NCT01381289

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.

Trial Health

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 22, 2011

Completed
5 days until next milestone

First Posted

Study publicly available on registry

June 27, 2011

Completed
Last Updated

February 9, 2012

Status Verified

February 1, 2012

First QC Date

June 22, 2011

Last Update Submit

February 8, 2012

Conditions

Cystic Fibrosis

Interventions

VX-770DRUG

150 mg tablet, oral use, twice daily every 12 hours (q12h)

Eligibility Criteria

Age6 Years+
Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

ivacaftor

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Study Design

Study Type
expanded access
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 22, 2011

First Posted

June 27, 2011

Last Updated

February 9, 2012

Record last verified: 2012-02