Clinical Trial

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

Recruiting Phase 2
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Summary
The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal nocturnal hemoglobinuria (PNH).
Protocol Amendment History 3 amendments
This ClinicalTrials.gov record has been amended 3 times since 2021-05-20; most recent amendment 2026-06-23.
Trial Details
NCT Number NCT04901936
Lead Sponsor Apellis Pharmaceuticals, Inc.
Conditions Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria
Enrollment 12 participants
Start Date 2021-02-04
Primary Completion 2028-12 (estimated)
Study Completion 2028-12 (estimated)
Updated on ClinicalTrials.gov 2026-06-26