The purpose of this study is to enable continued access to zilucoplan (RA101495) for patients with paroxysmal nocturnal hemoglobinuria (PNH) after they complete a zilucoplan clinical study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
19
Subjects will continue to receive the final maintenance dose they were receiving in the qualifying study.
Investigative Site 4
Los Angeles, California, United States
Investigative Site 19
Dallas, Texas, United States
Investigative Site 3
Gosford, Australia
Investigative Site 5
Percentage of Participants With Treatment Emergent Adverse Events (TEAEs)
TEAEs were defined as an AE that occurs after a participant's initial treatment zilucoplan start for this study (RA101495-01.202) that was not present at the time of treatment start, or an AE that increases in severity after treatment start in this study, if the event was present at the time of treatment start.
Time frame: From Day 1 until the Final Study Visit (up to Month 49)
Percentage of Participants With Serious TEAEs
Serious Adverse event (SAE) was defined as any untoward medical occurrence that:• results in death, • is life-threatening threatening (note that this refers to an event in which the participant was at risk of death at the time of the event; it does not refer to an event that hypothetically might have caused death if it were more severe), • requires hospitalization or prolongation of existing hospitalization, • results in persistent or significant disability/incapacity, and • results in a congenital anomaly/birth defect.
Time frame: From Day 1 until the Final Study Visit (up to Month 49)
Number of Participants With Anti-drug Antibodies (ADA)
Blood samples collection were planned to analyze for the presence/absence of ADAs to zilucoplan for immunogenicity assessments.
Time frame: At Day 1, Month 1, 2, 3, 6, 9, and 12
Change From Baseline in Serum Lactate Dehydrogenase (LDH) Levels at Each Time Point
Serum LDH levels were measure of intravascular hemolysis. As high level of LDH in the blood was indicative of hemolysis in participants with PNH.
Time frame: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
Change From Baseline in Total Bilirubin Values at Each Time Point
Total Bilirubin was monitored for signs and symptoms of hepatic or biliary dysfunction.
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Melbourne, Australia
Investigative Site 10
Toronto, Canada
Investigative Site 14
Helsinki, Finland
Investigative Site 9
Ulm, Germany
Investigative Site 17
Budapest, Hungary
Investigative Site 13
Christchurch, New Zealand
Investigative Site 12
Hamilton, New Zealand
...and 2 more locations
Time frame: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
Change From Baseline in Total Hemoglobin Values at Each Time Point
Total Hemoglobin Values were analyzed for hematology assessments.
Time frame: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
Change From Baseline in Free Hemoglobin Values at Each Time Point
Free Hemoglobin Values were analyzed for hematology assessments.
Time frame: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
Change From Baseline in Haptoglobin Values at Each Time Point
Haptoglobin values were analyzed for hematology assessments.
Time frame: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
Change From Baseline in Reticulocytes at Each Time Point
Reticulocytes values were analyzed for hematology assessments.
Time frame: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, and Final Study Visit (Month 49)
Change From Baseline in Hemoglobinuria Values at Each Time Point
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: Baseline, Month 1, 2, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48 and Final Study Visit (Month 49)
Plasma Concentrations of RA101495 and Its Major Metabolite(s)
Blood samples of RA101495 (zilucoplan) and its metabolites (RA102758 and RA103488) were collected for Plasma concentration analysis.
Time frame: Predose: At Day 1 (Screening), Month 1, 2, 3, 6, 9, 12, and Final Study Visit (Month 49)
Maximum Plasma Concentration (Cmax) of RA101495
Cmax is the maximum plasma concentration.
Time frame: At Day 1, Month 1, 2, 3, 6, 9, and 12
Time Corresponding to Cmax (Tmax) of RA101495
tmax is the time to corresponding Cmax.
Time frame: At Day 1, Month 1, 2, 3, 6, 9, and 12
Area Under the Drug Concentration-time Curve (AUC0-t) of RA101495
AUC0-t is area under the drug concentration-time curves.
Time frame: At Day 1, Month 1, 2, 3, 6, 9, and 12
Total Complement (CH50) Levels
Blood samples collection were planned to assess complement (CH50) levels. The planned analysis of CH50 was not performed because the CH50 assay was not able to be validated due to lack of reproducibility of the manufacturer's kits.
Time frame: At Day 1, Month 1, 2, 3, 6, 9, and 12
Change From Baseline in Sheep Red Blood Cell (sRBC) Values at Each Time Point
Blood samples were collected for measurement of sRBC lysis for the Classical Complement Pathways.
Time frame: Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49)
Change From Baseline in Wieslab Enzyme-linked Immunosorbent Assay (ELISA) Values for Alternative Complement Pathway at Each Time Point
Blood samples were collected for measurement of membrane attack complex (MAC) by Wieslab ELISA for alternative complement pathway.
Time frame: Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49)
Change From Baseline in Complement Component 5 (C5) Values at Each Time Point
Blood samples were collected for measurement of Complement component 5 (C5) levels.
Time frame: Baseline, Month 1, 2, 3, 6, 9, 12 and Final Study Visit (Month 49)