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 LDH at Week 12
Blood samples were collected for measurement of serum LDH at specified timepoints. Statistical analysis was performed using a mixed model for repeated measures (MMRM), including the percentage change from baseline of LDH at the scheduled visits from Week 1 to Week 12 as the dependent variable, with the fixed categorical effect of visit, fixed continuous effect of LDH baseline value as covariates, and participant as random effect. Assessed at baseline, 1, 2, 3, 4, 6, 8, 10, and 12 weeks, Week 12 reported. A decrease indicated a reduction in disease activity.
Time frame: Baseline, Week 12
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product that did not necessarily have to have a causal relationship with the study intervention. A TEAE was any AE that started during or after the first infusion of the study intervention. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Time frame: Baseline through Week 64
Serum Concentration of Eculizumab
Blood samples were collected for measurement of serum concentrations of eculizumab at specified timepoints. No study intervention administered on Day 449. Results reported as micrograms/milliliter (ug/mL).
Time frame: Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449
Change From Baseline in Serum Free Complement 5 (C5) Concentration
Blood samples were collected for measurement of serum concentration of free C5 at specified timepoints. No study intervention administered on Day 449. A decrease indicated a reduction in disease activity.
Time frame: Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449
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Research Site
Tianjin, China
Research Site
Tianjin, China
Research Site
Wuhan, China
Change From Baseline in Serum Total C5 Concentration
Blood samples were collected for measurement of serum concentration of total C5 at specified timepoints. No study intervention administered on Day 449. A decrease indicated a reduction in disease activity.
Time frame: Day 1, Day 29, Day 85, Day 169, Day 253, Day 337, Day 421, Day 449
Number of Participants With Treatment-emergent Antidrug Antibodies (ADAs) to Eculizumab
Blood samples were collected for measurement of treatment-emergent ADAs to eculizumab at specified timepoints.
Time frame: Baseline through Week 64
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score at Week 12
The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. The FACIT-Fatigue scale is a collection of quality of life (QoL) questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. Participants scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores were calculated as the sum from all 13 items and ranged from 0 to 52, with higher scores indicating an increased QoL. Analysis was performed using a MMRM, including FACIT-Fatigue score change from baseline values at the scheduled visits from Week 1 to Week 12 as the dependent variable, with the fixed categorical effect of study visit, fixed continuous effect of baseline value of FACIT-Fatigue, and participant as random effect. Assessed at baseline, 1, 2, 3, 4, 6, 8, 10, and 12 weeks, Week 12 reported.
Time frame: Baseline, Week 12
Percentage of Participants With Breakthrough Hemolysis
Breakthrough hemolysis was defined as at least 1 new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath \[dyspnea\], anemia \[hemoglobin \<10 grams/deciliter\], major adverse vascular event \[including thrombosis\], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 times (\*) upper limit of normal (ULN), after prior LDH reduction to \<1.5\* ULN on therapy.
Time frame: Baseline through Week 12
Percentage of Participants Achieving LDH Normalization
Blood samples were collected for measurement of LDH at specified timepoints. LDH normalization was defined as LDH ≤ULN.
Time frame: Baseline through Week 12
Percentage of Participants Achieving Transfusion Avoidance
Transfusion avoidance was defined as remaining transfusion free (that is, had not received any transfusion) and did not require transfusion as per protocol.
Time frame: Baseline through Week 12
Percentage of Participants With LDH ≤1.5* ULN at Week 12
Blood samples were collected for measurement of LDH at specified timepoints.
Time frame: Baseline through Week 12