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VX-770 Expanded Access Program

Primary Purpose

Cystic Fibrosis

Status
Approved for marketing
Phase
Locations
Study Type
Expanded Access
Intervention
VX-770
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Cystic Fibrosis

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All Sexes

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

    First Posted
    June 22, 2011
    Last Updated
    February 8, 2012
    Sponsor
    Vertex Pharmaceuticals Incorporated
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    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
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    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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