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

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
GlaxoSmithKline
Information provided by (Responsible Party):
Amicus Therapeutics
ClinicalTrials.gov Identifier:
NCT01218659
First received: October 6, 2010
Last updated: October 17, 2012
Last verified: October 2012
History of Changes
  Purpose

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 Intervention Phase
Fabry Disease
 Drug: migalastat hydrochloride
Biological: agalsidase
 Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: 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

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Amicus Therapeutics:

Primary Outcome Measures:
  • renal function assessed by iohexol Glomerular Filtration Rate (GFR) [ Time Frame: 18 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • renal function (assessed by estimated GFR and 24-hour urine protein) [ Time Frame: 18 months ] [ Designated as safety issue: No ]
  • composite clinical outcome (assessed by time to occurrence of renal, cardiac, cerebrovascular events or death) [ Time Frame: 18 months ] [ Designated as safety issue: No ]
  • cardiac function (assessed by echocardiography) [ Time Frame: 18 months ] [ Designated as safety issue: No ]
  • patient reported outcomes (pain and quality of life) [ Time Frame: 18 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 50
Study Start Date: December 2010
Estimated Study Completion Date: August 2015
Estimated Primary Completion Date: August 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   16 Years to 74 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01218659

  Show 40 Study Locations
Sponsors and Collaborators
Amicus Therapeutics
GlaxoSmithKline
Investigators
Study Director: Medical Monitor Clinical Research Amicus Therapeutics
  More Information

No publications provided

Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT01218659     History of Changes
Other Study ID Numbers: AT1001-012
Study First Received: October 6, 2010
Last Updated: October 17, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Amicus Therapeutics:
Fabry Disease

Additional relevant MeSH terms:
Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on June 17, 2013