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

Phase 3

Active, not recruiting

• Conditions: Paroxysmal Nocturnal Hemoglobinuria; PNH
• Interventions: Drug: Ravulizumab

• Registration Number: NCT06578949
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-08-28
• Study First Submitted QC: 2024-08-28
• Study First Posted: 2024-08-30
• Study Start: 2024-10-10
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-05
• Last Update Posted: 2025-03-06
• Primary Completion: 2025-05-17
• Study Completion: 2026-01-14

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 18

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

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Ravulizumab	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.

Primary Outcome Measures

Name	Time	Method
Percentage Change in Lactate Dehydrogenase (LDH) From Baseline to Day 183 (Week 26)	Baseline, Day 183 (Week 26)

Secondary Outcome Measures

Name	Time	Method
Number of Participants Achieving LDH <1.5 × Upper Limit of Normal (ULN) at Day 183 (Week 26)	Day 183 (Week 26)
Number of Participants Achieving Transfusion Avoidance (TA) Through Day 183 (Week 26)	Day 183 (Week 26)
Number of Participants Experiencing Breakthrough Hemolysis Through Day 183 (Week 26)	Day 183 (Week 26)
Change in Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue) Score From Baseline to Day 183 (Week 26)	Day 183 (Week 26)
Change in Hemoglobin (Hgb) From Baseline to Day 183 (Week 26)	Baseline, Day 183 (Week 26)

Trial Locations

Locations (1)

Research Site; 🇨🇳 Wuhan, China