Actively Recruiting
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
Led by Apellis Pharmaceuticals, Inc. · Updated on 2025-06-11
12
Participants Needed
12
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating pegcetacoplan, a complement (C3) inhibitor, in adolescents aged 12 to 17 years with paroxysmal nocturnal hemoglobinuria (PNH). The study aims to assess the safety, effectiveness, and how the medication is processed by the body in this younger population. This open-label phase 2 trial is sponsored by Apellis Pharmaceuticals, Inc. and focuses on understanding pegcetacoplan's biological activity and impact on hemoglobin and other blood markers. The study includes a 4-week screening period, followed by a 16-week treatment period where all participants receive pegcetacoplan via subcutaneous infusion twice weekly at home. Those switching from a C5 inhibitor will have an additional 4-week run-in period before treatment. After the treatment period, participants may continue in a long-term extension or enter a 2-month follow-up phase. Participants and caregivers are trained on how to administer the medication at home using two small needles. During the study, participants will have regular assessments including blood tests to measure hemoglobin, lactate dehydrogenase, reticulocyte count, and pegcetacoplan blood levels. Researchers will monitor adverse events, infections, and other safety concerns throughout the 16-week treatment. Longer-term effects will be evaluated during extension or follow-up periods. The study also includes quality of life assessments and tracks the need for blood transfusions. Total participation time varies depending on extension or follow-up enrollment.
CONDITIONS
Brief 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 are not being treated with an approved complement inhibitor prior to pegcetacoplan dosing and have hemolytic anemia defined as hemoglobin less than the lower limit of normal and LDH greater than 1.5 times the upper limit of normal
- Or currently receiving treatment with an approved complement inhibitor and have evidence of ongoing anemia defined as hemoglobin less than the lower limit of normal and absolute reticulocyte count greater than the upper limit of normal
- Have a platelet count greater than 75,000/mm3 and an absolute neutrophil count greater than 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
Your Study Journey
Duration - 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 4 weeks
Participants switching from a C5 inhibitor undergo a run-in period before starting treatment.
Visits as needed during the run-in period
Duration - 16 weeks
Participants receive pegcetacoplan via subcutaneous infusion twice a week at home.
Twice-weekly home infusions with training provided; periodic study visits for monitoring
Duration - 2 months or longer
Participants either enter a long-term extension period or a 2-month follow-up period after treatment ends.
Visits depending on extension or follow-up assignment
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
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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