The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with PNH.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
18
Ravulizumab will be administered by intravenous (IV) infusion.
Research Site
Beijing, China
Research Site
Guangzhou, China
Research Site
Hangzhou, China
Research Site
Nantong, China
Percentage Change in Lactate Dehydrogenase (LDH) From Baseline to Day 183 (Week 26)
LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicated reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first dose of study drug. The percent change in LDH was analyzed using a mixed-effect model for repeated measures (MMRM) with the fixed categorical effect of visit, fixed continuous effect of the LDH baseline value as covariates, and participant as random effect.
Time frame: Baseline, Day 183 (Week 26)
Percentage of Participants Achieving LDH <1.5 * Upper Limit of Normal (ULN) at Day 183 (Week 26)
LDH is an indicator of intravascular hemolysis that occurs in participants with paroxysmal nocturnal hemoglobinuria. A decrease in LDH indicated reduction (improvement) in hemolysis. Baseline was defined as the average of all available on-study assessments prior to the first dose of study drug.
Time frame: Day 183 (Week 26)
Percentage of Participants Achieving Transfusion Avoidance Through Day 183 (Week 26)
Transfusion avoidance was defined as the percentage of participants who remained transfusion free and did not require a transfusion per protocol-specified guidelines (hemoglobin value of ≤9 grams (g)/deciliter (dL) with signs or symptoms of sufficient severity to warrant a transfusion, or a hemoglobin value of ≤7 g/dL regardless of presence of clinical signs or symptoms) through Day 183.
Time frame: Day 183 (Week 26)
Percentage of Participants Experiencing Breakthrough Hemolysis Through Day 183 (Week 26)
Breakthrough hemolysis was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia \[hemoglobin \<10 g/dL\], major adverse vascular event \[including thrombosis\], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 times the ULN.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Research Site
Shanghai, China
Research Site
Tianjin, China
Research Site
Tianjin, China
Research Site
Wuhan, China
Time frame: Day 183 (Week 26)
Change in Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue) Score From Baseline to Day 183 (Week 26)
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. Participants scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with a higher score indicating better quality of life. Analysis was using a mixed-effect model for repeated measures (MMRM) with the fixed categorical effect of visit, fixed continuous effect of the baseline value of FACIT-Fatigue score as covariates, and participant as random effect.
Time frame: Baseline, Day 183 (Week 26)
Change in Hemoglobin (Hgb) From Baseline to Day 183 (Week 26)
Analysis was performed using MMRM with the fixed categorical effect of visit, fixed continuous effect of the Hgb baseline value as covariates, and participant as random effect.
Time frame: Baseline, Day 183 (Week 26)