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Completed Phase 3 Interventional Results available

Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease

ClinicalTrials.gov ID: NCT01218659

Public ClinicalTrials.gov record NCT01218659. 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, 7:19 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 Randomized, Open-Label Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT

Brief summary

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

Study to compare the efficacy and safety of migalastat and enzyme replacement therapy (ERT) in male and female participants with Fabry disease who are currently receiving ERT and who have an alpha galactosidase-A (α Gal-A) mutation that is amenable to migalastat, based on the clinical trial human embryonic kidney cell (HEK) assay.

Study identification

NCT ID
NCT01218659
Recruitment status
Completed
Study type
Interventional
Phase
Phase 3
Lead sponsor
Amicus Therapeutics
Industry
Enrollment
68 participants

Conditions and interventions

Conditions

Interventions

Drug · Biological

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
Sep 7, 2011
Primary completion
May 26, 2014
Completion
May 27, 2015
Last update posted
Oct 31, 2018

2011 – 2015

United States locations

U.S. sites
8
U.S. states
8
U.S. cities
8
Facility City State ZIP Site status
Not listed Los Angeles California 90048
Not listed Aurora Colorado 80045
Not listed Decatur Georgia 30033
Not listed Grand Rapids Michigan 49525
Not listed Portland Oregon 97239
Not listed Pittsburgh Pennsylvania 15224
Not listed Fairfax Virginia 22030
Not listed Milwaukee Wisconsin 53226

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 17 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT01218659, 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 31, 2018 · Synced Sep 2, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT01218659 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →