Recruiting
Phase 2

Pegcetacoplan

Sponsor:

Apellis Pharmaceuticals, Inc.

Code:

NCT04901936

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal Hemoglobinuria

Eligibility Criteria

Sex: All

Age: 12 - 17

Healthy Volunteers: Not accepted

Interventions

Pegcetacoplan

Study Details

Brief summary:

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).

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal Hemoglobinuria

Study ID

NCT04901936

Start date

Feb 4, 2021

Status verified date

Jun, 2026

Completion date

Dec, 2028

Anticipated

Primary completion date

Dec, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12 - 17

Healthy Volunteers: Not accepted

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 Design

Enrollment

12 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Pegcetacoplan

Interventions

Pegcetacoplan

Complement (C3) inhibitor

Primary outcome measure

  • Pegcetacoplan serum concentrations over the course of the 16-week treatment period [ Time Frame: 16 weeks ]
  • Change from baseline to Wk 16 in hemoglobin (Hb) [ Time Frame: 16 weeks ]
  • 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 ]
  • Change from baseline to wk 16 lactate dehydrogenase (LDH) [ Time Frame: 16 weeks ]
  • Change from baseline to wk 16 absolute reticulocyte count (ARC) [ Time Frame: 16 weeks ]

Central Contacts and Locations

Central contacts

Apellis Clinical Trial Information Line

1-833-284-6361 (833-CT Info-1)clinicaltrials@apellis.com

Locations

Children's Hospital of Atlanta

Recruiting

Atlanta, Georgia, United States, 30329

More Information

Sponsor

Apellis Pharmaceuticals, Inc.

Last update posted

Jun 26, 2026

Last verified

Jun, 2026

Keywords

  • Paroxysmal Nocturnal Hemoglobinuria
  • PNH
  • Pediatric
  • Adolescent
  • Apellis
  • Anemia, Hemolytic
  • Pegcetacoplan
  • APL-2

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Apellis Pharmaceuticals, Inc. on 2026-06-26.