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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT00526071




Registration number
NCT00526071
Ethics application status
Date submitted
5/09/2007
Date registered
6/09/2007
Date last updated
3/10/2018

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
FAB-CL-205
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

Intervention/exposure
Study type
Interventional
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

Outcomes
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])

Eligibility
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
test

- 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
Gender
Both males and females
Can healthy volunteers participate?
No
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
dialysis

- 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
Treatment
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
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
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
Georgia
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
Texas
Country [4] 0 0
Brazil
State/province [4] 0 0
Porto Alegre
Country [5] 0 0
France
State/province [5] 0 0
Garches
Country [6] 0 0
United Kingdom
State/province [6] 0 0
London
Country [7] 0 0
United Kingdom
State/province [7] 0 0
Salford

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

Ethics approval
Ethics application status

Summary
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
https://clinicaltrials.gov/show/NCT00526071
Trial related presentations / publications
Public notes

Contacts
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 https://clinicaltrials.gov/show/NCT00526071