Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

NCT ID: NCT06578949

Last Updated: 2026-01-21

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

18 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-10-10

Study Completion Date

2025-12-22

Brief Summary

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The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with PNH.

Detailed Description

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Conditions

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Paroxysmal Nocturnal Hemoglobinuria PNH

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Ravulizumab

During the Primary Treatment Period, participants will receive a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks. On Day 183, all participants will enter a 32-week Extension Treatment Period and receive ravulizumab. Beginning on Day 183, participants will receive a maintenance dose of ravulizumab q8w for an additional 32 weeks.

Group Type EXPERIMENTAL

Ravulizumab

Intervention Type DRUG

Ravulizumab will be administered by intravenous (IV) infusion.

Interventions

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Ravulizumab

Ravulizumab will be administered by intravenous (IV) infusion.

Intervention Type DRUG

Other Intervention Names

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ALXN1210

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

* Meningitidis infection or unresolved meningococcal disease
* History of bone marrow transplantation
* 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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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

Guangzhou, , 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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ALXN1210-PNH-323

Identifier Type: OTHER

Identifier Source: secondary_id

D9289C00008

Identifier Type: -

Identifier Source: org_study_id

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