Drug-Drug Interaction Study Between AT1001 and Agalsidase in Subjects With Fabry Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics
ClinicalTrials.gov Identifier:
NCT01196871
First received: September 7, 2010
Last updated: December 19, 2013
Last verified: December 2013
  Purpose

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.


Condition Intervention Phase
Fabry Disease
Drug: AT1001
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-label Phase 2A Study to Investigate Drug-Drug Interactions Between AT1001 and Agalsidase in Subjects With Fabry Disease.

Resource links provided by NLM:


Further study details as provided by Amicus Therapeutics:

Primary Outcome Measures:
  • Plasma pharmacokinetics [ Time Frame: Over 24-hour period after dosing ] [ Designated as safety issue: Yes ]

Enrollment: 21
Study Start Date: February 2011
Study Completion Date: October 2012
Primary Completion Date: June 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Low dose A
Lower dose of AT1001 with Fabrazyme.
Drug: AT1001
Capsules, single dose
Other Name: migalastat hydrochloride
Experimental: High dose A
Higher dose of AT1001 with Fabrazyme.
Drug: AT1001
Capsules, single dose
Other Name: migalastat hydrochloride
Experimental: Low dose B
Low dose AT1001 with Replagal
Drug: AT1001
Capsules, single dose
Other Name: migalastat hydrochloride
Experimental: High dose B
Higher dose of AT1001 with Replagal.
Drug: AT1001
Capsules, single dose
Other Name: migalastat hydrochloride

  Eligibility

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

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01196871

Locations
United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294
United States, Georgia
Emory University
Decatur, Georgia, United States, 30033
United States, Iowa
University of Iowa Children's Hospital
Iowa City, Iowa, United States, 52242
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Virginia
O & O Alpan LLC
Springfield, Virginia, United States, 22152
Australia, Victoria
Royal Melbourne Hospital
Parkville, Victoria, Australia, 3050
Australia, Western Australia
Linear Clinical Research
Perth, Western Australia, Australia, 6009
Belgium
Universitair Ziekenhuis Antwerpen
Edegem, Belgium, 2650
Canada, Quebec
Hopital du Sacre-Coeur
Montreal, Quebec, Canada, H4J1C5
Netherlands
Academisch Medisch Centrum (AMC)
Amsterdam, Netherlands, 1105AZ
Sponsors and Collaborators
Amicus Therapeutics
Investigators
Study Director: Medical Monitor Clinical Research Amicus Therapeutics
  More Information

No publications provided

Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT01196871     History of Changes
Other Study ID Numbers: AT1001-013
Study First Received: September 7, 2010
Last Updated: December 19, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Amicus Therapeutics:
Fabry disease
drug drug interaction

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
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
1-Deoxynojirimycin
Antiviral Agents
Anti-Infective Agents
Therapeutic Uses
Pharmacologic Actions
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on September 14, 2014