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

Phase 3

Completed

• Conditions: Paroxysmal Nocturnal Hemoglobinuria
• Interventions: Drug: Eculizumab

• Registration Number: NCT05886244
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief Summary

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

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.

Study Timeline

• Study First Submitted: 2023-05-24
• Study First Submitted QC: 2023-05-24
• Study First Posted: 2023-06-02
• Study Start: 2023-07-05
• Status Verified: 2025-04
• Primary Completion: 2025-04-14
• Study Completion: 2025-04-14
• Last Update Submitted: 2025-04-15
• Last Update Posted: 2025-04-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 25

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Eculizumab	Eculizumab	Eculizumab will be administered by IV infusion.

Primary Outcome Measures

Name	Time	Method
Percentage change from baseline in Lactate Dehydrogenase (LDH) at Week 12	Baseline, Week 12	To assess efficacy of eculizumab in participants with PNH

Secondary Outcome Measures

Name	Time	Method
Number(%) of participants achieving LDH Normalization	Baseline through Week 64	To assess the efficacy of eculizumab in participants with PNH
Number(%) of participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)	Baseline through Week 64	To assess the safety and tolerability of eculizumab in participants with PNH
Number(%) of participants needing Blood Transfusion	Baseline through Week 64	To assess the efficacy of eculizumab in participants with PNH
Change from baseline in functional assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 64	Baseline through Week 64	To assess the efficacy of eculizumab in participants with PNH
Pharmacokinetics (PK): Serum Eculizumab Concentration	Baseline through Week 64 (predose and postdose)	To characterize the pharmacokinetics of eculizumab in participants with PNH
Change from baseline in Serum Free and total Complement 5 (C5) Concentration	Baseline through week 64 (predose and postdose)	To characterize the pharmacodynamics of eculizumab in participants with PNH
Number(%) of participants with Antidrug Antibodies (ADAs) to Eculizumab	Baseline through Week 64	To characterize the immunogenicity of eculizumab in participants with PNH
Number(%) of participants with Breakthrough Hemolysis	Baseline through Week 64	To assess the efficacy of eculizumab in participants with PNH
Number(%) of participants changes from baseline in vital signs and laboratory parameters at all scheduled visits.	Baseline through Week 64	To characterize the safety profile of eculizumab in participants with PNH

Trial Locations

Locations (1)

Research Site; 🇨🇳 Wuhan, China