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VX-770 Expanded Access Program

This treatment has been approved for sale to the public.
ClinicalTrials.gov Identifier:
First Posted: June 27, 2011
Last Update Posted: February 9, 2012
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
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.

Condition Intervention
Cystic Fibrosis Drug: VX-770

Study Type: Expanded Access     What is Expanded Access?
Official Title: VX-770 Expanded Access Program (EAP)

Resource links provided by NLM:

Further study details as provided by Vertex Pharmaceuticals Incorporated:

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


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All

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
  Contacts and Locations
No Contacts or Locations Provided
  More Information

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT01381289     History of Changes
Other Study ID Numbers: VX11-770-901
First Submitted: June 22, 2011
First Posted: June 27, 2011
Last Update Posted: February 9, 2012
Last Verified: February 2012

Additional relevant MeSH terms:
Cystic Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action