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

Phase 3CompletedNCT06578949

Alexion Pharmaceuticals, Inc.18 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Paroxysmal Nocturnal Hemoglobinuria PNH

Interventions

RavulizumabDRUG

Ravulizumab will be administered by intravenous (IV) infusion.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (8)

Research Site

Beijing, China

Research Site

Guangzhou, China

Research Site

Hangzhou, China

Research Site

Nantong, China

Outcomes

Primary Outcomes

Percentage Change in Lactate Dehydrogenase (LDH) From Baseline to Day 183 (Week 26)

Time frame: Baseline, Day 183 (Week 26)

Secondary Outcomes

Number of Participants Achieving LDH <1.5 × Upper Limit of Normal (ULN) at Day 183 (Week 26)

Time frame: Day 183 (Week 26)

Number of Participants Achieving Transfusion Avoidance (TA) Through Day 183 (Week 26)

Time frame: Day 183 (Week 26)

Number of Participants Experiencing Breakthrough Hemolysis Through Day 183 (Week 26)

Time frame: 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: Day 183 (Week 26)

Change in Hemoglobin (Hgb) From Baseline to Day 183 (Week 26)

Time frame: Baseline, Day 183 (Week 26)

Data from ClinicalTrials.gov

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