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Clinical Trial Details — Status: Approved for marketing

Administrative data

NCT number NCT01476163
Other study ID # AT1001-188
Secondary ID
Status Approved for marketing
Phase
First received
Last updated

Study information

Verified date January 2019
Source Amicus Therapeutics
Contact n/a
Is FDA regulated No
Health authority
Study type Expanded Access

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Approved for marketing
Enrollment 0
Est. completion date
Est. primary completion date
Accepts healthy volunteers No
Gender All
Age group 16 Years to 74 Years
Eligibility 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.

Study Design


Related Conditions & MeSH terms


Intervention

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

Locations

Country Name City State
Australia Royal Melbourne Hospital Parkville Victoria
United States University of Maryland Medical Center Baltimore Maryland
United States Icahn School of Medicine at Mount Sinai New York New York
United States CHOC Children's Hospital Division of Metabolic Disease Orange California
United States Kidney Care and Transplant Services of New England Springfield Massachusetts

Sponsors (1)

Lead Sponsor Collaborator
Amicus Therapeutics

Countries where clinical trial is conducted

United States,  Australia, 

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