Status:
RECRUITING
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Lead Sponsor:
Apellis Pharmaceuticals, Inc.
Conditions:
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal Hemoglobinuria
Eligibility:
All Genders
12-17 years
Phase:
PHASE2
Brief Summary
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 (adolescen...
Detailed Description
This is an open-label study to evaluate pegcetacoplan in people with PNH who are 12-17 years old. The study will consist of a 4-week screening period followed by a 16-week treatment period. Participan...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
February 4 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 1 2028
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT04901936
Start Date
February 4 2021
End Date
December 1 2028
Last Update
June 11 2025
Active Locations (12)
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1
Children's Hospital of Atlanta
Atlanta, Georgia, United States, 30329
2
Motol University Hospital
Prague, Czechia, 150 06
3
Robert-Debré Hospital Paris
Paris, France, 75009
4
Hospital Ampang
Ampang, Malaysia, 68000