This Phase 1 study is being conducted to evaluate 3 increasing subcutaneous (SC) doses (50, mg, 100 mg or 200mg) of mipomersen in Japanese healthy volunteers. Eligible subjects will receive a single study injection of either mipomersen or placebo. Subjects will be enrolled into 1 of 3 treatment cohorts (Cohorts A, B, and C) in a dose-escalation design. Dose-escalation will proceed only if there is an acceptable safety profile from the previous dosing level.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
20
50 mg (cohort A), 100mg (cohort B) or 200mg (cohort C) subcutaneous (SC) single dose of study drug
50 mg (cohort A), 100mg (cohort B), or 200mg (cohort C) subcutaneous (SC) single dose of study drug
FOCUS Clinical Drug Development GmbH
Stresemannallee 6, Neuss, Germany
Maximum plasma concentration (Cmax)
plasma PK parameters
Time frame: Baseline up to Day 36 Post-Treatment
Time to maximal concentration (Tmax)
plasma PK parameters
Time frame: Baseline up to Day 36 Post-Treatment
Area Under the Curve (AUC)
plasma PK parameters
Time frame: Baseline up to Day 36 Post-Treatment
Number of Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Up to Day 36 Post-Treatment
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