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

May 11, 2026 updated by: Alexion Pharmaceuticals, Inc.

A Phase 3, Single-arm, Open-label, Multicenter Study to Assess the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Complement Inhibitor Treatment Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

18

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
      • Beijing, China, CN-100730
        • Research Site
      • Guangzhou, China, 510100
        • Research Site
      • Hangzhou, China, 310003
        • Research Site
      • Nantong, China, 226001
        • Research Site
      • Shanghai, China, 200040
        • Research Site
      • Tianjin, China, 300020
        • Research Site
      • Tianjin, China, 300050
        • Research Site
      • Wuhan, China, 430022
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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.
Drug: Ravulizumab
Ravulizumab will be administered by intravenous (IV) infusion.
Other Names:
  • ALXN1210

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage Change in Lactate Dehydrogenase (LDH) From Baseline to Day 183 (Week 26)
Time Frame: Baseline, Day 183 (Week 26)
LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicated reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first dose of study drug. The percent change in LDH was analyzed using a mixed-effect model for repeated measures (MMRM) with the fixed categorical effect of visit, fixed continuous effect of the LDH baseline value as covariates, and participant as random effect.
Baseline, Day 183 (Week 26)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of Participants Achieving LDH <1.5 * Upper Limit of Normal (ULN) at Day 183 (Week 26)
Time Frame: Day 183 (Week 26)
LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicated reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first dose of study drug.
Day 183 (Week 26)
Percentage of Participants Achieving Transfusion Avoidance Through Day 183 (Week 26)
Time Frame: Day 183 (Week 26)
Transfusion avoidance was defined as the percentage of participants who remained transfusion free and did not require a transfusion per protocol-specified guidelines (hemoglobin value of ≤9 grams (g)/deciliter (dL) with signs or symptoms of sufficient severity to warrant a transfusion, or a hemoglobin value of ≤7 g/dL regardless of presence of clinical signs or symptoms) through Day 183.
Day 183 (Week 26)
Percentage of Participants Experiencing Breakthrough Hemolysis Through Day 183 (Week 26)
Time Frame: Day 183 (Week 26)
Breakthrough hemolysis was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia [hemoglobin <10 g/dL], major adverse vascular event [including thrombosis], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 times the ULN.
Day 183 (Week 26)
Change in Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue) Score From Baseline to Day 183 (Week 26)
Time Frame: Baseline, Day 183 (Week 26)
The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with a higher score indicating better quality of life. Analysis was using a mixed-effect model for repeated measures (MMRM) with the fixed categorical effect of visit, fixed continuous effect of the baseline value of FACIT-Fatigue score as covariates, and participant as random effect.
Baseline, Day 183 (Week 26)
Change in Hemoglobin (Hgb) From Baseline to Day 183 (Week 26)
Time Frame: Baseline, Day 183 (Week 26)
Analysis was performed using MMRM with the fixed categorical effect of visit, fixed continuous effect of the Hgb baseline value as covariates, and participant as random effect.
Baseline, Day 183 (Week 26)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 10, 2024

Primary Completion (Actual)

May 15, 2025

Study Completion (Actual)

December 22, 2025

Study Registration Dates

First Submitted

August 28, 2024

First Submitted That Met QC Criteria

August 28, 2024

First Posted (Actual)

August 30, 2024

Study Record Updates

Last Update Posted (Actual)

June 5, 2026

Last Update Submitted That Met QC Criteria

May 11, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • D9289C00008
  • ALXN1210-PNH-323 (Other Identifier: Alexion)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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