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NCT01218659 · ClinicalTrials.gov registry record · Phase 3
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
A Phase 3 study of Fabry Disease, sponsored by Amicus Therapeutics.
- Completed
- Registry status
- Phase 3
- Development phase
- 68
- Enrollment target
- 20
- Study locations
NCT01218659: Completed Phase 3 study of Fabry Disease, sponsored by Amicus Therapeutics.
NCT01218659 is a Phase 3 study of Fabry Disease that has completed, run by Amicus Therapeutics. The registered enrollment target is 68 participants, below the 541-participant average among 32 other Fabry Disease trials with a reported enrollment target (87% lower). The trial reports 20 study locations across 11 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01218659, a Phase 3 study of Fabry Disease, has completed, sponsored by Amicus Therapeutics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 68 participants
- Enrollment target
- 20
- Study locations
Study Summary
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.
Primary Outcome
To assess renal function, measured glomerular filtration rate (GFR) was measured by the plasma clearance of unlabeled iohexol (mGFR-iohexol), a non-ionic contrast agent. The annualized rate of change in mGFR-iohexol from Baseline to Month 18 was analyzed using an analysis of covariance (ANCOVA) model with the following factors as covariates: treatment group, sex, age, Baseline GFR (mGFR-iohexol), and Baseline 24-hour (hr) urine protein. A threshold of \<2.2 milliliter (mL)/minute (min)/1.73 mete
Conditions Studied
Interventions
- DRUG migalastat hydrochloride
- BIOLOGICAL agalsidase
Study Locations (20)
Other
- - Vienna
- - Edegem
- - São Paulo
- - Copenhagen
- - Garches
- - Lille
- - Florence
- - Niigata
- - Osaka
- - Osaka
California
- - Los Angeles
Colorado
- - Aurora
Georgia
- - Decatur
Michigan
- - Grand Rapids
Oregon
- - Portland
Pennsylvania
- - Pittsburgh
Virginia
- - Fairfax
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 68 participants |
| Start Date | 2011-09-08 |
| Est. Completion | 2015-05-28 |
| Phase | Phase 3 |
What the finished NCT01218659 record still lists
NCT01218659 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 68 participants, a relatively small participant target, below the 541-participant average among 32 other Fabry Disease trials with a reported enrollment target (87% lower).
The record links to 1 condition, with Fabry Disease appearing as the primary indexed condition, and to 2 interventions - of which migalastat hydrochloride is the first listed.
NCT01218659 names 20 study sites across 11 states, led by Other, California, Colorado.
Frequently Asked Questions
What is clinical trial NCT01218659 about?
NCT01218659 is a clinical study titled "Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease". 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 em...
What is the current status of trial NCT01218659?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 68 participants. The study started on 2011-09-08. Estimated completion is 2015-05-28.
What conditions does trial NCT01218659 study?
This clinical trial studies the following conditions: Fabry Disease.
What interventions are being tested in trial NCT01218659?
The interventions under investigation include: migalastat hydrochloride (DRUG), agalsidase (BIOLOGICAL).
Who is sponsoring clinical trial NCT01218659?
This trial is sponsored by Amicus Therapeutics, which has 34 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01218659 being conducted?
This trial has 20 study locations across California, Colorado, Georgia, Michigan, Oregon. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Fabry Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT01218659's enrollment target sits among peer trials
68 11th of 32 higher than 22 of 32 other Fabry Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Fabry Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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