RecruitingPhase 2NCT04901936

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

Sponsor

Apellis Pharmaceuticals, Inc.

Enrollment

12 participants

Start Date

Feb 4, 2021

Study Type

INTERVENTIONAL

Conditions

Paroxysmal Nocturnal Hemoglobinuria, PNH Paroxysmal Hemoglobinuria

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).

Eligibility

Min Age: 12 YearsMax Age: 17 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Pegcetacoplan for people with paroxysmal hemoglobinuria and paroxysmal nocturnal hemoglobinuria, pnh. The study is currently recruiting participants at 12 locations. People eligible for this study include aged 12 Years to 17 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGPegcetacoplan

Complement (C3) inhibitor

Locations(12)

Children's Hospital of Atlanta

Atlanta, Georgia, United States

Motol University Hospital

Prague, Czechia

Robert-Debré Hospital Paris

Paris, France

Hospital Ampang

Ampang, Malaysia

Radboud University Hospital Nijmegen

Nijmegen, Netherlands

University Medical Center Utrecht

Utrecht, Netherlands

University Children's Hospital

Belgrade, Serbia

University Hospital Vall d'Hebron

Barcelona, Spain

University Hospital 12 de Octubre

Madrid, Spain

Phramongkutklao Hospital and College of Medicine

Bangkok, Thailand

Maharaj Nakorn Chiang Mai hospital

Chiang Mai, Thailand

St. Mary's Hospital

London, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT04901936

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