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

NCT04901936 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2025-06-11

No results posted yet for this study

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

Interventions

DRUG

Pegcetacoplan

Complement (C3) inhibitor

Sponsors & Collaborators

  • Apellis Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-02-04
Primary Completion
2028-12-31
Completion
2028-12-31
FDA Drug
Yes

Countries

  • United States
  • Czechia
  • France
  • Malaysia
  • Netherlands
  • Serbia
  • Spain
  • Thailand
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04901936 on ClinicalTrials.gov