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Completed

NCT Number: NCT01710826

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

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.

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Key information

Conditions

Healthy

Age range

18 year–45 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Austin, Texas, United States

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Genz-682452

Biological

Capsules for oral administration.

Placebo

Biological

Placebo capsules matching the Genz-682452 capsules.

Primary outcomes

  1. Participants with Treatment-Emergent Adverse Events (TEAEs)

    Time frame: Day 1 through Day 14

Secondary outcomes

  1. Pharmacokinetics as measure by plasma parameters

    Time frame: 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)

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

    Time frame: Day 1, through Day 14

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

    Time frame: Day 1 through Day 14

Sponsors and collaborators

Lead sponsor

Genzyme, a Sanofi Company

Industry

Registry information

Official study 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

Important dates

Study start
2012
Primary completion
2013
Study completion
2013
First posted
Oct 19, 2012
Registry last updated
Mar 19, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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