A Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Genz-682452 in Healthy Volunteers

Genzyme, a Sanofi Company36 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

BASIC_SCIENCE

Masking

DOUBLE

Enrollment

Conditions

Healthy

Interventions

Genz-682452BIOLOGICAL

Capsules for oral administration.

PlaceboBIOLOGICAL

Placebo capsules matching the Genz-682452 capsules.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 45 YearsHealthy volunteers:

Medical Language ↔ Plain English

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.

Locations (1)

Unnamed facility

Austin, Texas, United States

Outcomes

Primary Outcomes

Participants with Treatment-Emergent Adverse Events (TEAEs)

Time frame: Day 1 through Day 14

Secondary Outcomes

Pharmacokinetics as measure by plasma parameters

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)

Time frame: Day 1 through Day 14

Pharmacodynamics as measure by Plasma concentrations of Glucosylceramide (GL-1), Globotriaosylceramide (GL-3) and GM3 ganglioside (GM3)

Time frame: Day 1, through Day 14

Pharmacokinetics Urine parameter as measure by assessment of Genz-682452 in Urine, Ae0-t, fe0-t (Exploratory)

Time frame: Day 1 through Day 14

Data from ClinicalTrials.gov

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