Study of Anti-CD38 Antibody Treatment for Children Ages 6 to 17 With Primary Immune Thrombocytopenia Evaluating Safety and Effectiveness in Patients Not Responding to Standard Therapies

What this Trial Is About

Researchers are evaluating the safety and effectiveness of an Anti-CD38 antibody treatment for children aged 6 to 17 years with primary immune thrombocytopenia ITP who have not responded well or have relapsed after initial and at least one second-line therapy, including Anti-CD20 antibody or TPO receptor agonists. ITP is an autoimmune disease causing low platelet counts and bleeding, with current treatments often insufficient for some patients, especially those with recurrent or resistant forms. This study aims to explore a new approach targeting long-lived plasma cells that may contribute to treatment resistance. Participants will receive intravenous Anti-CD38 antibody at a dose of 16 mgkg once weekly for 8 weeks during the main treatment phase. After this, there is a follow-up period from weeks 9 to 24 to monitor safety and ongoing effects, followed by an extension phase lasting up to 5 years to observe long-term safety and sustained efficacy. The study is designed as a single-arm, open-label trial involving 60 children. During the study, participants will have their platelet counts and immune responses regularly evaluated, along with monitoring for any bleeding events using standard scales. Researchers will measure various immune cell subsets and antibody levels over the course of up to 5 years. Safety assessments will continue throughout the entire study duration, which includes treatment, follow-up, and long-term observation to understand the antibodys impact on pediatric ITP.

Anti-CD38 Antibody Treating Pediatric Primary Immune Thrombocytopenia (ITP)