NCT01381289

VX-770 Expanded Access Program

VX-770 Expanded Access Program (EAP)

• Status: Approved For Marketing
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: —

Summary

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.

Detailed description

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.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	VX-770	150 mg tablet, oral use, twice daily every 12 hours (q12h)

Timeline

• First posted: 2011-06-27
• Last updated: 2012-02-09