Pegcetacoplan treatment for adolescents (12–17) with 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
This trial will try pegcetacoplan given by twice-weekly home subcutaneous infusion in 12–17 year-olds with PNH to see if it is safe and helps their anemia and blood counts.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 12 Years to 17 Years |
| Sex | All |
| Sponsor | Apellis Pharmaceuticals, Inc. Industry-sponsored |
| Drugs / interventions | eculizumab, ravulizumab |
| Locations | 12 sites (Atlanta, Georgia and 11 other locations) |
| Trial ID | NCT04901936 on ClinicalTrials.gov |
What this trial studies
This open-label Phase 2 trial enrolls adolescents aged 12–17 with confirmed PNH and includes a 4-week screening period followed by a 16-week treatment period, with a 4-week run-in for those switching from a C5 inhibitor. All participants receive pegcetacoplan administered by subcutaneous infusion twice weekly at home after training for self- or caregiver-administration. The study measures safety, biological activity (how the drug is processed), and effectiveness on hemolysis and blood counts, with participants either entering a long-term extension or a 2-month follow-up after treatment. Regular clinic visits at participating sites are required for monitoring and laboratory testing.
Who should consider this trial
Good fit: Ideal candidates are 12–17 years old, weigh at least 20 kg, have PNH confirmed by high-sensitivity flow cytometry (granulocyte or monocyte clone >10%), and either have untreated hemolytic anemia (Hb < LLN and LDH >1.5×ULN) or are on eculizumab/ravulizumab with ongoing anemia (Hb < LLN and ARC > ULN), with platelets >75,000/mm3 and ANC >1000/mm3.
Not a fit: Patients unlikely to benefit include adults (≥18 years), those with known hereditary fructose intolerance or hereditary complement deficiency, those who do not meet the hemolysis or blood-count criteria, or those with low platelet or neutrophil counts below entry thresholds.
Why it matters
Potential benefit: If successful, pegcetacoplan could reduce hemolysis and raise hemoglobin levels in adolescents with PNH, potentially decreasing transfusion needs and improving symptoms.
How similar studies have performed: Similar pegcetacoplan studies in adults have shown improved hemoglobin and reduced transfusion rates compared with C5 inhibitors, but pediatric data are limited and this population remains less studied.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Atlanta, Georgia and 11 other locations
- Children's Hospital of Atlanta — Atlanta, Georgia, United States (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 (Recruiting)
- Maharaj Nakorn Chiang Mai hospital — Chiang Mai, Thailand (Recruiting)
- St. Mary's Hospital — London, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Apellis Clinical Trial Information Line
- Email: clinicaltrials@apellis.com
- Phone: 1-833-284-6361 (833-CT Info-1)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.