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Terminated Phase 1 Interventional

A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101

ClinicalTrials.gov ID: NCT00875160

Public ClinicalTrials.gov record NCT00875160. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 12, 2026, 2:35 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

An Open-Label Study to Assess the Pharmacokinetics, Safety, and Pharmacodynamics of Repeated Doses of Orally Administered AT2101 in Adult Patients With Type 1 Gaucher Disease

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

This is an open-label study designed to assess if AT2101 is safe in patients with Gaucher disease and how AT2101 gets through the body after it is taken by mouth. The study is being offered to adult patients with type 1 Gaucher disease who are currently receiving a stable dose of enzyme replacement therapy (ERT) with imiglucerase. During the study, subjects will not be receiving ERT (up to 35 days). The study consists of a screening period (\~14 days), a treatment period (12 days) and a follow-up period (7 days after last dose). At two points in the study, subjects will be housed in an in-patient treatment facility for 3 days/2 nights to accommodate all necessary blood draws.

Study identification

NCT ID
NCT00875160
Recruitment status
Terminated
Study type
Interventional
Phase
Phase 1
Lead sponsor
Amicus Therapeutics
Industry
Enrollment
1 participant

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
18 Years to 65 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Mar 31, 2009
Primary completion
Jun 30, 2009
Completion
Jun 30, 2009
Last update posted
Aug 18, 2010

2009

United States locations

U.S. sites
1
U.S. states
1
U.S. cities
1
Facility City State ZIP Site status
Comprehensive Phase One Miramar Florida 33025

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT00875160, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Aug 18, 2010 · Synced Sep 12, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT00875160 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →