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

• Sponsor: Genzyme, a Sanofi Company (Industry)
• Overall Status: Completed
• CT.gov ID: NCT01710826
• Collaborator: (none)
• Enrollment: 36
• Location: 1
• Arms: 2
• Duration (Months): 4
• Patients Per Site Per Month: 8.9

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Healthy	Biological: Genz-682452; Biological: Placebo	Phase 1

Study Design

• Study Type: Interventional
• Actual Enrollment: 36 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Double (Participant, Investigator)
• Primary Purpose: Basic Science
• 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
• Study Start Date: Oct 1, 2012
• Actual Primary Completion Date: Feb 1, 2013
• Actual Study Completion Date: Feb 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
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.	Biological: Genz-682452; Capsules for oral administration.
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.	Biological: Placebo; Placebo capsules matching the Genz-682452 capsules.

Outcome Measures

Primary Outcome Measures

1. Participants with Treatment-Emergent Adverse Events (TEAEs) [Day 1 through Day 14]

Secondary Outcome Measures

1. 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)

2. Pharmacodynamics as measure by Plasma concentrations of Glucosylceramide (GL-1), Globotriaosylceramide (GL-3) and GM3 ganglioside (GM3) [Day 1, through Day 14]

3. Pharmacokinetics Urine parameter as measure by assessment of Genz-682452 in Urine, Ae0-t, fe0-t (Exploratory) [Day 1 through Day 14]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 45 Years
• Sexes Eligible for Study: All
• Accepts 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1		Austin	Texas	United States

Sponsors and Collaborators

• Genzyme, a Sanofi Company

Investigators

• Study Director: Medical Monitor, Genzyme, a Sanofi Company

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Genzyme, a Sanofi Company
• ClinicalTrials.gov Identifier: NCT01710826
• Other Study ID Numbers: GZFD00311; TDR12768
• First Posted: Oct 19, 2012
• Last Update Posted: Mar 19, 2014
• Last Verified: Mar 1, 2014

Study Results

No Results Posted as of Mar 19, 2014