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

NCT04901936 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: APL2-PNH-209
• Study Type: interventional
• Target: 12
• Locations: 9 countries
• Sites: 12

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

Keywords

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

Outcome Measures

Primary Outcomes (5)

• 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 Outcomes (9)

• 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

Study Arms (1)

Pegcetacoplan

EXPERIMENTAL

Drug: Pegcetacoplan

Interventions

Pegcetacoplan DRUG

Complement (C3) inhibitor

Pegcetacoplan

Eligibility Criteria

• Age: 12 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Apellis Pharmaceuticals, Inc.: lead

Study Sites (12)

Children's Hospital of Atlanta

Atlanta, Georgia, 30329, United States

RECRUITING

Motol University Hospital

Prague, 150 06, Czechia

COMPLETED

Robert-Debré Hospital Paris

Paris, 75009, France

NOT YET RECRUITING

Hospital Ampang

Ampang, 68000, Malaysia

RECRUITING

Radboud University Hospital Nijmegen

Nijmegen, 6525 GA, Netherlands

COMPLETED

University Medical Center Utrecht

Utrecht, 3508 GA, Netherlands

RECRUITING

University Children's Hospital

Belgrade, 11000, Serbia

RECRUITING

University Hospital Vall d'Hebron

Barcelona, E-08035, Spain

RECRUITING

University Hospital 12 de Octubre

Madrid, E-28041, Spain

RECRUITING

Phramongkutklao Hospital and College of Medicine

Bangkok, 10400, Thailand

RECRUITING

Maharaj Nakorn Chiang Mai hospital

Chiang Mai, 50200, Thailand

RECRUITING

St. Mary's Hospital

London, W2 1NY, United Kingdom

RECRUITING

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal; Anemia, Hemolytic

Interventions

pegcetacoplan

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Central Study Contacts

Apellis Clinical Trial Information Line

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 8, 2021
• First Posted: May 26, 2021
• Study Start: February 4, 2021
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: June 11, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1); GB (1); ES (2); NL (2); US (1); SE (1); MY (1); TH (2); CZ (1)