- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01381289
VX-770 Expanded Access Program
VX-770 Expanded Access Program (EAP)
Study Overview
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.
Study Type
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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 Plan
How is the study designed?
Collaborators and Investigators
Study record dates
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- VX11-770-901
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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