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Clinical Trials in the UK / NCT04901936
Recruiting Phase 2

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

NCT04901936 · tracked via the Priya Life Science UK tracker
Sponsor
Apellis Pharmaceuticals, Inc.
Phase
Phase 2
Started
2021-02-04
Last updated
2026-06-26

Condition(s) studied

Paroxysmal Nocturnal Hemoglobinuria (PNH)Paroxysmal Hemoglobinuria

Investigational drug(s) / intervention(s)

Pegcetacoplan

Pegcetacoplan: Complement (C3) inhibitor

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).

Eligibility

Sex
ALL
Min age
12 Years
Max age
17 Years
Healthy volunteers
No
Inclusion Criteria: * 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 Criteria: * 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

Primary outcome measure(s)

Trial sites (12)

FacilityCityRegionStatus
Children's Hospital of Atlanta Atlanta Georgia Recruiting
Motol University Hospital Prague Czechia Completed
Robert-Debré Hospital Paris Paris France Not Yet Recruiting
Hospital Ampang Ampang Malaysia Recruiting
Radboud University Hospital Nijmegen Nijmegen Netherlands Completed
University Medical Center Utrecht Utrecht Netherlands Recruiting
University Children's Hospital Belgrade Serbia Recruiting
University Hospital Vall d'Hebron Barcelona Spain Recruiting
University Hospital 12 de Octubre Madrid Spain Recruiting
Phramongkutklao Hospital and College of Medicine Bangkok Thailand Completed
Maharaj Nakorn Chiang Mai hospital Chiang Mai Thailand Recruiting
St. Mary's Hospital London United Kingdom Completed
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04901936 on ClinicalTrials.gov ↗ ← All trials in the UK