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
Sex
ALL
Min age
12 Years
Max age
17 Years
Healthy volunteers
No
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
Primary outcome measure(s)
Pegcetacoplan serum concentrations over the course of the 16-week treatment period — 16 weeks
Change from baseline to Wk 16 in hemoglobin (Hb) — 16 weeks
Incidence and severity of treatment-emergent adverse events (TEAEs) over the course of the 16-week treatment period, including monitoring bacterial infections — 16 weeks
Change from baseline to wk 16 lactate dehydrogenase (LDH) — 16 weeks
Change from baseline to wk 16 absolute reticulocyte count (ARC) — 16 weeks
Trial sites (12)
Facility
City
Region
Status
Children's Hospital of Atlanta
Atlanta
Georgia
Recruiting
Motol University Hospital
Prague
Czechia
Completed
Robert-Debré Hospital Paris
Paris
France
Not Yet Recruiting
Hospital Ampang
Ampang
Malaysia
Recruiting
Radboud University Hospital Nijmegen
Nijmegen
Netherlands
Completed
University Medical Center Utrecht
Utrecht
Netherlands
Recruiting
University Children's Hospital
Belgrade
Serbia
Recruiting
University Hospital Vall d'Hebron
Barcelona
Spain
Recruiting
University Hospital 12 de Octubre
Madrid
Spain
Recruiting
Phramongkutklao Hospital and College of Medicine
Bangkok
Thailand
Completed
Maharaj Nakorn Chiang Mai hospital
Chiang Mai
Thailand
Recruiting
St. Mary's Hospital
London
United Kingdom
Completed
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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