VX-770 Expanded Access Program

NCT ID: NCT01381289

Last Updated: 2012-02-09

Basic Information

• Recruitment Status: APPROVED_FOR_MARKETING
• Study Classification: EXPANDED_ACCESS

Brief 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

VX-770

150 mg tablet, oral use, twice daily every 12 hours (q12h)

Intervention Type: DRUG

Eligibility 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

• Minimum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Other Identifiers

VX11-770-901

Identifier Type: -

Identifier Source: org_study_id