NCT01710826

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
36

participants targeted

Target at P50-P75 for phase_1 healthy

Timeline
Completed

Started Oct 2012

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2012

Completed
16 days until next milestone

First Submitted

Initial submission to the registry

October 17, 2012

Completed
2 days until next milestone

First Posted

Study publicly available on registry

October 19, 2012

Completed
4 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2013

Completed
Last Updated

March 19, 2014

Status Verified

March 1, 2014

Enrollment Period

4 months

First QC Date

October 17, 2012

Last Update Submit

March 17, 2014

Conditions

Healthy

Outcome Measures

Primary Outcomes (1)

  • Participants with Treatment-Emergent Adverse Events (TEAEs)

    Day 1 through Day 14

Secondary Outcomes (3)

  • Pharmacokinetics as measure by plasma parameters

    Day 1 through Day 14

  • 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

Study Arms (2)

Genz-682452

EXPERIMENTAL

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

Placebo

PLACEBO COMPARATOR

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

Interventions

Genz-682452BIOLOGICAL

Capsules for oral administration.

Genz-682452
PlaceboBIOLOGICAL

Placebo capsules matching the Genz-682452 capsules.

Placebo

Eligibility Criteria

Age18 Years - 45 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Unknown Facility

Austin, Texas, United States

Location

Related Publications (1)

  • Peterschmitt MJ, Crawford NPS, Gaemers SJM, Ji AJ, Sharma J, Pham TT. Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Oral Venglustat in Healthy Volunteers. Clin Pharmacol Drug Dev. 2021 Jan;10(1):86-98. doi: 10.1002/cpdd.865. Epub 2020 Aug 26.

    PMID: 32851809DERIVED

MeSH Terms

Interventions

venglustat

Study Officials

  • Medical Monitor

    Genzyme, a Sanofi Company

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
BASIC SCIENCE
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 17, 2012

First Posted

October 19, 2012

Study Start

October 1, 2012

Primary Completion

February 1, 2013

Study Completion

February 1, 2013

Last Updated

March 19, 2014

Record last verified: 2014-03

Locations

US(1)