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NCT Number: NCT04901936

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

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).

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Key information

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH) Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Myelodysplastic Syndromes Paroxysmal Hemoglobinuria

Age range

12 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Motol University Hospital, Prague, Czechia

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Pegcetacoplan

Drug

Complement (C3) inhibitor

Primary outcomes

  1. Pegcetacoplan serum concentrations over the course of the 16-week treatment period

    Time frame: 16 weeks

  2. Change from baseline to Wk 16 in hemoglobin (Hb)

    Time frame: 16 weeks

  3. Incidence and severity of treatment-emergent adverse events (TEAEs) over the course of the 16-week treatment period, including monitoring bacterial infections

    Time frame: 16 weeks

  4. Change from baseline to wk 16 lactate dehydrogenase (LDH)

    Time frame: 16 weeks

  5. Change from baseline to wk 16 absolute reticulocyte count (ARC)

    Time frame: 16 weeks

Secondary outcomes

  1. Change from baseline from week 16 to week 52 of C3 deposition on RBC cells

    Time frame: Week 16-52

  2. Incidence of thromboembolic events (major adverse vascular events [MAVE]) over the course of the 16-week treatment period and over 52 weeks of treatment with pegcetacoplan

    Time frame: 52 weeks

  3. Occurrence of breakthrough hemolysis over 16 and 52 weeks of treatment with pegcetacoplan

    Time frame: Week 16-52

  4. Change from baseline to Week 52, and from Week 16 to Week 52 , in Hb

    Time frame: Week 16-52

  5. Change from baseline to Week 16 and to Week 52 in Health-Related Quality of Life (HRQOL) assessments

    Time frame: Week 16-52

  6. Number of packed red blood cell (PRBC) units

    Time frame: Week 16-52

  7. Total units (mL/kg) transfused over 16 and 52 weeks of treatment with pegcetacoplan

    Time frame: Week 16-52

  8. Change from baseline to Week 52, and from Week 16 to Week 52, in LDH

    Time frame: Week 16-52

  9. Change from baseline to Week 52, and from Week 16 to Week 52 ARC

    Time frame: Week 16-52

Study contacts

Contact information is provided by the study sponsor or research team.

Apellis Clinical Trial Information Line

CONTACT

[email protected]

1-833-284-6361 (833-CT Info-1)

Sponsors and collaborators

Lead sponsor

Apellis Pharmaceuticals, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2028
Study completion
2028
First posted
May 26, 2021
Registry last updated
Jun 26, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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