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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT00925301
Registration number
NCT00925301
Ethics application status
Date submitted
19/06/2009
Date registered
22/06/2009
Date last updated
30/10/2018
Titles & IDs
Public title
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
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Scientific title
A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Pharmacodynamics of AT1001 in Patients With Fabry Disease and AT1001-Responsive GLA Mutations
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Secondary ID [1]
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FACETS
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Secondary ID [2]
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AT1001-011
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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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Other metabolic disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - migalastat hydrochloride
Treatment: Drugs - Placebo
Experimental: Migalastat - Migalastat 150-mg capsule taken orally every other day (QOD) for 6 months and an open-label 6-month treatment extension, followed by an optional, 12-month, open-label treatment extension.
Placebo comparator: Placebo - Placebo capsule taken orally QOD for 6 months.
Treatment: Drugs: migalastat hydrochloride
Oral capsule QOD
Treatment: Drugs: Placebo
Oral capsule QOD
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Intervention code [1]
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Treatment: Drugs
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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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Percentage Of Participants With At Least A 50% Reduction From Baseline To Month 6 In The Average Number Of Kidney Interstitial Capillary (IC) Globotriaosylceramide (GL-3) Inclusions
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Assessment method [1]
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Renal biopsies were taken at Baseline and Month 6 (Stage 1). The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Baseline and Month 6. Assessments were made using digital images. A responder was defined as a participant with a =50% reduction from Baseline to Month 6 in the average number of kidney IC GL-3 inclusions.
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Timepoint [1]
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Baseline, Month 6
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Secondary outcome [1]
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Percent Change In Kidney IC GL-3 Inclusions From Baseline To Month 6
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Assessment method [1]
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Renal biopsies were taken at Baseline and Month 6. The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Baseline and Month 6. Assessments were made using digital images.
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Timepoint [1]
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Baseline, Month 6
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Secondary outcome [2]
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Change From Baseline Through Month 24 In Urine GL-3 Levels
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Assessment method [2]
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The effect of migalastat versus placebo on urine GL-3 levels was measured by liquid chromatography-mass spectrometry/mass spectrometry. The 24-hour urine samples were collected at Baseline, Month 6 (Stage 1), Month 12 (Stage 2), and Month 24 (OLE). Results are presented as changes in nanograms (ng)/mg creatinine from Baseline to the end of the 3 stages.
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Timepoint [2]
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Baseline, Months 6, 12, and 24
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Eligibility
Key inclusion criteria
* Male or female between the ages of 16 and 74 diagnosed with Fabry disease.
* Confirmed mutant form of a-galactosidase A shown to be responsive to migalastat in vitro.
* Participant has never been treated with enzyme replacement therapy (ERT) or has not received ERT for 6 consecutive months or longer before the screening visit for the study.
* Urine GL-3 =4 times the upper limit of normal at screening.
* Participants taking angiotensin converting enzyme inhibitors or angiotensin receptor blockers must be on a stable dose for a minimum of 4 weeks before the baseline visit.
* Females who can become pregnant and all males agree to be sexually abstinent or use medically accepted methods of birth control during the study and for 30 days after study completion.
* Participant is willing and able to provide written informed consent and assent, if applicable.
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Minimum age
16
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Maximum age
74
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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
* Participant has undergone or is scheduled to undergo kidney transplantation, or is currently on dialysis.
* Estimated glomerular filtration rate <30 milliliters per minute per 1.73 meters squared (chronic kidney disease Stage 4 or 5) based on the Modification of Diet in Renal Disease equation at screening.
* Pregnant or breast-feeding.
* History of allergy or sensitivity to study medication (including excipients) or other iminosugars (for example, miglustat, miglitol).
* Participant is treated or has been treated with any investigational drug within 30 days of study start.
* Participant is currently treated or has ever been treated with migalastat.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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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
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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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
23/10/2009
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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
29/01/2014
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Sample size
Target
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Accrual to date
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Final
67
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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- Adelaide
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Recruitment hospital [2]
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- Parkville
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Recruitment postcode(s) [1]
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5006 - Adelaide
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Recruitment postcode(s) [2]
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3050 - Parkville
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Recruitment outside Australia
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United States of America
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California
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United States of America
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Georgia
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Illinois
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Kansas
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Massachusetts
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Michigan
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New York
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Pennsylvania
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Virginia
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Washington
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Argentina
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Buenos Aires
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Brazil
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Porto Alegre
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Brazil
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Sao Paulo
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Canada
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Quebec
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Denmark
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Kobenhavn
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Egypt
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Cairo
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France
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Garches
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Italy
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Roma
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Poland
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Warszawa
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Barcelona
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Spain
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Zaragoza
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Turkey
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Ankara
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United Kingdom
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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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Ethics approval
Ethics application status
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Summary
Brief summary
The primary objective of this study was to compare the effect of migalastat (123 milligrams \[mg\] of migalastat \[equivalent to 150 mg of migalastat hydrochloride\]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).
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Trial website
https://clinicaltrials.gov/study/NCT00925301
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Trial related presentations / publications
Bichet DG, Aerts JM, Auray-Blais C, Maruyama H, Mehta AB, Skuban N, Krusinska E, Schiffmann R. Assessment of plasma lyso-Gb3 for clinical monitoring of treatment response in migalastat-treated patients with Fabry disease. Genet Med. 2021 Jan;23(1):192-201. doi: 10.1038/s41436-020-00968-z. Epub 2020 Sep 30. Erratum In: Genet Med. 2021 Jan;23(1):238. doi: 10.1038/s41436-020-01041-5. Germain DP, Nicholls K, Giugliani R, Bichet DG, Hughes DA, Barisoni LM, Colvin RB, Jennette JC, Skuban N, Castelli JP, Benjamin E, Barth JA, Viereck C. Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study. Genet Med. 2019 Sep;21(9):1987-1997. doi: 10.1038/s41436-019-0451-z. Epub 2019 Feb 6. Schiffmann R, Bichet DG, Jovanovic A, Hughes DA, Giugliani R, Feldt-Rasmussen U, Shankar SP, Barisoni L, Colvin RB, Jennette JC, Holdbrook F, Mulberg A, Castelli JP, Skuban N, Barth JA, Nicholls K. Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial. Orphanet J Rare Dis. 2018 Apr 27;13(1):68. doi: 10.1186/s13023-018-0813-7. Benjamin ER, Della Valle MC, Wu X, Katz E, Pruthi F, Bond S, Bronfin B, Williams H, Yu J, Bichet DG, Germain DP, Giugliani R, Hughes D, Schiffmann R, Wilcox WR, Desnick RJ, Kirk J, Barth J, Barlow C, Valenzano KJ, Castelli J, Lockhart DJ. The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat. Genet Med. 2017 Apr;19(4):430-438. doi: 10.1038/gim.2016.122. Epub 2016 Sep 22. Germain DP, Hughes DA, Nicholls K, Bichet DG, Giugliani R, Wilcox WR, Feliciani C, Shankar SP, Ezgu F, Amartino H, Bratkovic D, Feldt-Rasmussen U, Nedd K, Sharaf El Din U, Lourenco CM, Banikazemi M, Charrow J, Dasouki M, Finegold D, Giraldo P, Goker-Alpan O, Longo N, Scott CR, Torra R, Tuffaha A, Jovanovic A, Waldek S, Packman S, Ludington E, Viereck C, Kirk J, Yu J, Benjamin ER, Johnson F, Lockhart DJ, Skuban N, Castelli J, Barth J, Barlow C, Schiffmann R. Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat. N Engl J Med. 2016 Aug 11;375(6):545-55. doi: 10.1056/NEJMoa1510198.
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Public notes
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Contacts
Principal investigator
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Medical Monitor, Clinical Research
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Address
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Amicus Therapeutics
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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/NCT00925301
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