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| ID | Type | Description | Link |
|---|---|---|---|
| 2013-005553-75 | EudraCT Number |
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The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of IV migalastat HCl alone and to determine absolute bioavailability for oral migalastat HCl as compared to IV administered migalastat HCl in healthy volunteers. The data from this study will help us understand how migalastat works in the body and will help us determine what would be an effective dose in future studies with migalastat hydrochloride.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 0.3 mg/kg | Experimental | IV infusion of migalastat HCl or placebo |
|
| 1 mg/kg | Experimental | IV infusion of migalastat HCl or placebo |
|
| 10 mg/kg | Experimental | IV infusion of migalastat HCl or placebo |
|
| 150 mg IV | Experimental | 150 mg single IV infusion |
|
| 150 mg oral | Experimental | 150 mg single oral dose |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| IV migalastat HCl | Drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| Plasma pharmacokinetics of migalastat | To investigate the effect on the body of migalastat following a single 2 hour IV infusion in healthy subjects. | Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48 |
| Safety and tolerability of migalastat | Adverse events, clinical laboratory test values, vital signs, ECG, physical examinations | 48 hours |
| Measure | Description | Time Frame |
|---|---|---|
| Plasma pharmacokinetics of migalastat | To assess the dose proportionality of migalastat following a single 2 hour IV infusion | Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48 |
| Urinary pharmacokinetics |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Monitor Clinical Research | Amicus Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| PRA International | Groningen | 9713 GZ | Netherlands |
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| ID | Term |
|---|---|
| D000795 | Fabry Disease |
| D016464 | Lysosomal Storage Diseases |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
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| ID | Term |
|---|---|
| C090092 | migalastat |
| C525167 | larazotide acetate |
| D012965 | Sodium Chloride |
| ID | Term |
|---|---|
| D002712 | Chlorides |
| D006851 | Hydrochloric Acid |
| D017606 | Chlorine Compounds |
| D007287 | Inorganic Chemicals |
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| IV placebo | Drug |
|
|
| oral migalastat HCl | Drug |
|
|
To estimate the urinary excretion of unchanged migalastat following a single 2 hour IV infusion in healthy subjects
| Pre dose, between 0-6, 6-12 and 12-24 hours after start of infusion |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D059345 | Cerebral Small Vessel Diseases |
| D002561 | Cerebrovascular Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008661 | Metabolism, Inborn Errors |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |
| D017670 |
| Sodium Compounds |