The purpose of the study is to evaluate the safety and efficacy of zilucoplan (RA101495) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). There will be two groups of patients in the study: the first group will include patients who have never received eculizumab for treatment of PNH. The second group will include patients who have received treatment with eculizumab for at least 6 months prior to the study. Patients will be treated with RA101495 for 12 weeks.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
26
0.3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Investigative Site
Gosford, New South Wales, Australia
Investigative Site
Parkville, Australia
Investigative Site
Toronto, Ontario, Canada
Investigative Site
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level.
The primary efficacy endpoint is the change-from-baseline in serum LDH levels during this period, defined as the mean LDH values of Weeks 6, 8, 10, and 12 minus the baseline value of LDH.
Time frame: Through Week 12 of the study
Changes From Baseline in Bilirubin Values
Changes from baseline at each of the scheduled post-baseline time-points
Time frame: Through Week 12 of the study
Total Hemoglobin
Changes from baseline at each of the scheduled post-baseline time-points
Time frame: Through Week 12 of the Study
Changes From Baseline in Free Hemoglobin Values
Changes from baseline at each of the scheduled post-baseline time-points
Time frame: Through Week 12 of the study
Haptoglobin Values
Changes from baseline at each of the scheduled post-baseline time-points
Time frame: Through Week 12 of the Study
Reticulocyte Values
Changes from baseline at each of the scheduled post-baseline time-points
Time frame: Through Week 12 of the Study
Hemoglobinuria Values
Changes from baseline at each of the scheduled post-baseline time-points; Hemoglobinuria was assessed using a urine colorimetric scoring system with a score of 1 through 10. Where 1 represents no hemoglobinuria and 10 represents maximum hemoglobinuria.
Time frame: Through Week 12 of the Study
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Copenhagen, Denmark
Investigative Site
Helsinki, Finland
Investigative Site
Essen, Germany
Investigative Site
Ulm, Germany
Investigative Site
Budapest, Hungary
Investigative Site
Christchurch, New Zealand
Investigative Site
Hamilton, New Zealand
...and 2 more locations