VX-770 Expanded Access Program

This treatment has been approved for sale to the public.
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01381289
First received: June 22, 2011
Last updated: February 8, 2012
Last verified: February 2012
  Purpose

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.

  Eligibility

Ages Eligible for Study:   6 Years and older
Genders Eligible for Study:   Both
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

No Contacts or Locations Provided
  More Information

No publications provided

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT01381289     History of Changes
Other Study ID Numbers: VX11-770-901
Study First Received: June 22, 2011
Last Updated: February 8, 2012
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Cystic Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases

ClinicalTrials.gov processed this record on September 29, 2014