Open-Label, Multicenter Study to Assess the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Complement Inhibitor Treatment Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

Alexion Pharmaceuticals, Inc.1 site in 1 country25 target enrollmentJuly 5, 2023

ConditionsParoxysmal Nocturnal Hemoglobinuria

InterventionsEculizumab

DrugsEculizumab

Overview

Phase: Phase 3
Intervention: Eculizumab
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Sponsor: Alexion Pharmaceuticals, Inc.
Enrollment: 25
Locations: 1
Primary Endpoint: Percentage change from baseline in Lactate Dehydrogenase (LDH) at Week 12
Status: Completed
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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

Registry

clinicaltrials.gov

Start Date

July 5, 2023

End Date

April 14, 2025

Last Updated

last year

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Alexion Pharmaceuticals, Inc.

Responsible Party

Sponsor

Eligibility Criteria

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

Arms & Interventions

Eculizumab

Eculizumab will be administered by IV infusion.

Intervention: Eculizumab

Outcomes

Primary Outcomes

Percentage change from baseline in Lactate Dehydrogenase (LDH) at Week 12

Time Frame: Baseline, Week 12

To assess efficacy of eculizumab in participants with PNH

Secondary Outcomes

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