Trial Review

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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
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
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.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
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
Timepoint [1] 0 0
Baseline, Month 42
Secondary outcome [2] 0 0
Pharmacokinetics Of Migalastat As Assessed By Plasma Concentration
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
Sex
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), Replagal™ (agalsidase alfa), Glyset® (miglitol), or Zavesca® (miglustat) within 2 weeks prior to enrollment

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
17/09/2007
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
8/09/2012
Sample size
Target
Accrual to date
Final
23
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/study/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/study/NCT00526071