Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01218659
Registration number
NCT01218659
Ethics application status
Date submitted
6/10/2010
Date registered
11/10/2010
Date last updated
1/11/2018
Titles & IDs
Public title
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
Query!
Scientific title
A Randomized, Open-Label Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT
Query!
Secondary ID [1]
0
0
ATTRACT
Query!
Secondary ID [2]
0
0
AT1001-012
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Fabry Disease
0
0
Query!
Condition category
Condition code
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Metabolic and Endocrine
0
0
0
0
Query!
Metabolic disorders
Query!
Metabolic and Endocrine
0
0
0
0
Query!
Other metabolic disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - migalastat hydrochloride
Other interventions - agalsidase
Experimental: Migalastat - Participants received 150 mg migalastat orally QOD during the 18-month randomized treatment period and the optional 12-month OLE period. Participants received an inactive reminder capsule on alternate days during both treatment periods.
Active Comparator: ERT - Participants received ERT (either agalsidase alfa or agalsidase beta) as prescribed by the participant's treating physician and administered in accordance with the approved prescribing information during the 18-month randomized treatment period. Participants were required to be given >80% of the currently labeled dose and regimen during the 18-month randomized treatment period. During the optional 12-month OLE period, participants received 150 mg migalastat orally QOD. Participants received an inactive reminder capsule on alternate days during the OLE.
Treatment: Drugs: migalastat hydrochloride
150-mg capsule administered orally QOD
Other interventions: agalsidase
Agalsidase via intravenous infusion as prescribed by the participant's treating physician and in accordance with the approved prescribing information
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Intervention code [2]
0
0
Other interventions
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Annualized Rate Of Change From Baseline To Month 18 In Measured Glomerular Filtration Rate
Query!
Assessment method [1]
0
0
To assess renal function, measured glomerular filtration rate (GFR) was measured by the plasma clearance of unlabeled iohexol (mGFR-iohexol), a non-ionic contrast agent. The annualized rate of change in mGFR-iohexol from Baseline to Month 18 was analyzed using an analysis of covariance (ANCOVA) model with the following factors as covariates: treatment group, sex, age, Baseline GFR (mGFR-iohexol), and Baseline 24-hour (hr) urine protein. A threshold of <2.2 milliliter (mL)/minute (min)/1.73 meter squared (m^2)/year was established to compare migalastat to ERT. This difference of 2.2 mL/min/1.73 m2/year is based on the smallest expected rate of decline in estimated glomerular filtration rate (eGFR) for participants treated with agalsidase alfa for 18 months.
Query!
Timepoint [1]
0
0
Baseline to Month 18
Query!
Primary outcome [2]
0
0
Annualized Rate Of Change From Baseline To Month 18 In eGFR
Query!
Assessment method [2]
0
0
The eGFR assessed by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation was calculated using the following:
eGFR-CKD-EPI = 141 x min (Serum Creatinine/?,1)^(a) x max(Serum Creatinine/?,1)^(-1.209) x 0.993^(Age) x 1.1018 (if female) x 1.159 (if African American or black) where: ? is 0.7 for females and 0.9 for males; a is -0.329 for females and -0.411 for males; min indicates the minimum of Serum Creatinine/? or 1; max indicates the maximum of Serum Creatinine/? or 1.
The annualized rate of change in eGFR-CKD-EPI from Baseline to Month 18 was analyzed using an ANCOVA model with the following factors as covariates: treatment group, sex, age, Baseline GFR (eGFR-CKD-EPI), and Baseline 24-hr urine protein. A threshold of <2.2 mL/min/1.73m^2/year was established to compare migalastat to ERT. This difference of 2.2 mL/min/1.73 m2/year is based on the smallest expected rate of decline in eGFR for participants treated with agalsidase alfa for 18 months.
Query!
Timepoint [2]
0
0
Baseline to Month 18
Query!
Secondary outcome [1]
0
0
Annualized Rate Of Change From Baseline To Month 18 In eGFR By The Modification Of Diet In Renal Disease Equation
Query!
Assessment method [1]
0
0
The GFR estimated by the Modification Of Diet In Renal Disease equation (eGFR-MDRD) was calculated using the following equation: eGFR-MDRD = 175 x (Serum Creatinine)^(-1.154) x (Age)^(-0.203) x 1.212 (if participant's race is black or African American) x 0.742 (if participant is female).
The eGFR-MDRD from Baseline to Month 18 was analyzed using an ANCOVA model with the following factors as covariates: treatment group, sex, age, Baseline GFR (eGFR-CKD-EPI), and Baseline 24-hr urine protein.
Query!
Timepoint [1]
0
0
Baseline to Month 18
Query!
Eligibility
Key inclusion criteria
- Male or female between the ages of 16 and 74 diagnosed with Fabry disease
- Confirmed a Gal-A mutation that is amenable to migalastat, based on the clinical trial
HEK assay
- Participant has been on ERT for at least 12 months before screening/baseline
- Dose level and regimen of ERT have been stable for 3 months before screening/baseline
and is at least 80% of the currently labeled dose and regimen for this time period
- Glomerular filtration rate (GFR) = 30 milliliter (mL)/minute (min) /1.73 m^2
- Participants taking angiotensin converting enzyme inhibitors or angiotensin receptor
blockers must be on a stable dose for at least 4 weeks before screening/baseline
- Women who can become pregnant and all men agree to be sexually abstinent or use
medically accepted methods of birth control throughout the duration of the study and
for up to 30 days after last dose of study medication
- Participant is willing and able to provide written informed consent and assent if
applicable
Query!
Minimum age
16
Years
Query!
Query!
Maximum age
74
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
- Participant has undergone, or is scheduled to undergo, kidney transplantation or any
other solid organ transplantation
- Participant is on regular dialysis that is specifically for the treatment of chronic
kidney disease
- Participant has had a documented transient ischemic attack, stroke, unstable angina,
or myocardial infarction within the 3 months before screening/baseline
- Participant has 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 (NYHA) class
III or IV congestive heart failure)
- Pregnant or breast-feeding
- History of allergy or sensitivity to study medication (including excipients) or other
iminosugars (for example, miglustat, miglitol)
- Participant has absolute contraindication to iohexol and/or inability to undergo
iohexol GFR testing
- Participant requires treatment with Glyset® (miglitol), or Zavesca® (miglustat)
- Participant received any investigational/experimental drug, biologic or device within
30 days of screening/baseline
- Any intercurrent illness or condition that may preclude the participant from
fulfilling the study requirements or suggests to the investigator that the participant
may have an unacceptable risk by participating in this study
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
8/09/2011
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
28/05/2015
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
68
Query!
Recruitment in Australia
Recruitment state(s)
VIC,West Australi
Query!
Recruitment hospital [1]
0
0
- Parkville
Query!
Recruitment hospital [2]
0
0
- Perth
Query!
Recruitment postcode(s) [1]
0
0
03050 - Parkville
Query!
Recruitment postcode(s) [2]
0
0
6000 - Perth
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
California
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Colorado
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Georgia
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Michigan
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Oregon
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Pennsylvania
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Virginia
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Wisconsin
Query!
Country [9]
0
0
Austria
Query!
State/province [9]
0
0
Vienna
Query!
Country [10]
0
0
Belgium
Query!
State/province [10]
0
0
Edegem
Query!
Country [11]
0
0
Brazil
Query!
State/province [11]
0
0
Sao Paulo
Query!
Country [12]
0
0
Denmark
Query!
State/province [12]
0
0
Copenhagen
Query!
Country [13]
0
0
France
Query!
State/province [13]
0
0
Garches
Query!
Country [14]
0
0
France
Query!
State/province [14]
0
0
Lille
Query!
Country [15]
0
0
Italy
Query!
State/province [15]
0
0
Firenze
Query!
Country [16]
0
0
Japan
Query!
State/province [16]
0
0
Niigata
Query!
Country [17]
0
0
Japan
Query!
State/province [17]
0
0
Osaka
Query!
Country [18]
0
0
Japan
Query!
State/province [18]
0
0
Tokyo
Query!
Country [19]
0
0
United Kingdom
Query!
State/province [19]
0
0
Cambridge
Query!
Country [20]
0
0
United Kingdom
Query!
State/province [20]
0
0
London
Query!
Country [21]
0
0
United Kingdom
Query!
State/province [21]
0
0
Salford
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
Amicus Therapeutics
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
Study to compare the efficacy and safety of migalastat and enzyme replacement therapy (ERT)
in male and female participants with Fabry disease who are currently receiving ERT and who
have an alpha galactosidase-A (a Gal-A) mutation that is amenable to migalastat, based on the
clinical trial human embryonic kidney cell (HEK) assay.
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT01218659
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Medical Monitor, Clinical Research
Query!
Address
0
0
Amicus Therapeutics
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01218659
Download to PDF