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A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease

NCT00446550 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 19

Last updated 2018-08-15

Study results available
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Summary

This study evaluated the safety and tolerability of afegostat tartrate in participants with type 1 Gaucher disease who were not receiving enzyme replacement therapy (ERT) or substrate reduction therapy (SRT).

Conditions

  • Gaucher Disease
  • Type 1 Gaucher Disease
  • Gaucher Disease, Type 1

Interventions

DRUG

afegostat tartrate

Sponsors & Collaborators

  • Amicus Therapeutics

    lead INDUSTRY

Principal Investigators

  • Medical Monitor, Clinical Research · Amicus Therapeutics

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
74 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-06-11
Primary Completion
2009-08-20
Completion
2009-08-20

Countries

  • United States
  • Israel
  • South Africa
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00446550 on ClinicalTrials.gov