Adults 16 to 74, any sex, with Fabry Disease. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
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) InclusionsPrimary· Baseline, Month 6
Renal biopsies were taken at Baseline and Month 6 (Stage 1). The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed th
Group
Value
95% CI
Migalastat
13
Placebo
9
Migalastat
19
Placebo
23
Percent Change In Kidney IC GL-3 Inclusions From Baseline To Month 6Secondary· Baseline, Month 6
Renal biopsies were taken at Baseline and Month 6. The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded
Group
Value
95% CI
Migalastat
-7.948
± 105.2736
Placebo
12.985
± 90.5131
Change From Baseline Through Month 24 In Urine GL-3 LevelsSecondary· Baseline, Months 6, 12, and 24
The effect of migalastat versus placebo on urine GL-3 levels was measured by liquid chromatography-mass spectrometry/mass spectrometry. The 24-hour urine samples were collected at Baseline, Month 6 (Stage 1), Month 12 (Stage 2), and Month 24 (OLE). Results are presented as changes in nanograms (ng)/mg creatinine from Baseline to the end of the 3 stages.
Stage 1
Group
Value
95% CI
Migalastat-Migalastat
-234.80
± 853.451
Placebo-Migalastat
-186.24
± 957.119
Stage 2
Group
Value
95% CI
Migalastat-Migalastat
-179.63
± 699.157
Placebo-Migalastat
-537.95
± 1169.883
OLE
Group
Value
95% CI
Migalastat-Migalastat
-62.37
± 615.944
Placebo-Migalastat
-177.42
± 288.224
Change From Month 6 To Month 12 In Average Number Of Kidney IC GL-3 InclusionsSecondary· Month 6, Month 12
Renal biopsies were taken at Month 6 and Month 12. The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded
Group
Value
95% CI
Placebo-Migalastat
-0.320
-0.5719 – -0.0677
Change From Baseline To Month 6 In Average Number Of Kidney IC GL-3 InclusionsSecondary· Baseline, Month 6
Renal biopsies were taken at Baseline and Month 6. The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded
Group
Value
95% CI
Migalastat
-0.250
± 0.5126
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse events (AEs) were monitored up to 25 months (±7 days) after the first dose of study drug. AEs were reported from the first dose of study medication at Baseline (Visit 1) in Stage 1 to 1 month (±7 days) after the date of the last study visit, whether during Stage 1, Stage 2, or the optional OLE, or after premature discontinuation from the study..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Migalastat (0-6 Months)
Serious: 2/34 (6%)
Deaths: —
Placebo (0-6 Months)
Serious: 4/33 (12%)
Deaths: —
All Migalastat (>6-12 Months)
Serious: 5/63 (8%)
Deaths: —
All Migalastat (>12-24 Months)
Serious: 11/57 (19%)
Deaths: —
Serious adverse events (25 terms)
Reaction
System
Migalastat (0-6 Months)
Placebo (0-6 Months)
All Migalastat (>6-12 Mont…
All Migalastat (>12-24 Mon…
Pulmonary Embolism
Respiratory, thoracic and mediastinal disorders
—
—
—
—
Deep Vein Thrombosis
Vascular disorders
—
—
—
—
Multiple Fractures
Injury, poisoning and procedural complications
—
—
—
—
Post Procedural Haematoma
Injury, poisoning and procedural complications
—
—
—
—
Post Procedural Haemorrhage
Injury, poisoning and procedural complications
—
—
—
—
Anaplastic Large Cell Lymphoma T - and Null - Cell Types
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
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).
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT01458119 — Open-Label Phase 3 Long-Term Safety Study of Migalastat
· Phase 3
· terminated
NCT01218659 — Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
· Phase 3
· completed
Other recruiting trials for Fabry Disease
Currently open trials in the same condition.
NCT07187440 — A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease
· recruiting
NCT06776419 — the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease
· recruiting
NCT06539624 — Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease
· NA
· recruiting
NCT07277361 — Study of the Quality of Life of Patients With Fabry Disease Aged 65 and Over With and Without Specific Treatment
· recruiting
NCT06270316 — Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
· Phase 1, PHASE2
· recruiting
Other Amicus Therapeutics trials
Trials by the same sponsor.
NCT06121011 — A Global Prospective Observational Registry of Patients With Pompe Disease
· recruiting
NCT04804566 — Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
· completed
NCT04020055 — A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
· Phase 3
· active not recruiting
NCT04281537 — A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme R
· completed
NCT04138277 — A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LO
· Phase 3
· completed
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Amicus Therapeutics
Last refreshed: 30 October 2018
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00925301.