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

Study 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).

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Hemoglobinuria

Interventions / Drugs

Pegcetacoplan

View on ClinicalTrials.gov ↗

Sponsor

APLS

Apellis Pharmaceuticals

Complement Inhibitors

D36/100

Study Details

Enrollment12 participants

Primary Completion—

Lead SponsorApellis Pharmaceuticals, Inc.

Data Retrieved2026-04-11 02:54:08.046095+00:00

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