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

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 evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01218659
Recruitment Status : Completed
First Posted : October 11, 2010
Last Update Posted : July 20, 2015
Information provided by (Responsible Party):
Amicus Therapeutics

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Fabry Disease Drug: migalastat hydrochloride Biological: agalsidase Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Study Start Date : December 2010
Actual Primary Completion Date : May 2014
Actual Study Completion Date : May 2015

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Active Comparator: Enzyme Replacement Therapy Biological: agalsidase
intravenous infusion in accordance with the local approved product labeling
Other Names:
  • agalsidase beta; Fabrazyme
  • agalsidase alpha; Replagal

Experimental: AT1001 Drug: migalastat hydrochloride
oral AT1001 capsules and inactive reminder capsules taken every day
Other Name: AT1001

Primary Outcome Measures :
  1. renal function assessed by iohexol Glomerular Filtration Rate (GFR) [ Time Frame: 18 months ]

Secondary Outcome Measures :
  1. renal function (assessed by estimated GFR and 24-hour urine protein) [ Time Frame: 18 months ]
  2. composite clinical outcome (assessed by time to occurrence of renal, cardiac, cerebrovascular events or death) [ Time Frame: 18 months ]
  3. cardiac function (assessed by echocardiography) [ Time Frame: 18 months ]
  4. patient reported outcomes (pain and quality of life) [ Time Frame: 18 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 74 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01218659

  Show 40 Study Locations
Sponsors and Collaborators
Amicus Therapeutics
Study Director: Medical Monitor Clinical Research Amicus Therapeutics

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Amicus Therapeutics Identifier: NCT01218659     History of Changes
Other Study ID Numbers: AT1001-012
First Posted: October 11, 2010    Key Record Dates
Last Update Posted: July 20, 2015
Last Verified: June 2015

Keywords provided by Amicus Therapeutics:
Fabry Disease

Additional relevant MeSH terms:
Fabry Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders