Brief Summary

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Official Title: "A Single-center, Double-blind, Randomized, Placebo-controlled Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Ascending 14-day Repeated Oral Doses of Genz-682452 in Healthy Male and Female Subjects"

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
• Study Design: Allocation: Randomized, Endpoint Classification: Pharmacokinetics/Dynamics Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Basic Science
• Study Primary Completion Date: February 2013

Interventions Used in this Clinical Trial

• Biological: Genz-682452
  • Capsules for oral administration.
• Biological: Placebo
  • Placebo capsules matching the Genz-682452 capsules.

Arms, Groups and Cohorts in this Clinical Trial

• Experimental: Genz-682452
  • This study will include three cohorts for doses of Genz-682452: Dose 1, Dose 2, Dose 3. Each cohort will include 12 participants, 9 of whom will be administered Genz-682452 and 3 will be administered placebo.
• Placebo Comparator: Placebo
  • Placebo comparator taken by participants randomized to the placebo arm in each of the three cohorts. Each cohort will include 12 participants, 9 of whom will be administered Genz-682452 and 3 will be administered placebo.

Outcome Measures for this Clinical Trial

Primary Measures

• Participants with Treatment-Emergent Adverse Events (TEAEs)
  • Time Frame: Day 1 through Day 14
    Safety Issue?: Yes

Secondary Measures

• Pharmacokinetics as measure by plasma parameters
  • Time Frame: Day 1 through Day 14
    Safety Issue?: No
• Pharmacodynamics as measure by Plasma concentrations of Glucosylceramide (GL-1), Globotriaosylceramide (GL-3) and GM3 ganglioside (GM3)
  • Time Frame: Day 1, through Day 14
    Safety Issue?: No
• Pharmacokinetics Urine parameter as measure by assessment of Genz-682452 in Urine, Ae0-t, fe0-t (Exploratory)
  • Time Frame: Day 1 through Day 14
    Safety Issue?: No

Criteria for Participation in this Clinical Trial

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.

Gender Eligibility for this Clinical Trial: Both

Minimum Age for this Clinical Trial: 18 Years

Maximum Age for this Clinical Trial: 45 Years

Are Healthy Volunteers Accepted for this Clinical Trial: Accepts Healthy Volunteers

Clinical Trial Investigator Information

• Lead Sponsor
  • Genzyme
• Provider of Information About this Clinical Study
  • Sponsor
• Overall Official(s)
  • Medical Monitor, Study Director, Genzyme