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A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers

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ClinicalTrials.gov Identifier: NCT02082327
Recruitment Status : Completed
First Posted : March 10, 2014
Last Update Posted : June 18, 2014
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Fabry Disease Drug: IV migalastat HCl Drug: IV placebo Drug: oral migalastat HCl Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 31 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Dose Escalation Trial to Evaluate the Safety, Tolerability and Pharmacokinetics of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers With an Open-Label, Randomized, Two-Way Crossover Arm
Study Start Date : March 2014
Actual Primary Completion Date : June 2014
Actual Study Completion Date : June 2014


Arm Intervention/treatment
Experimental: 0.3 mg/kg
IV infusion of migalastat HCl or placebo
Drug: IV migalastat HCl
Other Name: AT1001

Drug: IV placebo
Other Name: Sodium Chloride 0.9%

Experimental: 1 mg/kg
IV infusion of migalastat HCl or placebo
Drug: IV migalastat HCl
Other Name: AT1001

Drug: IV placebo
Other Name: Sodium Chloride 0.9%

Experimental: 10 mg/kg
IV infusion of migalastat HCl or placebo
Drug: IV migalastat HCl
Other Name: AT1001

Drug: IV placebo
Other Name: Sodium Chloride 0.9%

Experimental: 150 mg IV
150 mg single IV infusion
Drug: IV migalastat HCl
Other Name: AT1001

Drug: oral migalastat HCl
Other Name: AT1001

Experimental: 150 mg oral
150 mg single oral dose
Drug: IV migalastat HCl
Other Name: AT1001

Drug: oral migalastat HCl
Other Name: AT1001




Primary Outcome Measures :
  1. Plasma pharmacokinetics of migalastat [ Time Frame: Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48 ]
    To investigate the effect on the body of migalastat following a single 2 hour IV infusion in healthy subjects.

  2. Safety and tolerability of migalastat [ Time Frame: 48 hours ]
    Adverse events, clinical laboratory test values, vital signs, ECG, physical examinations


Secondary Outcome Measures :
  1. Plasma pharmacokinetics of migalastat [ Time Frame: Pre-dose, 0.25, 0.5, 1, 1.5, 2.0, 2.5, 3, 4, 6, 8, 10, 12, 16, 24, 48 ]
    To assess the dose proportionality of migalastat following a single 2 hour IV infusion

  2. Urinary pharmacokinetics [ Time Frame: Pre dose, between 0-6, 6-12 and 12-24 hours after start of infusion ]
    To estimate the urinary excretion of unchanged migalastat following a single 2 hour IV infusion in healthy subjects



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Ages Eligible for Study:   18 Years to 45 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • Males and females between 18 and 45 years of age.
  • Body weight range ≥ 50 kg ≤ 100 kg and BMI within the range 18.5 - 29.9 kg/m2.
  • Healthy as determined by a responsible and experienced physician, based on a medial evaluation.
  • Male and female subjects of childbearing potential agree to adhere to the contraception requirements.
  • Capable of giving written informed consent.

Exclusion Criteria:

  • History of sensitivity to migalastat HCl or related iminosugars (eg, miglitol, miglustat), or other significant drug allergy.
  • Past medical history, or physical examination findings, of clinically significant abnormalities that may put the subject at risk or interfere with outcome variables.
  • Positive pre-study drug/alcohol screen.
  • Pregnant or lactating females.
  • The subject has participated in a clinical trial and has received an investigational product within 60 days prior to the first dosing day in the current study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02082327


Locations
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Netherlands
PRA International
Groningen, Netherlands, 9713 GZ
Sponsors and Collaborators
Amicus Therapeutics
Investigators
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Study Director: Medical Monitor Clinical Research Amicus Therapeutics
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Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT02082327    
Other Study ID Numbers: AT1001-018
2013-005553-75 ( EudraCT Number )
First Posted: March 10, 2014    Key Record Dates
Last Update Posted: June 18, 2014
Last Verified: April 2014
Keywords provided by Amicus Therapeutics:
Fabry Disease
Fabry
Lysosomal storage disorders
LSD
Amicus
migalastat HCl
migalastat
AT1001
Additional relevant MeSH terms:
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Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
1-Deoxynojirimycin
Antiviral Agents
Anti-Infective Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action