Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
NCT ID: NCT00925301
Last Updated: 2018-10-30
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
67 participants
INTERVENTIONAL
2009-10-23
2014-01-29
Brief Summary
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Detailed Description
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Stage 1 included a screening period of up to 2 months followed by a 6-month treatment period which involved 4 visits to the clinic. Participants were randomized in equal proportions to receive either migalastat or placebo.
After completing the 6-month double-blind phase, all participants entered Stage 2 of the study and received migalastat in an open-label manner. Stage 2 treatment lasted for 6 months and involved up to 4 visits to the clinic.
Participants who completed both Stage 1 and Stage 2 of the study as scheduled were offered the opportunity to participate in an open-label treatment extension phase with migalastat. The open-label treatment extension phase lasted 12 months and involved 2 visits to the clinic. A follow-up visit was undertaken 1 month following completion or discontinuation from the open-label treatment extension. Participants completing the 12-month open-label treatment extension and providing consent to enter a separate long-term extension were not required to complete this follow-up visit.
Study assessments included clinical laboratory tests, 12-lead electrocardiogram, kidney biopsy, kidney function testing, echocardiography, and patient-reported outcomes.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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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.
migalastat hydrochloride
Oral capsule QOD
Placebo
Placebo capsule taken orally QOD for 6 months.
Placebo
Oral capsule QOD
Interventions
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migalastat hydrochloride
Oral capsule QOD
Placebo
Oral capsule QOD
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Confirmed mutant form of α-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.
Exclusion Criteria
* 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.
16 Years
74 Years
ALL
No
Sponsors
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Amicus Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Monitor, Clinical Research
Role: STUDY_DIRECTOR
Amicus Therapeutics
Locations
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Los Angeles, California, United States
San Francisco, California, United States
Decatur, Georgia, United States
Chicago, Illinois, United States
Kansas City, Kansas, United States
Boston, Massachusetts, United States
Grand Rapids, Michigan, United States
New York, New York, United States
Pittsburgh, Pennsylvania, United States
Dallas, Texas, United States
Salt Lake City, Utah, United States
Springfield, Virginia, United States
Seattle, Washington, United States
Buenos Aires, , Argentina
Adelaide, , Australia
Parkville, , Australia
Porto Alegre, , Brazil
São Paulo, , Brazil
Montreal, Quebec, Canada
Copenhagen, , Denmark
Cairo, , Egypt
Garches, , France
Roma, , Italy
Warsaw, , Poland
Barcelona, , Spain
Zaragoza, , Spain
Ankara, , Turkey (Türkiye)
Salford, , United Kingdom
Countries
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References
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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.
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.
Other Identifiers
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FACETS
Identifier Type: OTHER
Identifier Source: secondary_id
2009-013459-31
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
AT1001-011
Identifier Type: -
Identifier Source: org_study_id
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