interventions
Ianalumab
Eligibility Criteria
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.
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