NCT04901936RecruitingPhase 2
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (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
Source: ClinicalTrials.gov · Last updated 26 Jun 2026
Brief summary (registry, in English)
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).
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 12 (estimated)
- First submitted
- 8 Mar 2021
- Start date
- 4 Feb 2021
- Primary completion date
- Dec 2028
- Lead sponsor
- Apellis Pharmaceuticals, Inc.
- NCT number
- NCT04901936
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 12
- From 4 Feb 2021 to the primary completion date, Dec 2028
- 12 sites in 9 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- History of hereditary complement deficiency, bone marrow transplant, or meningococcal disease (meningitis, bacteremia or septicemia)
Age
- Are an adult, 18 years of age or older, with PNH
Other
- Known or suspected hereditary fructose intolerance (HFI)
- Females who are pregnant or breastfeeding
Who the study is looking for
- 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
Age in the registry: 12 years to 17 years · Sex: All · Does not accept healthy volunteers
Sites (12)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Motol University Hospital Coordinator of this site? Confirm its status | Prague | Czechia | Completed |
| University Hospital Vall d'Hebron Coordinator of this site? Confirm its status | Barcelona | Spain | Recruiting |
| University Hospital 12 de Octubre Coordinator of this site? Confirm its status | Madrid | Spain | Recruiting |
| Robert-Debré Hospital Paris Coordinator of this site? Confirm its status | Paris | France | Not yet recruiting |
| St Mary's Hospital Coordinator of this site? Confirm its status | London | United Kingdom | Completed |
| Hospital Ampang Coordinator of this site? Confirm its status | Ampang | Malaysia | Recruiting |
| Radboud University Hospital Nijmegen Coordinator of this site? Confirm its status | Nijmegen | Netherlands | Completed |
| University Medical Center Utrecht Coordinator of this site? Confirm its status | Utrecht | Netherlands | Recruiting |
| University Children's Hospital Coordinator of this site? Confirm its status | Belgrade | Serbia | Recruiting |
| Phramongkutklao Hospital and College of Medicine Coordinator of this site? Confirm its status | Bangkok | Thailand | Completed |
| Maharaj Nakorn Chiang Mai Hospital Coordinator of this site? Confirm its status | Chiang Mai | Thailand | Recruiting |
| Children's Hospital of Atlanta Coordinator of this site? Confirm its status | Atlanta | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.