Phase 2 Study of Pegcetacoplan Treatment in Children and Teens Aged 12 to 17 With Paroxysmal Nocturnal Hemoglobinuria

What this Trial Is About

Researchers are evaluating the safety, effectiveness, and biological activity of pegcetacoplan in adolescents aged 12 to 17 years who have paroxysmal nocturnal hemoglobinuria PNH, a rare blood disorder. This open-label phase 2 study aims to understand how this investigational medication is processed by the body and its impact on PNH symptoms in this pediatric population. Participants will receive pegcetacoplan, a complement C3 inhibitor, administered as a subcutaneous infusion twice a week at home using two small needles inserted under the skin. The study includes a 4-week screening period, followed by a 16-week treatment period. Those switching from a C5 inhibitor treatment will have an additional 4-week run-in period before treatment. After the treatment period, participants may enter a long-term extension phase or a 2-month follow-up period. During the study, participants andor their caregivers will be trained to administer the medication at home. Researchers will monitor blood levels of pegcetacoplan, hemoglobin, lactate dehydrogenase, and reticulocyte counts, as well as track any treatment-emergent adverse events including infections and thromboembolic events. Quality of life and blood transfusion needs will also be assessed throughout treatment and follow-up. Overall participation may last up to 52 weeks, including the long-term extension.

A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)