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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02194985




Registration number
NCT02194985
Ethics application status
Date submitted
17/07/2014
Date registered
21/07/2014
Date last updated
21/12/2020

Titles & IDs
Public title
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
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
2014-002701-38
Secondary ID [2] 0 0
AT1001-042
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(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - migalastat HCl 150 mg

Experimental: Migalastat HCl 150 mg - Migalastat HCl 150 milligram (mg).


Treatment: Drugs: migalastat HCl 150 mg
Migalastat HCl 150 mg (equivalent to 123 mg migalastat) was provided as capsules in blister packs. One capsule was taken orally every other day.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number Of Participants Experiencing Adverse Events (AEs)
Timepoint [1] 0 0
Day 1 after first dose to approximately 30 days after last treatment, median duration of 3.1 years
Secondary outcome [1] 0 0
Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR)
Timepoint [1] 0 0
Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Secondary outcome [2] 0 0
Change From Baseline In eGFR At End Of Study
Timepoint [2] 0 0
Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Secondary outcome [3] 0 0
Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study
Timepoint [3] 0 0
Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Secondary outcome [4] 0 0
Change From Baseline In White Blood Cell a-Gal A Activity To End Of Study
Timepoint [4] 0 0
Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Secondary outcome [5] 0 0
Change From Baseline In 24-hour Urine Protein To End Of Study
Timepoint [5] 0 0
Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Secondary outcome [6] 0 0
Change From Baseline In Left Ventricular Mass (LVM) To End Of Study
Timepoint [6] 0 0
Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Secondary outcome [7] 0 0
Change From Baseline In Left Ventricular Mass Index (LVMi) To End Of Study
Timepoint [7] 0 0
Baseline to approximately 30 days after last treatment, median duration of 3.1 years
Secondary outcome [8] 0 0
Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire
Timepoint [8] 0 0
Baseline to approximately 30 days after last treatment, median duration of 3.1 years

Eligibility
Key inclusion criteria
* Participant had completed treatment in a previous study of migalastat HCl given as a monotherapy
* Male and female participant agreed to use protocol-identified acceptable contraception
* Participant was willing to provide written informed consent and authorization for use and disclosure of Personal Health Information (PHI)
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Participant's last available estimated glomerular filtration rate (eGFR) in the previous study was <30 milliliter (mL)/minute (min)/1.73 meters squared (m^2); unless there was measured GFR available within 3 months of Baseline Visit, which was >30 mL/min/1.73 m^2
* Participant had undergone, or was scheduled to undergo kidney transplantation or was currently on dialysis
* Participant had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before Baseline Visit
* Participant had clinically significant unstable cardiac disease in the opinion of the investigator (for example, cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or New York Heart Association class III or IV congestive heart failure)
* Participant had a history of allergy or sensitivity to AT1001 (including excipients) or other iminosugars (for example, miglustat, miglitol)
* Participant required treatment with Glyset® (miglitol) or Zavesca® (miglustat)
* Participants with severe or unsuitable concomitant medical condition
* Participants with clinically significant abnormal laboratory value(s) and/or clinically significant electrocardiogram (ECG) findings

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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
14/03/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
23/10/2019
Sample size
Target
Accrual to date
Final
84
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Clinical Study Site - Adelaide
Recruitment hospital [2] 0 0
Clinical Study Site - 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
Kansas
Country [3] 0 0
United States of America
State/province [3] 0 0
Michigan
Country [4] 0 0
United States of America
State/province [4] 0 0
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
Oregon
Country [6] 0 0
United States of America
State/province [6] 0 0
Texas
Country [7] 0 0
United States of America
State/province [7] 0 0
Virginia
Country [8] 0 0
Argentina
State/province [8] 0 0
Pilar
Country [9] 0 0
Austria
State/province [9] 0 0
Wien
Country [10] 0 0
Belgium
State/province [10] 0 0
Edegem
Country [11] 0 0
Brazil
State/province [11] 0 0
Porto Alegre
Country [12] 0 0
Canada
State/province [12] 0 0
Quebec
Country [13] 0 0
Denmark
State/province [13] 0 0
Copenhagen
Country [14] 0 0
Egypt
State/province [14] 0 0
Cairo
Country [15] 0 0
France
State/province [15] 0 0
Garches
Country [16] 0 0
France
State/province [16] 0 0
Lille
Country [17] 0 0
Italy
State/province [17] 0 0
Firenze
Country [18] 0 0
Italy
State/province [18] 0 0
Roma
Country [19] 0 0
Japan
State/province [19] 0 0
Osaka
Country [20] 0 0
Japan
State/province [20] 0 0
Niigata
Country [21] 0 0
Japan
State/province [21] 0 0
Osaka-shi
Country [22] 0 0
Japan
State/province [22] 0 0
Tokyo
Country [23] 0 0
Spain
State/province [23] 0 0
Barcelona
Country [24] 0 0
Turkey
State/province [24] 0 0
Ankara
Country [25] 0 0
United Kingdom
State/province [25] 0 0
London

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?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT02194985