Ireland
--:--IST
Latest
Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide
Clinical Trials in France / NCT04901936
Recruiting Phase 2

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

NCT04901936 · tracked via the Priya Life Science France 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)

  • Pegcetacoplan serum concentrations over the course of the 16-week treatment period — 16 weeks
  • Change from baseline to Wk 16 in hemoglobin (Hb) — 16 weeks
  • Incidence and severity of treatment-emergent adverse events (TEAEs) over the course of the 16-week treatment period, including monitoring bacterial infections — 16 weeks
  • Change from baseline to wk 16 lactate dehydrogenase (LDH) — 16 weeks
  • Change from baseline to wk 16 absolute reticulocyte count (ARC) — 16 weeks

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

More Apellis Pharmaceuticals, Inc. trials in France

An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Participants With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis Active, not recruiting

Other trials for the same condition

Study of Ultomiris® (Ravulizumab) Safety in Pregnancy Recruiting
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 France