VX-770 Expanded Access Program (EAP)
DescriptionVX-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.
Inclusion and Exclusion Criteria
- Male or female with confirmed diagnosis of CF, with a sweat chloride >60 mmol/L OR 2 CF-causing mutations AND chronic sinopulmonary disease OR gastrointestinal/nutritional abnormalities.
- Have the G551D-CFTR mutation in at least 1 allele
- Will be aged 6 years or older on the date of signed informed consent form
- Highest FEV1 in the 6 months prior to screening is ≤ 40% predicted value or patient is documented to be active on the lung transplant wait list
- If female, currently pregnant
- Abnormal liver function, at screening on recent clinical laboratory testing, defined as >3 × upper limit of normal (ULN), of any 3 or more of the following: AST, ALT, GGT, serum alkaline phosphatase, total bilirubin
- Is currently requiring invasive mechanical ventilation
- Is currently participating, or has participated in the past 30 days in another therapeutic or clinical study
Please contact the trial administrator to learn more about where you can participate in this trial. Please use the contact form on the right side.