Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

Last updated: April 15, 2025
Sponsor: Alexion Pharmaceuticals, Inc.
Overall Status: Completed

Phase

3

Condition

Hemoglobinuria, Paroxysmal

Red Blood Cell Disorders

Aplastic Anemia

Treatment

Eculizumab

Clinical Study ID

NCT05886244
D7414C00001
  • Ages > 18
  • All Genders

Study Summary

This is a Phase 3b, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of eculizumab in adult participants with paraxysmal noturnal hemoglobinuria (PNH) in China.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Adult C5 inhibitor naïve PNH patients (age>=18), which is confirmed by flowcytometry evaluation

  • Must be vaccinated against N meningitidis

Exclusion

Exclusion Criteria:

  • Meningitidis infection or unresolved meningococcal disease

  • Significant bone marrow failure

  • Other significant systemic diseases that might have impact on efficacy and safetyassessment

Study Design

Total Participants: 25
Treatment Group(s): 1
Primary Treatment: Eculizumab
Phase: 3
Study Start date:
July 05, 2023
Estimated Completion Date:
April 14, 2025

Study Description

This is a Phase 3b, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of eculizumab in adult participants with PNH in China who previously have not been treated with complement inhibitors. Approximately 25 eligible participants in China will be enrolled.

Connect with a study center

  • Research Site

    Beijing, CN-100730
    China

    Site Not Available

  • Research Site

    Hangzhou, 310003
    China

    Site Not Available

  • Research Site

    Nantong, 226001
    China

    Site Not Available

  • Research Site

    Shanghai, 200040
    China

    Site Not Available

  • Research Site

    Tianjian, 300020
    China

    Site Not Available

  • Research Site

    Tianjin, 300020
    China

    Site Not Available

  • Research Site

    Wuhan, 430022
    China

    Site Not Available

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