VX-770 Expanded Access Program (EAP)
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
Physicians interested in participating as a site should contact 800-745-4484.
Approved for marketing | Cystic Fibrosis | Site Unknown
Prospective Observational Study to Evaluate Biomarkers of Aminoglycoside Nephrotoxicity in Patients With Cystic Fibrosis
The goal of this clinical study is to advance the acceptance of new biomarkers designed to
detect drug-induced kidney injury in clinical trials.
The Kidney Safety Project is being conducted at four major medical centers:
- University of Southern California
- University of Minnesota
- MD Anderson Cancer Center
- Dana-Farber Cancer Institute.
Blood and urine samples will be collected from patients undergoing treatment with either
cisplatin or aminoglycosides, which are two different drugs known to cause injuries to the
proximal tubule of the kidney. Aminoglycosides are a common antibiotic drug taken by patients
with cystic fibrosis. Cisplatin is a common chemotherapy drug taken by patients with head and
The Aminoglycoside Study of the Kidney Safety Project is being conducted at the University of
Southern California and the University of Minnesota and aims to evaluate aminoglycoside
induced acute kidney injury in patients with cystic fibrosis.
The companion study, the Cisplatin Study of the Kidney Safety Project, is being conducted at
the MD Anderson Cancer Center and the Dana-Farber Cancer Institute and aims to evaluate
cisplatin induced acute kidney injury in patients with head and neck cancer.
The data from the Kidney Safety Project, from both the Aminoglycoside Study and the Cisplatin
Study, will be combined for determination of the best biomarkers for predicting drug-induced
acute kidney injury.