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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

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

NCT ID

NCT01476163

First Posted

November 17, 2011

Last Updated

January 22, 2019

Sponsor

Amicus Therapeutics

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

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Primary Completion Date

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Study Completion Date

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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

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

Learn more about this trial

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

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