Healthy
Conditions
Brief summary
To assess in healthy adult subjects: * The tolerability and safety of ascending repeated oral doses of Genz-682452. * The pharmacokinetic parameters of Genz-682452 after ascending repeated oral doses. * The pharmacodynamics of Genz-682452 after ascending repeated oral doses.
Interventions
BIOLOGICALGenz-682452
Capsules for oral administration.
BIOLOGICALPlacebo
Placebo capsules matching the Genz-682452 capsules.
Sponsors
Genzyme, a Sanofi Company
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
BASIC_SCIENCE
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 45 Years
Healthy volunteers
Yes
Inclusion criteria
* Body weight between 50.0 and 95.0 kg, inclusive, if male and between 40.0 and 85.0 kg inclusive, if female, body mass index between 18.0 and 30.0 kg/m\^2, inclusive. * Certified as healthy by a comprehensive clinical assessment.
Exclusion criteria
* Any history or presence of clinically relevant cardiovascular, pulmonary, gastrointestinal, hepatic, renal, metabolic, hematological, neurological, osteomuscular, articular, psychiatric, systemic, ocular, gynecologic (if female), or infectious disease, or signs of acute illness. * Frequent headaches and/or migraine, recurrent nausea and/or vomiting. * Blood donation, any volume, within 2 months before inclusion. * Symptomatic postural hypotension. * History or presence of drug or alcohol abuse. * Positive result on any of the following tests: hepatitis B surface (HBs Ag) antigen, antihepatitis C virus (anti-HCV) antibodies, anti-human immunodeficiency virus 1 and 2 antibodies (anti-HIV1 and anti HIV2 Ab). * Positive result on urine drug screen.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Participants with Treatment-Emergent Adverse Events (TEAEs) | Day 1 through Day 14 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetics as measure by plasma parameters | Day 1 through Day 14 | Maximum plasma concentration (Cmax), Plasma Concentration Observed Just Before Treatment Administration During Repeated Dosing (Ctrough), First time to reach Cmax (Tmax), Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval 0-23 hours (AUC0-24), Terminal Half-Life (t1/2z), Apparent Total Body Clearance of Genz-682452 at steady state (CLss/F), plasma 4 beta-hydroxycholesterol (4β-HC) |
| Pharmacodynamics as measure by Plasma concentrations of Glucosylceramide (GL-1), Globotriaosylceramide (GL-3) and GM3 ganglioside (GM3) | Day 1, through Day 14 | — |
| Pharmacokinetics Urine parameter as measure by assessment of Genz-682452 in Urine, Ae0-t, fe0-t (Exploratory) | Day 1 through Day 14 | — |
Countries
United States
Outcome results
None listed