Cystic Fibrosis Clinical Trial
Official title:
VX-770 Expanded Access Program (EAP)
The purpose of this expanded access program is to provide VX-770 prior to its commercial availability to people with cystic fibrosis (CF) who have at least one copy of the G551D-CFTR mutation and who are in critical medical need and who are not eligible for participation in other Vertex-sponsored studies.
VX-770, a compound being developed by Vertex Pharmaceuticals Incorporated (Vertex) for the
treatment of CF, is an orally bioavailable small molecule that targets the underlying defect
in CF, the dysfunctional CFTR protein. In Phase 3 studies of VX-770 in patients with CF and
a G551D CFTR mutation, improvements in CFTR function (measured by reduction in sweat
chloride concentration) and improvements in lung function were observed.
Patients who are interested in the VX-770 Expanded Access should contact their CF physician
about participation.
Physicians interested in participating as a site should contact 800-745-4484.
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