VX-770 Expanded Access Program

• Status: Approved for marketing
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

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.

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated
• Treatments: Ivacaftor

Criteria

Inclusion 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

Exclusion Criteria:

• 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