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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01458119




Registration number
NCT01458119
Ethics application status
Date submitted
20/10/2011
Date registered
24/10/2011
Date last updated
2/10/2018

Titles & IDs
Public title
Open-Label Phase 3 Long-Term Safety Study of Migalastat
Scientific title
An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease
Secondary ID [1] 0 0
2011-004800-40
Secondary ID [2] 0 0
AT1001-041
Universal Trial Number (UTN)
Trial acronym
AT1001-041
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 hydrochloride

Experimental: Migalastat - Migalastat 150-mg capsule taken orally QOD. The median duration of exposure was 23.5 m.


Treatment: Drugs: migalastat hydrochloride
Oral capsule QOD
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number Of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
Baseline to End of Follow-up (30 days after the end of this 42-month study), with AE reporting occurring at each study visit, which occurred once every 6 months.
Secondary outcome [1] 0 0
Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR)
Timepoint [1] 0 0
Baseline, Every 6 m until the End of Study (42 m)

Eligibility
Key inclusion criteria
* Completed migalastat treatment in a previous Fabry disease protocol
* Both male and female participants were enrolled
* Age 16 years or older
* Male and female participants had to agree to use protocol-identified acceptable contraception
Minimum age
16 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Estimated glomerular filtration rate (eGFR) in the previous study was <30 milliliters/minute/1.73 square meters (mL/min/1.73 m^2) unless there was a measured GFR available within 3 m of the Baseline Visit that was >30 mL/min/1.73 m^2
* Had undergone, or was scheduled to undergo, kidney transplantation or was currently on dialysis
* Pregnant or breast feeding
* Treated with another investigational drug (except migalastat) within 30 days of study start
* Unable to comply with study requirements, or deemed otherwise unsuitable for study entry, in the opinion of the investigator
* Had documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 12 m before the Baseline Visit
* Had clinically significant, unstable cardiac disease in the opinion of the investigator
* Had a history of allergy or sensitivity to migalastat (including excipients) or to other iminosugars
* Required treatment with Glyset (miglitol) or Zavesca (miglustat)
* Had any intercurrent illness or condition that may have precluded the participant from fulfilling the protocol requirements
* Had a severe or unsuitable concomitant medical condition
* Had a clinically significant abnormal laboratory value and a clinically significant electrocardiogram finding at the Baseline Visit.

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
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 3
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
14/10/2011
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
17/02/2016
Sample size
Target
Accrual to date
Final
85
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
- Adelaide
Recruitment hospital [2] 0 0
- Parkville
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
3050 - 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
Illinois
Country [3] 0 0
United States of America
State/province [3] 0 0
Kansas
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Michigan
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
Oregon
Country [8] 0 0
United States of America
State/province [8] 0 0
Pennsylvania
Country [9] 0 0
United States of America
State/province [9] 0 0
Texas
Country [10] 0 0
United States of America
State/province [10] 0 0
Virginia
Country [11] 0 0
United States of America
State/province [11] 0 0
Washington
Country [12] 0 0
Argentina
State/province [12] 0 0
Pilar
Country [13] 0 0
Belgium
State/province [13] 0 0
Edegem
Country [14] 0 0
Brazil
State/province [14] 0 0
Porto Alegre
Country [15] 0 0
Canada
State/province [15] 0 0
Montreal
Country [16] 0 0
Denmark
State/province [16] 0 0
Copenhagen
Country [17] 0 0
Egypt
State/province [17] 0 0
Cairo
Country [18] 0 0
France
State/province [18] 0 0
Garches
Country [19] 0 0
Italy
State/province [19] 0 0
Roma
Country [20] 0 0
Spain
State/province [20] 0 0
Barcelona
Country [21] 0 0
Turkey
State/province [21] 0 0
Ankara
Country [22] 0 0
United Kingdom
State/province [22] 0 0
London
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Salford

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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

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 not provided in https://clinicaltrials.gov/study/NCT01458119