Actively Recruiting
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Led by Apellis Pharmaceuticals, Inc. · Updated on 2025-06-11
12
Participants Needed
12
Research Sites
408 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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
You will not qualify if you...
- 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
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 12 locations
1
Children's Hospital of Atlanta
Atlanta, Georgia, United States, 30329
Actively Recruiting
2
Motol University Hospital
Prague, Czechia, 150 06
Completed
3
Robert-Debré Hospital Paris
Paris, France, 75009
Not Yet Recruiting
4
Hospital Ampang
Ampang, Malaysia, 68000
Actively Recruiting
5
Radboud University Hospital Nijmegen
Nijmegen, Netherlands, 6525 GA
Completed
6
University Medical Center Utrecht
Utrecht, Netherlands, 3508 GA
Actively Recruiting
7
University Children's Hospital
Belgrade, Serbia, 11000
Actively Recruiting
8
University Hospital Vall d'Hebron
Barcelona, Spain, E-08035
Actively Recruiting
9
University Hospital 12 de Octubre
Madrid, Spain, E-28041
Actively Recruiting
10
Phramongkutklao Hospital and College of Medicine
Bangkok, Thailand, 10400
Actively Recruiting
11
Maharaj Nakorn Chiang Mai hospital
Chiang Mai, Thailand, 50200
Actively Recruiting
12
St. Mary's Hospital
London, United Kingdom, W2 1NY
Actively Recruiting
Research Team
A
Apellis Clinical Trial Information Line
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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