Drug-Drug Interaction Study Between AT1001 and Agalsidase in Subjects 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: NCT01196871
Recruitment Status : Completed
First Posted : September 9, 2010
Last Update Posted : January 27, 2014
Information provided by (Responsible Party):
Amicus Therapeutics

September 7, 2010
September 9, 2010
January 27, 2014
February 2011
June 2012   (Final data collection date for primary outcome measure)
Plasma pharmacokinetics [ Time Frame: Over 24-hour period after dosing ]
Same as current
Complete list of historical versions of study NCT01196871 on Archive Site
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Drug-Drug Interaction Study Between AT1001 and Agalsidase in Subjects With Fabry Disease
An Open-label Phase 2A Study to Investigate Drug-Drug Interactions Between AT1001 and Agalsidase in Subjects With Fabry Disease.
The purpose of this study is to determine the effects of AT1001 on the safety of agalsidase, and the effects of agalsidase on the safety of AT1001.
Not Provided
Phase 2
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Fabry Disease
Drug: AT1001
Capsules, single dose
Other Name: migalastat hydrochloride
  • Experimental: Low dose A
    Lower dose of AT1001 with Fabrazyme.
    Intervention: Drug: AT1001
  • Experimental: High dose A
    Higher dose of AT1001 with Fabrazyme.
    Intervention: Drug: AT1001
  • Experimental: Low dose B
    Low dose AT1001 with Replagal
    Intervention: Drug: AT1001
  • Experimental: High dose B
    Higher dose of AT1001 with Replagal.
    Intervention: Drug: AT1001
Warnock DG, Bichet DG, Holida M, Goker-Alpan O, Nicholls K, Thomas M, Eyskens F, Shankar S, Adera M, Sitaraman S, Khanna R, Flanagan JJ, Wustman BA, Barth J, Barlow C, Valenzano KJ, Lockhart DJ, Boudes P, Johnson FK. Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase. PLoS One. 2015 Aug 7;10(8):e0134341. doi: 10.1371/journal.pone.0134341. eCollection 2015.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
October 2012
June 2012   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Male diagnosed with Fabry disease and between 18 and 65 years of age, inclusive
  • Body mass index between 18-35
  • Subject initiated treatment with agalsidase at lease one month, having received at least two infusions, before the Screening Visit
  • Subject's dose level, dosing regimen and form of agalsidase have been stable for at least one month before Screening Visit
  • Subject has an estimated creatinine clearance greater than or equal to 50mL/min at Screening
  • Subject agrees to use medically-accepted methods of contraception during the study and for 30 days after study completion
  • Subject is willing and able to provide written informed consent

Exclusion Criteria:

  • Subject has had a documented transient ischemic attack, ischemic stroke, unstable angina, or myocardial infarction within the 3 months before Screening
  • Subject has clinically significant unstable cardiac disease (e.g., cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or NYHA class III or IV congestive heart failure)
  • Subject has a history of allergy or sensitivity to study drug (including excipients) or other iminosugars (e.g., miglustat, miglitol)
  • Subject requires a concomitant medication prohibited by the protocol: Glyset® (miglitol), or Zavesca® (miglustat)
  • Any investigational/experimental drug or device within 30 days of Screening, except for use of investigational ERT for Fabry Disease
  • Subject has any intercurrent illness or condition that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study
Sexes Eligible for Study: Male
18 Years to 65 Years   (Adult)
Contact information is only displayed when the study is recruiting subjects
Australia,   Belgium,   Canada,   Netherlands,   United States
France,   United Kingdom
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Amicus Therapeutics
Amicus Therapeutics
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Study Director: Medical Monitor Clinical Research Amicus Therapeutics
Amicus Therapeutics
December 2013

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