Status:

COMPLETED

Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation

Lead Sponsor:

Vertex Pharmaceuticals Incorporated

Conditions:

Cystic Fibrosis

Eligibility:

All Genders

18+ years

Phase:

PHASE2

Brief Summary

The purpose of this study is to evaluate of the safety, efficacy, pharmacokinetics (PK) and pharmacodynamic (PD) effects of lumacaftor (VX-809) alone and when coadministered with ivacaftor (VX-770) in...

Eligibility Criteria

Inclusion

  • Male or female participants with confirmed diagnosis of CF
  • Must have the F508del-CFTR mutation on at least 1 allele.
  • FEV1 greater than equal (\>=) 40% of predicted normal for age, gender, and height (Knudson standards)(Cohort 1, 2, and 3); FEV1 40-90% of predicted normal for age, gender, and height (Hankinson standards (Cohort 4)
  • Participant of child-bearing potential and who are sexually active must meet the contraception requirements

Exclusion

  • History of any illness or condition that, in the opinion of the investigator might confound the results of the study or pose an additional risk in administering study drug to the participant (e.g., cirrhosis with portal hypertension).
  • An acute illness including acute upper or lower respiratory infection, pulmonary exacerbation or changes in therapy (including antibiotics) for pulmonary disease within 14 days (Cohort 1, 2, and 3) or 28 days (Cohort 4) before receiving the first dose of study drug.
  • History of solid organ or hematological transplantation.
  • History of alcohol abuse or drug addiction in the past year, including cannabis, cocaine, and opiates.
  • Ongoing participation in another therapeutic clinical study, or prior participation in an investigational drug study without appropriate washout
  • Pregnant or nursing females; females of childbearing potential who are unwilling or unable to use an acceptable method of non-hormonal contraception
  • Participants enrolled in Cohort 1 or Cohort 2 will not be eligible for Cohort 3
  • Ongoing or prior participation in an investigational drug study within 30 days of the Screening Visit
  • Heterozygous participants who participated in Cohort 2 and meet the eligibility criteria for Cohort 4 may participate in Cohort 4
  • Evidence of lens opacity or cataract as determined by the ophthalmologic examination (Cohort 4 only)

Key Trial Info

Start Date :

October 1 2010

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

April 1 2014

Estimated Enrollment :

312 Patients enrolled

Trial Details

Trial ID

NCT01225211

Start Date

October 1 2010

End Date

April 1 2014

Last Update

October 5 2015

Active Locations (55)

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Page 1 of 14 (55 locations)

1

Birmingham, Alabama, United States

2

Anchorage, Alaska, United States

3

La Jolla, California, United States

4

Long Beach, California, United States