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

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).

Study Overview

• Status: Recruiting
• Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH); Paroxysmal Hemoglobinuria
• Intervention / Treatment: Drug: Pegcetacoplan

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Estimated): 12
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Apellis Clinical Trial Information Line; Phone Number: 1-833-284-6361 (833-CT Info-1); Email: clinicaltrials@apellis.com

Study Locations

• Czechia
  • Prague, Czechia, 150 06
    • Completed
    • Motol University Hospital
• France
  • Paris, France, 75009
    • Not yet recruiting
    • Robert-Debré Hospital Paris
• Malaysia
  • Ampang, Malaysia, 68000
    • Recruiting
    • Hospital Ampang
• Netherlands
  • Nijmegen, Netherlands, 6525 GA
    • Completed
    • Radboud University Hospital Nijmegen
  • Utrecht, Netherlands, 3508 GA
    • Recruiting
    • University Medical Center Utrecht
• Serbia
  • Belgrade, Serbia, 11000
    • Recruiting
    • University Children's Hospital
• Spain
  • Barcelona, Spain, E-08035
    • Recruiting
    • University Hospital Vall d'Hebron
  • Madrid, Spain, E-28041
    • Recruiting
    • University Hospital 12 de Octubre
• Thailand
  • Bangkok, Thailand, 10400
    • Recruiting
    • Phramongkutklao hospital and College of Medicine
  • Chiang Mai, Thailand, 50200
    • Recruiting
    • Maharaj Nakorn Chiang Mai Hospital
• United Kingdom
  • London, United Kingdom, W2 1NY
    • Recruiting
    • St. Mary's Hospital
• United States
  • Georgia
    • Atlanta, Georgia, United States, 30329
      • Recruiting
      • Children's Hospital of Atlanta

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Pegcetacoplan	Drug: Pegcetacoplan; Complement (C3) inhibitor

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Pegcetacoplan serum concentrations over the course of the 16-week treatment period	16 weeks
Change from baseline to Wk 16 in hemoglobin (Hb)	16 weeks
Incidence and severity of treatment-emergent adverse events (TEAEs) over the course of the 16-week treatment period, including monitoring bacterial infections	16 weeks
Change from baseline to wk 16 lactate dehydrogenase (LDH)	16 weeks
Change from baseline to wk 16 absolute reticulocyte count (ARC)	16 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Change from baseline from week 16 to week 52 of C3 deposition on RBC cells	Week 16-52
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	52 weeks
Occurrence of breakthrough hemolysis over 16 and 52 weeks of treatment with pegcetacoplan	Week 16-52
Change from baseline to Week 52, and from Week 16 to Week 52 , in Hb	Week 16-52
Change from baseline to Week 16 and to Week 52 in Health-Related Quality of Life (HRQOL) assessments	Week 16-52
Number of packed red blood cell (PRBC) units	Week 16-52
Total units (mL/kg) transfused over 16 and 52 weeks of treatment with pegcetacoplan	Week 16-52
Change from baseline to Week 52, and from Week 16 to Week 52, in LDH	Week 16-52
Change from baseline to Week 52, and from Week 16 to Week 52 ARC	Week 16-52

Collaborators and Investigators

• Sponsor: Apellis Pharmaceuticals, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): February 4, 2021
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2028

Study Registration Dates

• First Submitted: March 8, 2021
• First Submitted That Met QC Criteria: May 20, 2021
• First Posted (Actual): May 26, 2021

Study Record Updates

• Last Update Posted (Actual): June 11, 2025
• Last Update Submitted That Met QC Criteria: June 6, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Keywords: Adolescent; Pediatric; PNH; Paroxysmal Nocturnal Hemoglobinuria; APL-2; Pegcetacoplan; Anemia, Hemolytic; Apellis
• Additional Relevant MeSH Terms: Urogenital Diseases; Male Urogenital Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urination Disorders; Urological Manifestations; Hematologic Diseases; Bone Marrow Diseases; Anemia, Hemolytic; Anemia; Myelodysplastic Syndromes; Proteinuria; Hemoglobinuria; Hemoglobinuria, Paroxysmal
• Other Study ID Numbers: APL2-PNH-209

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No