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Completed Phase 3 Interventional Results available

Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

ClinicalTrials.gov ID: NCT00925301

Public ClinicalTrials.gov record NCT00925301. 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 2, 2026, 1:36 AM EDT

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

Official title

A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Pharmacodynamics of AT1001 in Patients With Fabry Disease and AT1001-Responsive GLA Mutations

Brief summary

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

The primary objective of this study was to compare the effect of migalastat (123 milligrams \[mg\] of migalastat \[equivalent to 150 mg of migalastat hydrochloride\]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).

Study identification

NCT ID
NCT00925301
Recruitment status
Completed
Study type
Interventional
Phase
Phase 3
Lead sponsor
Amicus Therapeutics
Industry
Enrollment
67 participants

Conditions and interventions

Conditions

Interventions

Drug

Eligibility (public fields only)

Age range
16 Years to 74 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
Oct 22, 2009
Primary completion
Jun 11, 2012
Completion
Jan 28, 2014
Last update posted
Oct 29, 2018

2009 – 2014

United States locations

U.S. sites
13
U.S. states
12
U.S. cities
13
Facility City State ZIP Site status
Not listed Los Angeles California 90048
Not listed San Francisco California 94143
Not listed Decatur Georgia 30033
Not listed Chicago Illinois 60611
Not listed Kansas City Kansas 66160
Not listed Boston Massachusetts 02114
Not listed Grand Rapids Michigan 49525
Not listed New York New York 10032
Not listed Pittsburgh Pennsylvania 15224
Not listed Dallas Texas 75226
Not listed Salt Lake City Utah 84132
Not listed Springfield Virginia 22152
Not listed Seattle Washington 98195

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 15 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT00925301, 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 Oct 29, 2018 · Synced Sep 2, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT00925301 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →