NCT04901936

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

An Open Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Apellis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 12 Years – 17 Years
• Healthy volunteers: Not accepted

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).

Detailed description

This is an open-label study to evaluate pegcetacoplan in people with PNH who are 12-17 years old. The study will consist of a 4-week screening period followed by a 16-week treatment period. Participants switching from a C5 inhibitor will have an additional 4 week run-in period between the screening and treatment periods. At the completion of the study treatment period, participants will either enter a long-term extension period or a 2-month follow-up period. All eligible study participants will receive pegcetacoplan, administered via subcutaneous infusion twice a week at home. The subcutaneous infusion requires two small needles to be inserted into the fatty layer of tissue under the skin and the investigational medication will flow into the body. Study participants and/or caregivers will be trained on home administration of pegcetacoplan.

Conditions

• Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Paroxysmal Hemoglobinuria

Interventions

Type	Name	Description
DRUG	Pegcetacoplan	Complement (C3) inhibitor

Timeline

• Start date: 2021-02-04
• Primary completion: 2028-12-01
• Completion: 2028-12-01
• First posted: 2021-05-26
• Last updated: 2025-06-11

Locations

12 sites across 9 countries: United States, Czechia, France, Malaysia, Netherlands, Serbia, Spain, Thailand, United Kingdom

Regulatory

• FDA-regulated drug study