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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/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
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Scientific title
Open-label Extension Study to Evaluate the Long-term Safety, Tolerability and Pharmacodynamics of AT1001 in Patients With Fabry Disease
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Secondary ID [1]
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FAB-CL-205
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Fabry Disease
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Metabolic and Endocrine
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Metabolic disorders
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Metabolic and Endocrine
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0
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Other metabolic disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
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.
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
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Assessment method [1]
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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.
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Timepoint [1]
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Day 1 (after dosing) through EOS (up to 56 months) or follow-up (28 days after EOS)
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Secondary outcome [1]
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Absolute Change From Baseline In a-Galactosidase A (a-Gal A) Activity In Leukocytes To Month 42
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline, Month 42
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Secondary outcome [2]
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Pharmacokinetics Of Migalastat As Assessed By Plasma Concentration
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Assessment method [2]
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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.
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Timepoint [2]
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0 (predose on Day 1; start of DEP), 3 hr (postdose at Month 2; during DEP])
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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
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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), Replagal™ (agalsidase alfa), Glyset® (miglitol), or Zavesca® (miglustat) within 2 weeks prior to enrollment
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Stopped early
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
17/09/2007
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
8/09/2012
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Sample size
Target
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Accrual to date
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Final
23
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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- Parkville
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Recruitment postcode(s) [1]
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- Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Georgia
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Country [2]
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United States of America
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State/province [2]
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New York
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Country [3]
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United States of America
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State/province [3]
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Texas
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Country [4]
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Brazil
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State/province [4]
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Porto Alegre
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Country [5]
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France
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State/province [5]
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Garches
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Country [6]
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United Kingdom
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State/province [6]
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London
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Country [7]
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United Kingdom
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State/province [7]
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Salford
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Amicus Therapeutics
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
Study to evaluate the long-term safety, tolerability, and pharmacodynamics (PD) of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease
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Trial website
https://clinicaltrials.gov/study/NCT00526071
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Medical Monitor, Clinical Research
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Address
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Amicus Therapeutics
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT00526071
Download to PDF