A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Apellis Pharmaceuticals, Inc. · NCT04901936
Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.
Key facts
- Compensation
- Not posted. Ask the study team before screening.
- Who can join
- Ages 12–17 · All sexes
- Healthy volunteers
- Not accepted (a condition is required)
- Study type
- Interventional · Phase 2
- Target enrollment
- 12
- US sites recruiting
- 1
- Study start
- February 4, 2021
- Record last updated
- June 26, 2026
Status re-checked against ClinicalTrials.gov within the last 12 hours.
What the study is about
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).
Being studied: Pegcetacoplan
Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH); Paroxysmal Hemoglobinuria
Eligibility criteria
Copied from the official record. The study team makes the final decision at screening.
Show full 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
Recruiting US locations
Children's Hospital of Atlanta
Atlanta, GA 30329
Study contacts (from the official record)
- Apellis Clinical Trial Information Line · 1-833-284-6361 (833-CT Info-1) · clinicaltrials@apellis.com