A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Recruiting now · Phase 2
Conditions studied: Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria
In brief
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).
Key facts
- Study ID
- NCT04901936
- Run by
- Apellis Pharmaceuticals, Inc.
- People needed
- 12
- Starts
- 2021-02-04
- Expected to finish
- 2028-12-01
- Last updated by the study team
- 2026-06-26
Who can join
Age: 12 and older, up to 17. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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 may not qualify if…
- 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
Where it is running
- Children's Hospital of Atlanta — Atlanta, Georgia, United States (enrolling)
- University Medical Center Utrecht — Utrecht, Netherlands (enrolling)
- University Children's Hospital — Belgrade, Serbia (enrolling)
- University Hospital Vall d'Hebron — Barcelona, Spain (enrolling)
- University Hospital 12 de Octubre — Madrid, Spain (enrolling)
- Maharaj Nakorn Chiang Mai hospital — Chiang Mai, Thailand (enrolling)
- Hospital Ampang — Ampang, Malaysia (enrolling)
- Motol University Hospital — Prague, Czechia
- Robert-Debré Hospital Paris — Paris, France
- St. Mary's Hospital — London, United Kingdom
- Radboud University Hospital Nijmegen — Nijmegen, Netherlands
- Phramongkutklao Hospital and College of Medicine — Bangkok, Thailand
Full record on ClinicalTrials.gov
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