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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

January 27, 2006
August 17, 2010
March 2006
May 2008   (final data collection date for primary outcome measure)
Primary: Safety and tolerability.
Same as current
Complete list of historical versions of study NCT00283959 on ClinicalTrials.gov Archive Site
Secondary: Pharmacodynamic and functional (cardiac, CNS/PNS, and renal) parameters.
Same as current
Not Provided
Not Provided
 
A 12-Week Safety and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease
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

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.

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.

Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Fabry Disease
Drug: AT1001 (migalastat hydrochloride)
Not Provided
Germain DP, Giugliani R, Hughes DA, Mehta A, Nicholls K, Barisoni L, Jennette CJ, Bragat A, Castelli J, Sitaraman S, Lockhart DJ, Boudes PF. Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies. Orphanet J Rare Dis. 2012 Nov 24;7:91. doi: 10.1186/1750-1172-7-91.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
8
May 2008
May 2008   (final data collection date for primary outcome measure)

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
Male
18 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
Australia,   Brazil
 
NCT00283959
AA1565521 (FAB-CL-202)
Not Provided
Not Provided
Amicus Therapeutics
Not Provided
Principal Investigator: Roberto Giugliani, MD, PhD Serviço de Genética Médica, Hospital de Clínicas de Porto Alegre
Amicus Therapeutics
August 2010

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