A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age12-17
SponsorApellis Pharmaceuticals, Inc.
About this trial
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 criteria
Qualifiers
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%)
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
Disqualifiers
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
Trial design
Treatments tested in this trial
- Pegcetacoplan
Treatment groups
12 Participants
are divided into 1 treatment groupLocations
12Czechia
Motol University Hospital150 06, Prague Czechia
France
Robert-Debré Hospital Paris75009, Paris France
Malaysia
Hospital Ampang68000, Ampang Malaysia
Netherlands
Radboud University Hospital Nijmegen6525 GA, Nijmegen Netherlands
Sponsors and collaborators
Apellis Pharmaceuticals, Inc.
Lead sponsor
Source ClinicalTrials.gov