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A 12-Week Safety and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease

This study has been completed.
Sponsor:
Information provided by:
Amicus Therapeutics
ClinicalTrials.gov Identifier:
NCT00283959
First received: January 27, 2006
Last updated: August 17, 2010
Last verified: August 2010
History of Changes
  Purpose

The purpose of this study is to collect information on the safety of AT1001 (migalastat hydrochloride) and how AT1001 works in patients with Fabry disease.


Condition Intervention Phase
Fabry Disease
 Drug: AT1001 (migalastat hydrochloride)
 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 2, Open-Label, Single Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients With Fabry Disease

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Amicus Therapeutics:

Primary Outcome Measures:
  • Primary: Safety and tolerability.

Secondary Outcome Measures:
  • Secondary: Pharmacodynamic and functional (cardiac, CNS/PNS, and renal) parameters.

Estimated Enrollment: 8
Study Start Date: March 2006
Study Completion Date: May 2008
Primary Completion Date: May 2008 (Final data collection date for primary outcome measure)
Detailed Description:

The purpose of this study is to determine the effect of AT1001 given orally to patients with Fabry disease. Patients will visit the clinic 4 weeks prior to dosing to determine their eligibility for the study, and then return for a second visit for baseline and safety assessments, which will include skin, cardiac, and renal biopsies. Patients will receive oral doses of AT1001 for 12 weeks and will visit the clinic 3 more times, once every 4 weeks, for evaluation and tests. Skin, cardiac, and renal biopsies, and functional assessments will be performed at the end of the 12 week period. Patients may be given the opportunity to enter a study extension phase for an additional 36 weeks, which will require three more clinic visits, and a final set of functional tests and biopsies. All study participants will have a final follow up visit 2 weeks after the end of the study.

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Males between 18 and 65 years of age (inclusive)
  • Hemizygous for Fabry disease
  • Have a confirmed diagnosis of Fabry disease with a documented missense gene mutation (individual or familial)
  • Have enhanceable enzyme activity based on in vitro tests
  • Have documented evidence of cardiac and/or renal dysfunction (e.g., abnormal ECG, left ventricular hypertrophy, renal insufficiency)
  • Must be previously untreated by ERT or substrate depletion for Fabry disease and be willing to undergo two kidney, two cardiac, and three skin biopsies
  • Agree to be sexually abstinent or use a condom with spermicide when engaging in sexual activity during the course of the study and for a period of 30 days following their completion of the study
  • Willing and able to sign an informed consent form

Exclusion Criteria:

  • History of significant disease other than Fabry disease
  • History of organ transplant
  • Serum creatinine greater than 1.5 mg/dL on Day -2
  • Screening 12-lead ECG demonstrating QTc > 450 msec prior to dosing
  • Pacemaker or other contraindication for MRI scanning
  • Taking a medication prohibited by the protocol: Fabrazyme (agalsidase beta), Replagal (agalsidase alfa), Glyset (miglitol), Zavesca (miglustat), or any experimental therapy for any indication
  • Participated in a clinical trial in the last 30 days
  • Any other condition, which, in the opinion of the investigator, would jeopardize the safety of the patient or impact the validity of the study results
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00283959

Locations
Australia, Victoria
Royal Melbourne Hospital, Department of Nephrology
Parkville, Victoria, Australia, 3050
Brazil
Serviço de Genética Médica, Hospital de Clínicas de Porto Alegre
Porto Alegre, Brazil
Sponsors and Collaborators
Amicus Therapeutics
Investigators
Principal Investigator: Roberto Giugliani, MD, PhD Serviço de Genética Médica, Hospital de Clínicas de Porto Alegre
  More Information

No publications provided by Amicus Therapeutics

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

ClinicalTrials.gov Identifier: NCT00283959     History of Changes
Other Study ID Numbers: AA1565521 (FAB-CL-202)
Study First Received: January 27, 2006
Last Updated: August 17, 2010
Health Authority: Brazil: National Health Surveillance Agency
Australia: Department of Health and Ageing Therapeutic Goods Administration

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 May 16, 2013