About this data
Pipeline and clinical trial information here comes primarily from ClinicalTrials.gov. A listing on ClinicalTrials.gov means a study has been registered — not that a treatment is proven safe, effective, or FDA-approved. Investigational treatments may never be approved. Who can join a trial is decided only by that trial's own study team.
Migalastat HCl
- Company
- Amicus Therapeutics
- Mechanism
- Mechanism not yet reported
- Route
- Route not yet reported
- Recruiting trials
- 3 recruiting
Efficacy results: Not yet reported
3 recruiting studies currently listed on ClinicalTrials.gov.
Data source: ClinicalTrials.gov · trial data as of Oct 23, 2026
Find a Clinical Trial Near Me
See recruiting studies for Migalastat HCl and check eligibility with the study team.
Trial details
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Recruiting NCT NCT04252066
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Amicus Therapeutics 20 participants Est. completion 2030-02 1 siteOther conditions: Fabry DiseaseView official trial listing → -
Recruiting NCT NCT06904261
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Not yet reported Amicus Therapeutics 8 participants Est. completion 2028-12 12 sites Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Recruiting NCT NCT06906367
A Study of Patients With Fabry Disease (US Specific)
Amicus Therapeutics 450 participants Est. completion 2032-06 8 sitesOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT00214500
A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Not yet reported Amicus Therapeutics 9 participants Est. completion 2008-01-29 5 sites Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT00283933
A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Not yet reported Amicus Therapeutics 5 participants Est. completion 2008-03-12 2 sites Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT00283959
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Not yet reported Amicus Therapeutics 4 participants Est. completion 2008-05-08 2 sites Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT00304512
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
Not yet reported Amicus Therapeutics 9 participants Est. completion 2008-05-09 6 sites Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT00362856
Safety and Tolerability Study of Larazotide Acetate in Celiac Disease Subjects
Not yet reported 9 Meters Biopharma, Inc. 80 participants Est. completion 2007-03-06 9 sitesOther conditions: Celiac DiseaseView official trial listing → -
Completed NCT NCT00386165
Safety Study of Larazotide Acetate to Treat Celiac Disease.
Not yet reported 9 Meters Biopharma, Inc. 21 participants Est. completion 2006-01-26 1 siteOther conditions: Celiac DiseaseView official trial listing → -
Completed NCT NCT00386490
Safety of Larazotide Acetate in Healthy Volunteers
Not yet reported 9 Meters Biopharma, Inc. 24 participants Est. completion 2006-02-27 1 siteOther conditions: HealthyView official trial listing → -
Completed NCT NCT00492960
Study to Assess the Efficacy of Larazotide Acetate for the Treatment of Celiac Disease
Not yet reported 9 Meters Biopharma, Inc. 171 participants Est. completion 2009-03 23 sitesOther conditions: Celiac DiseaseView official trial listing → -
Terminated NCT NCT00526071
Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study
Not yet reported Amicus Therapeutics 23 participants Est. completion 2012-09-08 8 sites Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT00620451
Randomized, Double-Blind, Placebo-Controlled Study of Larazotide Acetate in Subjects With Active Celiac Disease
Not yet reported 9 Meters Biopharma, Inc. 105 participants Est. completion 2009-12 33 sitesOther conditions: Celiac DiseaseView official trial listing → -
Completed NCT NCT00889473
Study of the Efficacy of Larazotide Acetate to Treat Celiac Disease
Not yet reported 9 Meters Biopharma, Inc. 42 participants Est. completion 2010-04 15 sitesOther conditions: Celiac DiseaseView official trial listing → -
Completed NCT NCT00925301
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
Not yet reported Amicus Therapeutics 67 participants Est. completion 2014-01-29 28 sitesOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT01196871
Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease
Not yet reported Amicus Therapeutics 20 participants Est. completion 2012-10-09 10 sites Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT01218659
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
Not yet reported Amicus Therapeutics 68 participants Est. completion 2015-05-28 25 sitesOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT01396213
A Double-blind Placebo-controlled Study to Evaluate Larazotide Acetate for the Treatment of Celiac Disease
Not yet reported 9 Meters Biopharma, Inc. 342 participants Est. completion 2013-08-20 62 sitesOther conditions: Celiac DiseaseView official trial listing → -
Terminated NCT NCT01458119
Open-Label Phase 3 Long-Term Safety Study of Migalastat
Not yet reported Amicus Therapeutics 85 participants Est. completion 2016-02-17 25 sitesOther conditions: Fabry DiseaseView official trial listing → -
Available NCT NCT01476163
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
Amicus TherapeuticsOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT01489995
Migalastat Food Effect Study
Not yet reported Amicus Therapeutics 20 participants Est. completion 2011-12 1 site Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT01730469
Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function
Not yet reported Amicus Therapeutics 32 participants Est. completion 2012-04 4 sites Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT01730482
A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)
Not yet reported Amicus Therapeutics 6 participants Est. completion 2011-09 Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT01853852
A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects
Not yet reported Amicus Therapeutics 14 participants Est. completion 2011-12 1 site Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT02082327
A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers
Not yet reported Amicus Therapeutics 31 participants Est. completion 2014-06 1 site Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT02194985
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
Not yet reported Amicus Therapeutics 84 participants Est. completion 2019-10-23 28 sites Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT02930655
A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease
Not yet reported Idorsia Pharmaceuticals Ltd. 14 participants Est. completion 2016-02-01 1 siteOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT02944474
A Study to Assess the Safety, Tolerability and Pharmacokinetics of Lucerastat (CDP923) After Multiple Dosing in Healthy Subjects
Not yet reported Idorsia Pharmaceuticals Ltd. 37 participants Est. completion 2004-05 1 siteOther conditions: Healthy SubjectsView official trial listing → -
Completed NCT NCT02944487
A Study to Assess the Safety and Pharmacokinetics of Lucerastat (OGT 923) in Healthy Subjects
Not yet reported Idorsia Pharmaceuticals Ltd. 39 participants Est. completion 2002-12 1 siteOther conditions: Healthy SubjectsView official trial listing → -
Completed NCT NCT03135197
German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl.
University Hospital Muenster 75 participants Est. completion 2020-06-30 1 siteOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT03380455
Effect of Cimetidine on the Pharmacokinetics of Lucerastat in Healthy Subjects
Not yet reported Idorsia Pharmaceuticals Ltd. 14 participants Est. completion 2018-01-29 1 siteOther conditions: Healthy SubjectsView official trial listing → -
Completed NCT NCT03425539
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
Not yet reported Idorsia Pharmaceuticals Ltd. 118 participants Est. completion 2021-09-02 49 sitesOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT03500094
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
Not yet reported Amicus Therapeutics 22 participants Est. completion 2021-02-06 8 sites Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Status unknown NCT NCT03683966
MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study
Wuerzburg University Hospital 30 participants Est. completion 2023-12-31 1 siteOther conditions: Rare Diseases, Fabry Disease, Adherence, Medication, Quality of LifeView official trial listing → -
Active, not recruiting NCT NCT03737214
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
Not yet reported Idorsia Pharmaceuticals Ltd. 107 participants Est. completion 2029-11 42 sitesOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT03832452
A Study in Healthy Subjects to Investigate Whether Administration of Lucerastat Can Affect Normal Heart Function
Not yet reported Idorsia Pharmaceuticals Ltd. 44 participants Est. completion 2019-04-22 1 siteOther conditions: HealthyView official trial listing → -
Completed NCT NCT03838237
Effect of Migalastat on Cardiac Involvement in Fabry Disease
Ospedale San Donato 18 participants Est. completion 2021-01-22 1 siteOther conditions: Fabry Disease, Heart DiseasesView official trial listing → -
Status unknown NCT NCT03949920
A Study of Migalastat in Fabry Disease
Manchester University NHS Foundation Trust 21 participants Est. completion 2023-12-31 2 sitesOther conditions: Fabry DiseaseView official trial listing → -
Active, not recruiting NCT NCT04020055
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
Not yet reported Amicus Therapeutics 14 participants Est. completion 2026-12-31 12 sites Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT04049760
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
Not yet reported Amicus Therapeutics 16 participants Est. completion 2024-11-29 6 sites Stand-aloneOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT04602364
French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat
Amicus Therapeutics France SAS 48 participants Est. completion 2023-06-30 13 sitesOther conditions: Fabry DiseaseView official trial listing → -
Status unknown NCT NCT04639999
Impact of MIgalastat TheRApy on CaRdiac Function in patiEnts With Fabry's Cardiomyopathy (MIRACRE-Fabry Trial)
Yonsei University 20 participants Est. completion 2025-04 1 siteOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT04804566
Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
Amicus Therapeutics 44 participants Est. completion 2023-11-01 1 siteOther conditions: Fabry DiseaseView official trial listing → -
Terminated NCT NCT05022303
AT1001 for the Treatment of COVID-19 Related MIS-C
Not yet reported Massachusetts General Hospital 12 participants Est. completion 2024-05-15 1 site Stand-aloneOther conditions: Covid19, Multisystem Inflammatory Syndrome in ChildrenView official trial listing → -
Active, not recruiting NCT NCT05280548
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
Not yet reported Sanofi 104 participants Est. completion 2027-12-06 54 sitesOther conditions: Fabry DiseaseView official trial listing → -
Completed NCT NCT05747534
AT1001 for the Treatment of Long COVID
Not yet reported Massachusetts General Hospital 107 participants Est. completion 2026-06-18 1 site Stand-aloneOther conditions: Long COVID, Long COVID-19, Post Acute COVID-19 Syndrome, Post Acute Sequelae of COVID-19View official trial listing → -
Available NCT NCT07762638
Post Trial Access to Lucerastat for Patients With Fabry Disease Who Participated in Study ID-069A302
Idorsia Pharmaceuticals Ltd.Other conditions: Fabry DiseaseView official trial listing → -
Not yet recruiting NCT NCT07778667
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
Not yet reported Idorsia Pharmaceuticals Ltd. 16 participants Est. completion 2029-03Other conditions: Fabry DiseaseView official trial listing →
Efficacy history
Not yet reported.
Safety
Not yet reported.
Sources
Also known as: Migalastat HCl, Migalastat HCl 20 mg, AT1001, Galafold
Related On The Pen coverage
No related coverage yet.
This drug may be investigational and not yet approved for any use. Whether someone can join a clinical trial is determined only by that trial's own study team — On The Pen does not determine eligibility. Information here can change; always confirm current details on the official ClinicalTrials.gov listing or with the sponsor.