Phase 3
Completed N=67
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
Source: ClinicalTrials.gov NCT00925301 ↗Enrolled (actual)
67
Serious AEs
11.8%
Results posted
Oct 2018
Primary outcomePrimary: Percentage Of Participants With At Least A 50% Reduction From Baseline To Month 6 In The Average Number Of Kidney Interstitial Capillary (IC) Globotriaosylceramide (GL-3) Inclusions — 13; 9; 19; 23 Participants — p=0.2996
◆ Published Evidence
Highly cited
556citations · ~56 / year
Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat.
Summary
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).
Linked Publications (5)
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Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat.
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The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat.
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Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study.
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Assessment of plasma lyso-Gb<sub>3</sub> for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease.
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Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage Of Participants With At Least A 50% Reduction From Baseline To Month 6 In The Average Number Of Kidney Interstitial Capillary (IC) Globotriaosylceramide (GL-3) Inclusions |
13; 9; 19; 23 | 0.2996 |
| SECONDARY Percent Change In Kidney IC GL-3 Inclusions From Baseline To Month 6 |
-7.948; 12.985 | — |
| SECONDARY Change From Baseline Through Month 24 In Urine GL-3 Levels |
-234.80; -186.24; -179.63; -537.95; -62.37; -177.42 | — |
Eligibility Criteria
Inclusion Criteria
- Male or female between the ages of 16 and 74 diagnosed with Fabry disease.
- Confirmed mutant form of α-galactosidase A shown to be responsive to migalastat in vitro.
- Participant has never been treated with enzyme replacement therapy (ERT) or has not received ERT for 6 consecutive months or longer before the screening visit for the study.
- Urine GL-3 ≥4 times the upper limit of normal at screening.
- Participants taking angiotensin converting enzyme inhibitors or angiotensin receptor blockers must be on a stable dose for a minimum of 4 weeks before the baseline visit.
- Females who can become pregnant and all males agree to be sexually abstinent or use medically accepted methods of birth control during the study and for 30 days after study completion.
- Participant is willing and able to provide written informed consent and assent, if applicable.
Exclusion Criteria
- Participant has undergone or is scheduled to undergo kidney transplantation, or is currently on dialysis.
- Estimated glomerular filtration rate <30 milliliters per minute per 1.73 meters squared (chronic kidney disease Stage 4 or 5) based on the Modification of Diet in Renal Disease equation at screening.
- Pregnant or breast-feeding.
- History of allergy or sensitivity to study medication (including excipients) or other iminosugars (for example, miglustat, miglitol).
- Participant is treated or has been treated with any investigational drug within 30 days of study start.
- Participant is currently treated or has ever been treated with migalastat.
Data sourced from ClinicalTrials.gov (NCT00925301) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.