Home › Paroxysmal Hemoglobinuria › NCT04901936
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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
Who can join
Ages 12 to 17 · All sexes
Full eligibility criteria
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
About the study
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).
What is being tested
- Pegcetacoplan (drug)
Sponsor: Apellis Pharmaceuticals, Inc. · Participants: 12 · Started: Feb 4, 2021
Contact the study team
- Apellis Clinical Trial Information Line · Phone: 1-833-284-6361 (833-CT Info-1)
Official record on ClinicalTrials.gov — NCT04901936
Locations in the U.S.
| Georgia | Children's Hospital of Atlanta, Atlanta |
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.