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Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease

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ClinicalTrials.gov Identifier: NCT01218659
Recruitment Status : Completed
First Posted : October 11, 2010
Last Update Posted : July 20, 2015
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

October 6, 2010
October 11, 2010
July 20, 2015
December 2010
May 2014   (Final data collection date for primary outcome measure)
renal function assessed by iohexol Glomerular Filtration Rate (GFR) [ Time Frame: 18 months ]
Same as current
Complete list of historical versions of study NCT01218659 on ClinicalTrials.gov Archive Site
  • renal function (assessed by estimated GFR and 24-hour urine protein) [ Time Frame: 18 months ]
  • composite clinical outcome (assessed by time to occurrence of renal, cardiac, cerebrovascular events or death) [ Time Frame: 18 months ]
  • cardiac function (assessed by echocardiography) [ Time Frame: 18 months ]
  • patient reported outcomes (pain and quality of life) [ Time Frame: 18 months ]
Same as current
Not Provided
Not Provided
 
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
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
Study to compare the efficacy and safety of AT1001 and enzyme replacement therapy (ERT) in male and female patients with Fabry disease who are currently receiving ERT and who have AT1001-responsive GLA mutations.
Not Provided
Interventional
Phase 3
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Fabry Disease
  • Drug: migalastat hydrochloride
    oral AT1001 capsules and inactive reminder capsules taken every day
    Other Name: AT1001
  • Biological: agalsidase
    intravenous infusion in accordance with the local approved product labeling
    Other Names:
    • agalsidase beta; Fabrazyme
    • agalsidase alpha; Replagal
  • Active Comparator: Enzyme Replacement Therapy
    Intervention: Biological: agalsidase
  • Experimental: AT1001
    Intervention: Drug: migalastat hydrochloride
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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
60
Same as current
May 2015
May 2014   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Male or female between the ages of 16 and 74 diagnosed with Fabry disease
  • Confirmed GLA mutation that has shown to be responsive to AT1001 in vitro
  • Subject has been on ERT for at least 12 months before Visit 2
  • Dose level and regimen of ERT have been stable for 3 months before Visit 2 and is at least 80% of the currently labeled dose and regimen for this time period
  • GFR ≥ 30mL/min/1.73 m2
  • Subjects taking angiotensin converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs) must be on a stable dose for at least 4 weeks before Visit 1
  • 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

    * For sites in Italy: Women who can become pregnant and all men, agree to plan (or have their partner plan) with his/her physician a birth control strategy (or method) in order to avoid pregnancy throughout the duration of the study and for up to 30 days after the last dose of study medication

  • Subject is willing and able to provide written informed consent and assent if applicable

Exclusion Criteria:

  • Subject has undergone, or is scheduled to undergo, kidney transplantation or any other solid organ transplantation
  • Subject is on regular dialysis that is specifically for the treatment of chronic kidney disease
  • Subject has had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before Visit 1
  • Subject has clinically significant unstable cardiac disease in the opinion of the investigator (e.g., cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or 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 (e.g., miglustat, miglitol)
  • Subject has absolute contraindication to iohexol and/or inability to undergo iohexol GFR testing
  • Subject requires treatment with Glyset® (miglitol), or Zavesca® (miglustat)
  • Subject received any investigational/experimental drug, biologic or device within 30 days of Visit 1
  • Any intercurrent illness or condition that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the subject may have an unacceptable risk by participating in this study
  • Otherwise unsuitable for the study, in the opinion of the investigator
Sexes Eligible for Study: All
16 Years to 74 Years   (Child, Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
Argentina,   Australia,   Austria,   Belgium,   Brazil,   Denmark,   France,   Germany,   Italy,   Japan,   Switzerland,   Turkey,   United Kingdom,   United States
Greece,   Poland,   Taiwan
 
NCT01218659
AT1001-012
Yes
Not Provided
Not Provided
Amicus Therapeutics
Amicus Therapeutics
Not Provided
Study Director: Medical Monitor Clinical Research Amicus Therapeutics
Amicus Therapeutics
June 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP