ClinicalTrials.gov record NCT04901936

A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age12 years old and older
SexAll
LocationCzechia, France, Malaysia, Netherlands, Serbia, Spain, Thailand, United Kingdom, United States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Open-label, single-arm Phase 2 study of pegcetacoplan in adolescents aged 12–17 with PNH. Participants receive twice-weekly subcutaneous pegcetacoplan at home after a 4‑week screening period; those switching from a C5 inhibitor have a 4‑week run-in. The main treatment period is 16 weeks, after which participants may enter a long-term extension or a 2‑month follow-up. The study measures drug levels, blood tests, and treatment-emergent adverse events.

What it is studying. The study is testing pegcetacoplan in 12–17 year-olds with PNH to evaluate safety, how the drug is processed in the body (serum concentrations), and its biological effects on blood measures such as hemoglobin, lactate dehydrogenase (LDH), and absolute reticulocyte count (ARC). It also monitors treatment-emergent adverse events, including bacterial infections.

What the researchers hope to learn. To evaluate the safety profile and pharmacokinetics of pegcetacoplan in adolescents with PNH and to measure its biological activity on blood markers (hemoglobin, LDH, ARC, C3 deposition), incidence of infections and thromboembolic events, breakthrough hemolysis, transfusion use, and quality-of-life measures over 16 and 52 weeks.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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).

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Pegcetacoplan

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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. Participants switching from a C5 inhibitor will have an additional 4 week run-in period between the screening and treatment periods. At the completion of the study treatment period, participants will either enter a long-term extension period or a 2-month follow-up period. All eligible study participants will receive pegcetacoplan, administered via subcutaneous infusion twice a week at home. The subcutaneous infusion requires two small needles to be inserted into the fatty layer of tissue under the skin and the investigational medication will flow into the body. Study participants and/or caregivers will be trained on home administration of pegcetacoplan.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

After a 4‑week screening period, participants receive pegcetacoplan by subcutaneous infusion twice weekly at home (two small needles into the fatty layer under the skin). Those switching from a C5 inhibitor have an extra 4‑week run‑in before the 16‑week treatment period. Study staff will train participants and/or caregivers to give the home doses and will perform regular visits and blood tests to measure drug levels, hemoglobin, LDH, reticulocyte count and to monitor safety (including infections); at the end participants enter a long‑term extension or a 2‑month follow‑up period.

How the study is structured. Interventional, open-label, single-group Phase 2 study. All participants receive pegcetacoplan by subcutaneous infusion twice weekly at home. The schedule includes a 4‑week screening period, a 16‑week treatment period, and either a long-term extension or a 2‑month follow-up; there is an additional 4‑week run-in for participants switching from an approved C5 inhibitor. The study collects blood tests and safety monitoring throughout treatment.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in Czechia, France, Malaysia, Netherlands, Serbia, Spain, Thailand, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on paroxysmal nocturnal hemoglobinuria

Clinical trials44 trials listed for paroxysmal nocturnal hemoglobinuriaEvery trial the registry lists for paroxysmal nocturnal hemoglobinuria, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in paroxysmal nocturnal hemoglobinuria researchLatest monthly update: September 2026. Includes: Treatment access. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Feb 4, 2021
Primary completion
December 2028 (estimated)
Study completion
December 2028 (estimated)
Target enrollment
12 participants (estimated)
Last updated on registry
Jun 26, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Apellis Pharmaceuticals, Inc.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (12)

  • Children's Hospital of AtlantaRecruiting

    Atlanta, Georgia, 30329 · United States

  • Motol University HospitalCompleted

    Prague, 150 06 · Czechia

  • Robert-Debré Hospital ParisNot Yet Recruiting

    Paris, 75009 · France

  • Hospital AmpangRecruiting

    Ampang, 68000 · Malaysia

  • Radboud University Hospital NijmegenCompleted

    Nijmegen, 6525 GA · Netherlands

  • University Medical Center UtrechtRecruiting

    Utrecht, 3508 GA · Netherlands

  • University Children's HospitalRecruiting

    Belgrade, 11000 · Serbia

  • University Hospital Vall d'HebronRecruiting

    Barcelona, E-08035 · Spain

  • University Hospital 12 de OctubreRecruiting

    Madrid, E-28041 · Spain

  • Phramongkutklao Hospital and College of MedicineCompleted

    Bangkok, 10400 · Thailand

Terms used on this page · explanations by MedicalUpdates.ai

investigational
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
absolute neutrophil count
The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
bone marrow transplant
A procedure that replaces blood-forming cells after high-dose treatment. 'Autologous' uses the person's own cells collected beforehand; 'allogeneic' uses cells from a donor.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
screening period
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
run-in period
A period at the start of a study, before the study treatment begins, used for example to confirm that participants meet the criteria or to record their usual symptoms.
extension period
A continuation study in which participants who completed an earlier study can keep receiving the treatment, usually with everyone knowing what they are given.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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