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Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease

Primary Purpose

Fabry Disease

Status
Approved for marketing
Phase
Locations
International
Study Type
Expanded Access
Intervention
migalastat HCl
Sponsored by
Amicus Therapeutics
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Fabry Disease focused on measuring AT1001, Lysosomal storage disease, Lysosomal Storage Disorder, migalastat, Fabry disease, Fabry, Amicus

Eligibility Criteria

16 Years - 74 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Confirmed GLA mutation predicted to be responsive migalastat in the human embryonic kidney (HEK-293) cell-based assay
  • 16-74 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

Exclusion Criteria:

  • Estimated glomerular filtration rate (eGFR) or GFR <30 mL/minute
  • Scheduled for renal or other organ transplant or replacement therapy
  • 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.

Sites / Locations

  • CHOC Children's Hospital Division of Metabolic Disease
  • University of Maryland Medical Center
  • Kidney Care and Transplant Services of New England
  • Icahn School of Medicine at Mount Sinai
  • Royal Melbourne Hospital

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
November 17, 2011
Last Updated
January 22, 2019
Sponsor
Amicus Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT01476163
Brief Title
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
Official Title
Physician Initiated Expanded Access Request for Treatment Use of Migalastat Hydrochloride (AT1001), an Investigational Treatment for Individual Patients With Fabry Disease (AT1001-188)
Study Type
Expanded Access

2. Study Status

Record Verification Date
January 2019
Overall Recruitment Status
Approved for marketing
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Amicus Therapeutics

4. Oversight

5. Study Description

Brief Summary
This program allows physicians to request permission from Amicus Therapeutics (Amicus) 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.
Detailed Description
This Physician Initiated Request program allows physicians to request permission from Amicus to receive migalastat HCl for specific patients with Fabry disease who have a mutation amenable to this treatment, who do not have access to available treatment alternatives, or do not meet requirements for participation in an existing migalastat clinical study. Up to 20 patients worldwide may be treated. Patients must meet specific criteria to receive Amicus permission for participation. Key criteria for participation include: 16-74 years old; 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease
Keywords
AT1001, Lysosomal storage disease, Lysosomal Storage Disorder, migalastat, Fabry disease, Fabry, Amicus

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
migalastat HCl
Other Intervention Name(s)
AT1001, migalastat
Intervention Description
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
16 Years
Maximum Age & Unit of Time
74 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Confirmed GLA mutation predicted to be responsive migalastat in the human embryonic kidney (HEK-293) cell-based assay 16-74 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 Exclusion Criteria: Estimated glomerular filtration rate (eGFR) or GFR <30 mL/minute Scheduled for renal or other organ transplant or replacement therapy 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor, Clinical Research
Organizational Affiliation
Amicus Therapeutics
Official's Role
Study Director
Facility Information:
Facility Name
CHOC Children's Hospital Division of Metabolic Disease
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
University of Maryland Medical Center
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21201
Country
United States
Facility Name
Kidney Care and Transplant Services of New England
City
Springfield
State/Province
Massachusetts
ZIP/Postal Code
01104
Country
United States
Facility Name
Icahn School of Medicine at Mount Sinai
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Royal Melbourne Hospital
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3050
Country
Australia

12. IPD Sharing Statement

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Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease

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