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Trial details imported from

For full trial details, please see the original record at

Registration number
Ethics application status
Date submitted
Date registered
Date last updated

Titles & IDs
Public title
Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study
Scientific title
Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Pharmacodynamics of AT1001 in Patients With Fabry Disease
Secondary ID [1] 0 0
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fabry Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Study type
Description of intervention(s) / exposure
Treatment: Drugs - migalastat HCl

Experimental: Migalastat - Migalastat was administered orally, 150 mg QOD, 250 mg QD for 3 days, 4 days off per week for 2 months, or 500 mg QD for 3 days, 4 days off per week for up to 10 months, depending on the approval date of the protocol amendments at each site. Participants received migalastat for up to 56 months.

Treatment: Drugs: migalastat HCl

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Primary outcome [1] 0 0
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) - A TEAE was defined as any adverse event (AE) with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe AE was defined as an AE that was incapacitating and required medical intervention. The number of participants who experienced one or more severe TEAEs after dosing on Day 1 through End of Study (EOS) or follow-up (for participants who did not enroll in Study AT1001-041) are presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
Timepoint [1] 0 0
Day 1 (after dosing) through EOS (up to 56 months) or follow-up (28 days after EOS)
Secondary outcome [1] 0 0
Absolute Change From Baseline In a-Galactosidase A (a-Gal A) Activity In Leukocytes To Month 42 - The activity of the a-Gal A enzyme was measured in leukocyte lysate by a validated fluorometric assay method, using 4-methylumbelliferone as a reference. The activity values obtained were normalized to protein (measured using a colorimetric assay). Baseline was defined as the last non-missing pre-treatment value for each participant in his or her respective preceding migalastat Phase 2 study. A negative change from Baseline indicates that a-Gal A activity decreased. a-Gal A activity levels are presented for Baseline and Month 42.
Timepoint [1] 0 0
Baseline, Month 42
Secondary outcome [2] 0 0
Pharmacokinetics Of Migalastat As Assessed By Plasma Concentration - The concentration of migalastat was evaluated in plasma following a dose of 250 mg and 500 mg. Blood samples were taken at trough (predose or Time 0; just prior to the third dose during a 3 day on, 4 days off dosing regimen) and at peak (3 hr postdose; after the third dose during a 3 day on, 4 days off dosing regimen) during the Day 1 (250 mg) and Month 2 (500 mg) visits. This outcome presents the lowest and highest concentrations of migalastat measured in any of the participants for predose and 3 hr postdose.
Timepoint [2] 0 0
0 (predose on Day 1; start of DEP), 3 hr (postdose at Month 2; during DEP])

Key inclusion criteria
- Must have completed another Phase 2 study of migalastat in Fabry Disease

- Women of childbearing potential must have had a negative result on their pregnancy

- Male and female participants agreed to use a reliable method of contraception during
study treatment and for 4 weeks after study treatment termination

- Were willing and able to provide written informed consent
Minimum age
18 Years
Maximum age
No limit
Both males and females
Can healthy volunteers participate?
Key exclusion criteria
- Had not completed a Phase 2 study of migalastat in Fabry Disease

- Had a major protocol violation in the preceding migalastat trial and was discontinued

- Had undergone or was scheduled to undergo kidney transplantation or was currently on

- Had been treated with another investigational drug (except migalastat) within 30 days
of study start

- Had been treated with Fabrazyme® (agalsidase beta), Replagalâ„¢ (agalsidase alfa),
Glyset® (miglitol), or Zavesca® (miglustat) within 2 weeks prior to enrollment

Study design
Purpose of the study
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?

Intervention assignment
Single group
Other design features
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment status
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Date of last participant enrolment
Date of last data collection
Sample size
Accrual to date
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
- Parkville
Recruitment postcode(s) [1] 0 0
- Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Country [2] 0 0
United States of America
State/province [2] 0 0
New York
Country [3] 0 0
United States of America
State/province [3] 0 0
Country [4] 0 0
State/province [4] 0 0
Porto Alegre
Country [5] 0 0
State/province [5] 0 0
Country [6] 0 0
United Kingdom
State/province [6] 0 0
Country [7] 0 0
United Kingdom
State/province [7] 0 0

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Amicus Therapeutics

Ethics approval
Ethics application status

Brief summary
Study to evaluate the long-term safety, tolerability, and pharmacodynamics (PD) of migalastat
hydrochloride (HCl) (migalastat) in participants with Fabry disease
Trial website
Trial related presentations / publications
Public notes

Principal investigator
Name 0 0
Medical Monitor, Clinical Research
Address 0 0
Amicus Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see