VX-770 Expanded Access Program

Cystic Fibrosis Clinical Trial

Official title:

VX-770 Expanded Access Program (EAP)

Clinical Trial Details — Status: Approved for marketing

Administrative data

• NCT number: NCT01381289
• Other study ID #: VX11-770-901
• Status: Approved for marketing
• Phase: N/A
• First received: June 22, 2011
• Last updated: February 8, 2012

Study information

• Verified date: February 2012
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Expanded Access

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Approved for marketing
• Enrollment: 0
• Gender: Both
• Age group: 6 Years and older

Eligibility

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

Study Design

N/A

Related Conditions & MeSH terms

• Cystic Fibrosis

Intervention

Drug:
• VX-770
150 mg tablet, oral use, twice daily every 12 hours (q12h)

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated