Adults 18 to 55, male only, 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.
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)Primary· Day 1 (after dosing) through Week 96
TEAEs were defined as any adverse event with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe adverse event was defined as an adverse event that was incapacitating and required medical intervention. The number of participants who experienced one or more severe TEAEs after dosing on Day 1 through Week 96 is presented. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
Group
Value
95% CI
Migalastat
2
PK: Area Under The Concentration Versus Time Curve (AUC) After Administration Of MigalastatSecondary· 0 (predose), 0.5, 1, 2, 3, 4, 5, 6, 8, and 10 hr (postdose)
The AUC from time zero to 12 hours (hr) postdose (AUC0-12) was evaluated in plasma following a single dose of migalastat 25, 100, and 250 mg on Days 1, 15, and 29, respectively. In addition, AUC0-12 was assessed following multiple doses (14 days) of migalastat 25, 100, and 250 mg on Days 14, 28, and 42, respectively.
AUC0-12: Single Dose
Group
Value
95% CI
Migalastat 25 mg
1052.96
± 29.9
Migalastat 100 mg
4217.95
± 32.0
Migalastat 250 mg
10880.66
± 32.4
AUC0-12: Multiple Dose
Group
Value
95% CI
Migalastat 25 mg
1360.69
± 32.9
Migalastat 100 mg
5643.50
± 25.3
Migalastat 250 mg
12244.47
± 26.0
α-Galactosidase A (α-Gal A) Activity In Leukocytes At Baseline, Week 12, And Week 96Secondary· Baseline, Week 12 (end of treatment period), Week 96 (end of extension period)
Leukocytes were isolated from whole blood and lysed, and α-Gal A activity was measured using a validated fluorometric assay, with catalysis to fluorescent 4-methylumbelliferone (4-MU) as the activity measure. The activity values obtained were normalized to protein (measured using a colorimetric assay) and reported as enzyme activity (nanomole \[nmol\] 4-MU/hr) per mg of protein. On Day 1 of the first visit and at every visit thereafter, the samples were collected prior to dosing with migalastat. α-Gal A activity in leukocytes are presented by individual participants.
Participant 1: Baseline
Group
Value
95% CI
Migalastat
5.2
Participant 1: Week 12
Group
Value
95% CI
Migalastat
17.4
Participant 1: Week 96
Group
Value
95% CI
Migalastat
20.3
Participant 2: Baseline
Group
Value
95% CI
Migalastat
4.7
Participant 2: Week 12
Group
Value
95% CI
Migalastat
24.6
Participant 2: Week 96
Group
Value
95% CI
Migalastat
22.8
Participant 3: Baseline
Group
Value
95% CI
Migalastat
6.6
Participant 3: Week 12
Group
Value
95% CI
Migalastat
24.6
Adverse events — posted to ClinicalTrials.gov
Time frame: Day 1 after dosing through Week 96 (end of extension period).
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
Publications & conference data
6 peer-reviewed publications reference this trial (live from Europe PMC):
NCT00526071 — Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Compl
· Phase 2
· terminated
NCT00304512 — A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Diseas
· Phase 2
· completed
NCT00283959 — A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
· Phase 2
· completed
NCT00283933 — A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
· Phase 2
· 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/NCT00214500.