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Completed

NCT Number: NCT02082327

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

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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Key information

Conditions

Fabry Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Cardiovascular Diseases Central Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses Vascular Diseases

Age range

18 year–45 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

PRA International

Groningen, 9713 GZ, Netherlands

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

IV migalastat HCl

Drug

Other names: AT1001

IV Placebo

Drug

Other names: Sodium Chloride 0.9%

oral migalastat HCl

Drug

Other names: AT1001

Primary outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

Amicus Therapeutics

Industry

Registry information

Official study 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

Important dates

Study start
2014
Primary completion
2014
Study completion
2014
First posted
Mar 10, 2014
Registry last updated
Jun 18, 2014

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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