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.
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 Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
25
Participants will receive 600 milligrams (mg) once a week on Day 1, 8, 15, and 22 followed by 900 mg every 2 weeks from Day 29 to Day 435.
Research Site
Beijing, China
Research Site
Hangzhou, China
Research Site
Nantong, China
Research Site
Shanghai, China
Percentage change from baseline in Lactate Dehydrogenase (LDH) at Week 12
To assess efficacy of eculizumab in participants with PNH
Time frame: Baseline, Week 12
Number(%) of participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
To assess the safety and tolerability of eculizumab in participants with PNH
Time frame: Baseline through Week 64
Pharmacokinetics (PK): Serum Eculizumab Concentration
To characterize the pharmacokinetics of eculizumab in participants with PNH
Time frame: Baseline through Week 64 (predose and postdose)
Change from baseline in Serum Free and total Complement 5 (C5) Concentration
To characterize the pharmacodynamics of eculizumab in participants with PNH
Time frame: Baseline through week 64 (predose and postdose)
Number(%) of participants with Antidrug Antibodies (ADAs) to Eculizumab
To characterize the immunogenicity of eculizumab in participants with PNH
Time frame: Baseline through Week 64
Change from baseline in functional assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 64
To assess the efficacy of eculizumab in participants with PNH
Time frame: Baseline through Week 64
Number(%) of participants with Breakthrough Hemolysis
To assess the efficacy of eculizumab in participants with PNH
Time frame: Baseline through Week 64
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Research Site
Tianjin, China
Research Site
Tianjin, China
Research Site
Wuhan, China
Number(%) of participants achieving LDH Normalization
To assess the efficacy of eculizumab in participants with PNH
Time frame: Baseline through Week 64
Number(%) of participants needing Blood Transfusion
To assess the efficacy of eculizumab in participants with PNH
Time frame: Baseline through Week 64
Number(%) of participants changes from baseline in vital signs and laboratory parameters at all scheduled visits.
To characterize the safety profile of eculizumab in participants with PNH
Time frame: Baseline through Week 64