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

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

Inclusion Criteria7

Are 12-17 years old at the time of screening
Weigh at least 20 kg (approx. 44 lbs)
Have the diagnosis of PNH, confirmed by high-sensitivity flow cytometry (granulocyte or monocyte clone >10%)
EITHER:
Not being treated with an approved complement inhibitor (eculizumab or ravulizumab) prior to start of pegcetacoplan dosing, AND have hemolytic anemia. Hemolytic anemia is defined as hemoglobin (Hb) less than the lower limit of normal (Hb < LLN) and LDH >1.5 times the upper limit of normal (ULN); OR
Currently receiving treatment with an approved complement inhibitor (eculizumab or ravulizumab) AND have evidence of ongoing anemia. Ongoing anemia is defined as Hb < LLN and ARC > ULN
Have a platelet count >75,000/mm3 and an absolute neutrophil count >1000/mm3

Exclusion Criteria4

Are an adult, 18 years of age or older, with PNH
Known or suspected hereditary fructose intolerance (HFI)
History of hereditary complement deficiency, bone marrow transplant, or meningococcal disease (meningitis, bacteremia or septicemia)
Females who are pregnant or breastfeeding

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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