Clinical trial NCT01476163: 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
Overall status: AVAILABLE
Conditions: Fabry Disease
Keywords: AT1001
migalastat
Fabry disease
Fabry
Amicus
lysosomal disease
lysosomal disorder
Lead sponsor: Amicus Therapeutics
Sponsor class: INDUSTRY
First posted: 2011-11-22
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no

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.

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 commercial Galafold or 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: At least 2 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.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Orange
country: United States
facility: CHOC Children's Hospital Division of Metabolic Disease
state: California
city: Baltimore
country: United States
facility: University of Maryland Medical Center
state: Maryland
city: Springfield
country: United States
facility: Kidney Care and Transplant Services of New England
state: Massachusetts
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
city: Parkville
country: Australia
facility: Royal Melbourne Hospital
state: Victoria
Study officials:
affiliation: Amicus Therapeutics
name: Medical Monitor, Clinical Research
role: STUDY_DIRECTOR
References: