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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT02082327
• Date of registration: 06/03/2014
• Prospective Registration: No
• Primary sponsor: Amicus Therapeutics
• Public title: A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers
• Scientific 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
• Date of first enrolment: March 2014
• Target sample size: 31
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT02082327
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

Netherlands

Contacts

• Name: Medical Monitor Clinical Research
• Affiliation: Amicus Therapeutics

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: 45 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: IV placebo

Drug: IV migalastat HCl

Drug: oral migalastat HCl

Primary Outcome(s)

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]

Safety and tolerability of migalastat [Time Frame: 48 hours]

Secondary Outcome(s)

Urinary pharmacokinetics [Time Frame: Pre dose, between 0-6, 6-12 and 12-24 hours after start of infusion]

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]

Secondary ID(s)

AT1001-018

2013-005553-75

Source(s) of Monetary Support

Please refer to primary and secondary sponsors