This study evaluated the safety and tolerability of single and multiple doses (400 and 800 milligrams \[mg\]) of ALXN1210 following intravenous administration to healthy Japanese participants.
A total of 3 cohorts were enrolled sequentially. Participants received different doses per Cohorts: Cohort 1, 400 mg single dose; Cohort 2, 800 mg single dose; and Cohort 3, 800 mg every 4 weeks for a total of 5 doses. The Safety Review Committee (SRC) conducted a review of the available clinical and safety data after the last participants in the 400 mg cohort (Cohort 1) completed Day 15 to determine if dose escalation to the single dose 800 mg (Cohort 2) could proceed. The SRC then conducted a review of all available clinical and safety data after the last participants in Cohort 2 completed Day 15 to determine if dosing of Cohort 3 could begin. A 120-day (Cohort 1) or 140-day (Cohort 2) Follow-up Period was performed for safety, pharmacokinetic (PK), pharmacodynamic (PD), and immunogenicity assessments. Participants in Cohort 3, however, had a 185-day Follow-up Period for safety, PK, PD, and immunogenicity assessments after the fifth dose of study drug.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
BASIC_SCIENCE
Masking
NONE
Enrollment
16
Participants received a single dose (400 mg or 800 mg) and multiple doses (800 mg) of ALXN1210.
Clinical Trial Site
London, United Kingdom
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An adverse event (AE) was defined as any unfavorable and unintended sign (for example, including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product or procedure, whether or not considered related to the medicinal product or procedure, which occurred during the course of the clinical study. TEAEs were defined as AEs that occurred on or after the date and time of study drug administration, or those that first occurred before dosing but worsened in frequency or severity after study drug administration. 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: Cohort 1: Baseline up to Day 120; Cohort 2: Baseline up to Day 140; Cohort 3: Baseline up to Day 298
Number of Participants With Anti-Drug Antibodies (ADAs) To ALXN1210
Blood samples were collected to evaluate antibody response through development of ADAs.
Time frame: Cohort 1: Baseline up to Day 120; Cohort 2: Baseline up to Day 140; Cohort 3: Baseline up to Day 298
Area Under The Serum Concentration Versus Time Curve From Time Zero To Infinity (AUCinf) For Single Dose of ALXN1210
Blood samples were collected for estimation of AUCinf using Phoenix WinNonlin software version 7.0.
Time frame: Cohort 1: Baseline (pre-dose) up to Day 120; Cohort 2: Baseline (pre-dose) up to Day 140
Area Under The Serum Concentration Versus Time Curve From Time Zero To Infinity (AUCinf) For Multiple Dose of ALXN1210
Blood samples were collected for estimation of AUCinf using Phoenix WinNonlin software version 7.0.
Time frame: Day 113 (pre-dose) up to Day 298
Maximum Observed Serum Concentration (Cmax) For Single Dose of ALXN1210
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Blood samples were collected for estimation of Cmax using Phoenix WinNonlin software version 7.0.
Time frame: Cohort 1: Baseline (pre-dose) up to Day 120; Cohort 2: Baseline (pre-dose) up to Day 140
Maximum Observed Serum Concentration (Cmax) For Multiple Dose of ALXN1210
Blood samples were collected for estimation of Cmax using Phoenix WinNonlin software version 7.0.
Time frame: Day 113 (pre-dose) up to Day 298
Percent Change From Baseline in Free Complement Protein C5 Concentration at Day 1
Time frame: Baseline, Day 1 (end of infusion)
Percent Change From Baseline in Free Complement Protein C5 Concentration at Day 113
Time frame: Baseline, Day 113 (end of infusion)
Percent Change From Baseline in Total C5 Concentration at Day 1
Time frame: Baseline, Day 1 (end of infusion)
Percent Change From Baseline in Total C5 Concentration at Day 113
Time frame: Baseline, Day 113 (end of infusion)
Percent Change From Baseline in Chicken Red Blood Cell Hemolysis at Day 1
Time frame: Baseline, Day 1 (end of infusion)
Percent Change From Baseline in Chicken Red Blood Cell Hemolysis at Day 113
Time frame: Baseline, Day 113 (end of infusion)