The primary purpose of this study is to evaluate the safety, tolerability, and efficacy of multiple intravenous (IV) doses of ravulizumab administered to complement inhibitor treatment-naïve participants with PNH.
The study consisted of a screening period of up to 30 days and a Treatment Period of up to 253 days for Cohorts 1-3 and 281 days for Cohort 4. After completion of the Treatment Period, all participants had the opportunity to enter the Extension Period, wherein participants continue to receive ravulizumab for up to 5 years. The first dose in the Extension Period occurred on Day 253 for Cohorts 1-3 and on Day 281 for Cohort 4. The data presented includes the Primary Completion date of the study for the Treatment Period. The results for the Extension Period will be reported after study completion.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
26
All treatments were given as IV infusions.
Clinical Trial Site
Toronto, Ontario, Canada
Clinical Trial Site
Lyon, Pierre-Bénite, France
Clinical Trial Site
Lille, France
Percent Change In LDH Levels From Baseline To Day 253 And Day 281
The percent change in LDH levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
Time frame: Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Percent Change In Free Hemoglobin Levels From Baseline To Day 253 And Day 281
The percent change in free hemoglobin levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
Time frame: Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Percent Change In Haptoglobin Levels From Baseline To Day 253 And Day 281
The percent change in haptoglobin levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
Time frame: Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Percent Change In Reticulocyte/Erythrocyte Count From Baseline To Day 253 And Day 281
The percent change in reticulocyte/erythrocyte count levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
Time frame: Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Percent Change In PNH RBC Types II And III Clone Size From Baseline To Day 253
The percent change in paroxysmal nocturnal hemoglobinuria (PNH) red blood cell (RBC), summed types II and III, clone size levels were assessed from Baseline to Day 253 for Cohorts 1 to 4.
Time frame: Baseline, Day 253 (Cohorts 1 to 4)
Percent Change In D-dimer From Baseline To Day 253 And Day 281
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Clinical Trial Site
Paris, France
Clinical Trial Site
Ulm, Baden-Wurttemberg, Germany
Clinical Trial Site
Aachen, North Rhine-Westphalia, Germany
Clinical Trial Site
Essen, North Rhine-Westphalia, Germany
Clinical Trial Site
Seoul, South Korea
Clinical Trial Site
Seoul, South Korea
Clinical Trial Site
Badalona, Barcelona, Spain
...and 6 more locations
The percent change in D-dimer levels were assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
Time frame: Baseline to Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Change In Clinical Manifestations Of PNH From Baseline To Day 253 And Day 281
Clinical manifestations were assessed from Baseline to Day 253 for Cohorts 1 to 3 and from Baseline to Day 281 for Cohort 4 only. Clinical manifestations were defined as fatigue, abdominal pain, dyspnea, dysphagia, chest pain, and erectile dysfunction (male participants only). Improvement was defined as present at Baseline and absent at Day endpoint. Worsening was defined as absent at Baseline and present at Day endpoint. No Change was defined as no change from Baseline and time point of endpoint.
Time frame: Baseline, Day 253 (Cohorts 1 to 3) and Day 281 (Cohort 4)