Evaluating Anti-CD38 Antibody Daratumumab Treatment for Children Aged 6 to 18 With Primary Immune Thrombocytopenia

What this Trial Is About

This trial studies children aged 6 to 18 with primary immune thrombocytopenia ITP who did not respond well to first-line treatments like steroids. ITP is an autoimmune disease causing low platelet counts and bleeding, with limited effective treatments especially for recurrent or resistant cases. The study focuses on evaluating the safety and effectiveness of an anti-CD38 antibody called daratumumab, which targets plasma cells believed to contribute to treatment resistance in ITP. Participants receive intravenous daratumumab at a dose of 16 mgkg once a week for 4 weeks during the core treatment phase. After this, there is a follow-up period lasting until week 24 to monitor ongoing safety and treatment effects. This is a single-arm, open-label study enrolling 30 children who will be observed for responses and side effects after the antibody treatment. During the study, researchers will measure platelet counts and response rates, track bleeding events using pediatric and WHO bleeding scales, and monitor adverse effects up to 24 weeks. They will also evaluate reduction in other medications and time to response. Safety assessments and ongoing efficacy evaluations will occur throughout the follow-up period, with the primary outcome measured at 8 weeks.

Anti-CD38 Antibody Treating Children With Primary Immune Thrombocytopenia (ITP)