Study BTI-101 is a Phase 1, randomized, double-blind, placebo-controlled, dose escalation, parallel design study to evaluate the safety, tolerability, and pharmacokinetics of IV rhu-pGSN or saline placebo administered as 5 doses each of 6, 12, 18, or 24 mg/kg of body weight. Each of 4 dosing cohorts will include 8 subjects randomized 3:1 rhu-pGSN:placebo (6 rhu-pGSN subjects:2 placebo subjects). Subjects will be healthy adult volunteers 18-55 years of age.
Doses will be administered at 0 hours (Day 1), 12 hours (Day 1), 36 hours (Day 2), 60 hours (Day 3), and 84 hours (Day 4). Subjects will be kept in-house until after the last blood sample is taken on Day 5. Subjects will return for follow-up 7 days after the initiation of therapy (Day 8) and on Day 28 for the End-of-Study (EOS) Visit. After each cohort has completed the Day 8 visit, review of the safety results (including labs) will be conducted (and unblinded where appropriate) before the initiation of the next higher dose cohort. To assess safety and tolerability, subjects will undergo physical examinations (including vital sign measurements), adverse event (AE) assessments, concomitant medication assessments, and safety laboratory testing. Blood samples will be collected for analysis of pGSN levels and antibodies against pGSN.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
32
intravenous administration of either 6, 12, 18, or 24 mg/kg at time 0, 12, 36, 60 and 84 hours
intravenous administration of saline control at time 0, 12, 36, 60 and 84 hours
Nucleus Network
Saint Paul, Minnesota, United States
Adverse Events
Number of subjects who had an Adverse Event
Time frame: 28 days
Serious Adverse Events
Number of subjects who had a Serious Adverse Event
Time frame: 28 days
Pharmacokinetics: pGSN Concentration
Concentration of pGSN at baseline, 12 hours after dose 1, and 24 hours after doses 2 and 5
Time frame: 108 hours
Pharmacokinetics: pGSN Area Under the Curve (AUC)
Area Under the Curve (AUC) of pGSN concentration at 12 hours after dose 1, and 24 hours after doses 2 and 5
Time frame: 108 hours
Pharmacokinetics: pGSN Half-life
Half-life of pGSN concentration 12 hours after dose 1, and 24 hours after doses 2 and 5
Time frame: 108 hours
Presence of Anti-drug Antibodies
Number of participants who tested positive for anti-pGSN antibodies at baseline, and on day 28
Time frame: 28 days
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