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

NCT01476163 | Unknown

• 1 other identifier: AT1001-188
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This program allows physicians to request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific patients with Fabry disease. Treatment is open label.

Conditions

Fabry Disease

Keywords

AT1001; migalastat; Fabry disease; Fabry; Amicus; lysosomal disease; lysosomal disorder

Interventions

migalastat HCl 150 mg DRUG

150 mg capsule

Also known as: AT1001, migalastat

migalastat HCl 20 mg DRUG

20 mg dispersible tablets; dosing will be based on body weight

Also known as: AT1001, migalastat

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Confirmed GLA mutation predicted to be responsive migalastat in the human embryonic kidney (HEK-293) cell-based assay
• At least 2 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

You may not qualify if:

• Scheduled for renal or other organ transplant or replacement therapy
• Receiving GLYSET® (miglitol), ZAVESCA® (miglustat) or enzyme replacement therapy FABRAZYME® (agalsidase beta), REPLAGAL™ (agalsidase alpha), or Elfabrio® (pegunigalsidase alfa)
• 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.

Sponsors & Collaborators

• Amicus Therapeutics: lead

MeSH Terms

Conditions

Fabry Disease

Interventions

migalastat; larazotide acetate

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Medical Monitor, Clinical Research

  Amicus Therapeutics

  STUDY DIRECTOR

Central Study Contacts

Amicus Therapeutics Patient Advocacy

CONTACT

patientadvocacy@amicusrx.com

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 17, 2011
• First Posted: November 22, 2011
• Last Updated: July 3, 2025

Record last verified: 2025-05