Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
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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.
Condition or disease
Drug: migalastat HCl
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.
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Ages Eligible for Study:
16 Years to 74 Years (Child, Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
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
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.