VX-770 Expanded Access Program
Primary Purpose
Cystic Fibrosis
Status
Approved for marketing
Phase
Locations
Study Type
Expanded Access
Intervention
VX-770
Sponsored by
About this trial
This is an expanded access trial for Cystic Fibrosis
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
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT01381289
First Posted
June 22, 2011
Last Updated
February 8, 2012
Sponsor
Vertex Pharmaceuticals Incorporated
1. Study Identification
Unique Protocol Identification Number
NCT01381289
Brief Title
VX-770 Expanded Access Program
Official Title
VX-770 Expanded Access Program (EAP)
Study Type
Expanded Access
2. Study Status
Record Verification Date
February 2012
Overall Recruitment Status
Approved for marketing
Study Start Date
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Primary Completion Date
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Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated
4. Oversight
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
VX-770
Intervention Description
150 mg tablet, oral use, twice daily every 12 hours (q12h)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Years
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
12. IPD Sharing Statement
Learn more about this trial
VX-770 Expanded Access Program
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