Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02194985
• Date of registration: 17/07/2014
• Prospective Registration: Yes
• Primary sponsor: Amicus Therapeutics
• Public title: Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
• Scientific title: An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease
• Date of first enrolment: October 2014
• Target sample size: 76
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02194985
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Austria, Belgium, Brazil, Canada, Denmark, Egypt, France, Italy, Japan, Spain, Turkey, United Kingdom, United States

Contacts

• Name: Medical Monitor, Clinical Research, MD
• Affiliation: Amicus Therapeutics, Inc.

Key inclusion & exclusion criteria

Inclusion Criteria:

- Subject had completed treatment in a previous study of migalastat HCl

- Age 18 years or older

- Male and female participant agrees to use protocol identified acceptable contraception

- Subject is willing to provide written informed consent and authorization for use and
disclosure of Personal Health Information (PHI)

Exclusion Criteria:

- Subjects last available estimated glomerular filtration rate (eGFR) in the previous
study was <30 mL/min/1.73m2; unless there is measured GFR available within 3 months of
Baseline Visit (Visit 1), which is >30 mL/min/1.73m2

- Subject has undergone, or is scheduled to undergo kidney transplantation or is
currently on dialysis

- Subject has a documented transient ischemic attack, stroke, unstable angina, or
myocardial infarction within the 3 months before Visit 1

- Subject has clinically significant unstable cardiac disease in the opinion of the
investigator (e.g., cardiac disease requiring active management, such as symptomatic
arrhythmia, unstable angina, or NYHA class III or IV congestive heart failure)

- Subject has a history of allergy or sensitivity to AT1001 (including excipients) or
other iminosugars (e.g., miglustat, miglitol)

- Subject requires treatment with Glyset® (miglitol) or Zavesca® (miglustat)

- Subjects with severe or unsuitable concomitant medical condition

- Subjects with clinically significant abnormal laboratory value(s) and clinically
significant electrocardiogram (ECG) findings at baseline



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: migalastat HCl 150 mg

Primary Outcome(s)

Change from baseline in laboratory parameters [Time Frame: up to 5 years]

Change from baseline in echocardiography [Time Frame: up to 5 years]

Incidence of adverse events (AEs) [Time Frame: up to 5 years]

Vital signs and body weight - change in baseline in vital signs and body weight [Time Frame: up to 5 years]

Change from baseline in electrocardiogram (ECG) [Time Frame: up to 5 years]

Withdrawal due to AEs [Time Frame: up to 5 years]

Secondary Outcome(s)

Estimated glomerular filtration rate (eGFR) [Time Frame: up to 5 years]

Measurement of left ventricular mass (LVM) and left ventricular mass index (LVMi) [Time Frame: up to 5 years]

Measurement of LV internal dimension (LVIDd and LVIDs) and wall thickness [Time Frame: up to 5 years]

Measurement of 24-hour urine protein [Time Frame: up to 5 years]

Measurement of white blood cell (WBC) a-Gal A activity [Time Frame: up to 5 years]

Ejection fraction [Time Frame: up to 5 years]

Fractional shortening [Time Frame: up to 5 years]

Measurement of subject reported Quality of Life [Time Frame: up to 5 years]

Plasma lyso-Gb3 [Time Frame: up to 5 years]

Secondary ID(s)

AT1001-042

Source(s) of Monetary Support

Please refer to primary and secondary sponsors