NCT01674036

Brief Summary

The objectives of this study are to assess the tolerability, safety and pharmacokinetic parameters of Genz-682452 in healthy adult males, in two parts. Part 1 (GZFD00111TDU12766): double-blind, randomized, placebo-controlled, sequential ascending single dose study; Part 2 (GZFD00211FED12767): open-label, randomized, 2-sequence, 2-period, 2-treatment crossover study with a minimum wash-out period; to obtain preliminary information on the pharmacokinetics, tolerability and safety of Genz-682452 after single oral doses in fed and fasted conditions. Subjects are not allowed to participate in more than 1 part of the study.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
63

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Aug 2012

Shorter than P25 for phase_1

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

August 1, 2012

Completed
23 days until next milestone

First Submitted

Initial submission to the registry

August 24, 2012

Completed
4 days until next milestone

First Posted

Study publicly available on registry

August 28, 2012

Completed
4 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2013

Completed
Last Updated

March 19, 2014

Status Verified

March 1, 2014

Enrollment Period

5 months

First QC Date

August 24, 2012

Last Update Submit

March 17, 2014

Conditions

Healthy Volunteer

Outcome Measures

Primary Outcomes (2)

  • Number of participants with adverse events in Part 1

    Up to 4 weeks

  • Number of participants with adverse events in Part 2

    Up to 5 weeks

Secondary Outcomes (3)

  • Pharmacokinetics Plasma Parameters for Part 1 as measured by Cmax, tmax, AUC last, AUC, t1/2z, Vss/F, CL/F

    Day 1 through Day 5

  • Pharmacokinetics Urine Parameters for Part 1 as measured by assessment of Genz-682452 urinary excretion, Ae0-t, fe0-t

    Day 1 through Day 5

  • Pharmacokinetics Plasma Parameters for Part 2 as measured by Cmax, tmax, tlag, AUC last, AUC, t1/2z

    Day 1 to Day 3

Study Arms (3)

Part 1 (GZFD00111/TDU12766): Genz-682452

EXPERIMENTAL

Participants will receive a single oral dose of Genz-682452. Six ascending single doses and an optional seventh dose under fasted conditions will be used.

Drug: Genz-682452

Part 1 (GZFD00111/TDU12766): Placebo

PLACEBO COMPARATOR

Participants will receive a single oral dose of placebo.

Drug: Placebo

Part 2 (GZFD00211/FED12767): Genz-682452

EXPERIMENTAL

Participants will receive two single doses of Genz-682452 separated by a 7-day wash-out period, one dose given under fed (standardized high-fat breakfast) and one under fasted conditions. The dose will be based on the blind review of the safety/tolerability/pharmacokinetic data of single dose level cohorts in Part 1.

Drug: Genz-682452

Interventions

Genz-682452DRUG

Capsules for oral administration.

Part 1 (GZFD00111/TDU12766): Genz-682452Part 2 (GZFD00211/FED12767): Genz-682452
PlaceboDRUG

Placebo to Genz-682452

Part 1 (GZFD00111/TDU12766): Placebo

Eligibility Criteria

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

You may qualify if:

  • Body weight between 50.0 and 100.0 kg, inclusive, body mass index between 18.0 and 32.0 kg/m\^2, inclusive.
  • Certified as healthy by a comprehensive clinical assessment (detailed medical history and complete physical examination).

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, or infectious disease, or signs of acute illness.
  • Frequent headaches and/or migraine, recurrent nausea and/or vomiting.
  • Presence or history of drug hypersensitivity, or allergic disease diagnosed and treated by a physician.
  • History or presence of drug or alcohol abuse.
  • Unable to stop smoking cigarettes during the study.
  • Excessive consumption of beverages containing xanthine bases.

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
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 24, 2012

First Posted

August 28, 2012

Study Start

August 1, 2012

Primary Completion

January 1, 2013

Study Completion

January 1, 2013

Last Updated

March 19, 2014

Record last verified: 2014-03

Locations

US(1)