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

NCT ID: NCT05886244

Last Updated: 2025-04-16

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-07-05

Study Completion Date

2025-04-14

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Paroxysmal Nocturnal Hemoglobinuria

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

open label
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Eculizumab

Eculizumab will be administered by IV infusion.

Group Type EXPERIMENTAL

Eculizumab

Intervention Type DRUG

Participants will receive 600 milligrams (mg) once a week on Day 1, 8, 15, and 22 followed by 900 mg every 2 weeks from Day 29 to Day 435.

Interventions

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Eculizumab

Participants will receive 600 milligrams (mg) once a week on Day 1, 8, 15, and 22 followed by 900 mg every 2 weeks from Day 29 to Day 435.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Adult C5 inhibitor naïve PNH patients (age\>=18), which is confirmed by flow cytometry evaluation
* Must be vaccinated against N meningitidis

Exclusion Criteria

* Meningitidis infection or unresolved meningococcal disease
* Significant bone marrow failure
* Other significant systemic diseases that might have impact on efficacy and safety assessment
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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AstraZeneca

INDUSTRY

Sponsor Role collaborator

Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Research Site

Beijing, , China

Site Status

Research Site

Hangzhou, , China

Site Status

Research Site

Nantong, , China

Site Status

Research Site

Shanghai, , China

Site Status

Research Site

Tianjin, , China

Site Status

Research Site

Tianjin, , China

Site Status

Research Site

Wuhan, , China

Site Status

Countries

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China

Other Identifiers

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D7414C00001

Identifier Type: -

Identifier Source: org_study_id

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