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Physician Initiated Request for Migalastat in Individual Patients With Fabry Disease

This study is currently recruiting participants.
Verified February 2013 by GlaxoSmithKline
Sponsor:
Collaborator:
Amicus Therapeutics
Information provided by (Responsible Party):
GlaxoSmithKline
ClinicalTrials.gov Identifier:
NCT01476163
First received: November 17, 2011
Last updated: February 28, 2013
Last verified: February 2013
History of Changes
  Purpose

This program allows physicians to request permission from GlaxoSmithKline (GSK) for treatment access to migalastat hydrochloride (HCl) for specific adult patients with Fabry disease. Treatment is open label for 6 months with renewal every 6 months.


Condition Intervention Phase
Fabry Disease
 Drug: migalastat HCl
 Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Physician Initiated Request for Treatment Use of Migalastat Hydrochloride (GR181413A/AT1001), an Investigational Treatment for Individual Patients With Fabry Disease (MGM116188)

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by GlaxoSmithKline:

Primary Outcome Measures:
  • Incidence of serious adverse events (SAEs), pregnancy [ Time Frame: 12/31/2014 ] [ Designated as safety issue: No ]
    Serious adverse events and reports of pregnancy


Secondary Outcome Measures:
  • Withdrawal from treatment due to adverse events (AEs) [ Time Frame: 12/31/2014 ] [ Designated as safety issue: No ]
    including non-serious and serious AEs


Estimated Enrollment: 20
Study Start Date: December 2011
Estimated Study Completion Date: December 2014
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
migalastat HCI
150 mg capsule taken every other day by mouth. An inactive reminder capsule may be provided to take on the days in between migalastat HCl
 Drug: migalastat HCl
150 mg capsule taken every other day by mouth. An inactive reminder capsule may be provided to take on the days in between migalastat HCl

Detailed Description:

This Physician Initiated Request program allows physicians to request GSK permission to receive migalastat HCL for specific patients with Fabry disease who do not meet requirements for participation in an existing migalastat clinical study. Requesting physicians must have served as an investigator in a migalastat HCl study. Up to 20 patients worldwide may be treated. Patients must meet specific criteria to receive GSK permission for participation. Key criteria for participation include: Adults; Confirmed GLA gene mutation shown to be responsive to migalastat; Have no treatment option because either unsuitable for enzyme replacement therapy (ERT) or unable to access ERT. Requirements for sufficient kidney function. If permission is granted, initial approval is for a 6 month supply of migalastat HCl with renewal every 6 months available upon meeting continued eligibility.

  Eligibility

Ages Eligible for Study:   18 Years to 74 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Confirmed GLA mutation shown to be responsive migalastat in the human embryonic kidney (HEK-293) cell-based assay
  • 18-74 years of age
  • Strong clinical indication for treatment of Fabry disease
  • No other treatment option including either unsuitable for ERT or unable to access ERT
  • Appropriate female and male contraception
  • Willing to receive treatment with migalastat HCl via this program including having signed an authorization for sharing clinical data with GSK

Exclusion Criteria:

  • Estimated glomerular filtration rate (eGFR) or GFR <30 mL/minute
  • On dialysis
  • Has undergone or is scheduled to undergo kidney or other organ transplant
  • Receiving GLYSET® (miglitol), ZAVESCA® (miglustat) or enzyme replacement therapy FABRAZYME® (agalsidase beta) or REPLAGAL™ (agalsidase alpha)
  • Contraindication to migalastat, i.e., sensitivity to other iminosugar such as miglustat, miglitol
  • Treated with another investigational drug within 30 days of start of migalastat HCl treatment
  • Unable to comply with study requirements or deemed otherwise unsuitable for study entry in the opinion of the investigator.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01476163

Contacts
Contact: US GSK Clinical Trials Call Center 877-379-3718 GSKClinicalSupportHD@gsk.com

Locations
Australia, Victoria
GSK Investigational Site Recruiting
Parkville, Victoria, Australia, 3050
Contact: US GSK Clinical Trials Call Center     877-379-3718     GSKClinicalSupportHD@gsk.com    
Contact: EU GSK Clinical Trials Call Center     +44 (0) 20 8990 4466     GSKClinicalSupportHD@gsk.com    
Sponsors and Collaborators
GlaxoSmithKline
Amicus Therapeutics
Investigators
Study Director: GSK Clinical Trials GlaxoSmithKline
  More Information

No publications provided

Responsible Party: GlaxoSmithKline
ClinicalTrials.gov Identifier: NCT01476163     History of Changes
Other Study ID Numbers: 116188
Study First Received: November 17, 2011
Last Updated: February 28, 2013
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration

Keywords provided by GlaxoSmithKline:
GR181413
AT1001
Lysosomal storage disease
Lysosomal Storage Disorder

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 19, 2013