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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01225211
First received: October 15, 2010
Last updated: October 2, 2012
Last verified: October 2012
History of Changes

October 15, 2010
October 2, 2012
October 2010
April 2013   (final data collection date for primary outcome measure)
  • Change in sweat chloride when VX-770 is administered in combination with VX-809 [ Time Frame: From Day 14 to Day 21 (Cohort 1); Day 28 to Day 56 (Cohort 2 and Cohort 3) ] [ Designated as safety issue: No ]
  • Safety and Tolerability [ Time Frame: Through Day 35 (Cohort 1); Day 70 (Cohort 2 and Cohort 3) ] [ Designated as safety issue: No ]
    Assessments will be measured based on adverse events, plasma samples (hematology, clinical chemistry, coagulation), urinalysis, electrocardiograms, and vital signs
  • Change in sweat chloride when VX-770 is administered in combination with VX-809 [ Time Frame: From Day 15 to Day 21 ] [ Designated as safety issue: No ]
  • Safety and tolerability [ Time Frame: Through Day 35 ] [ Designated as safety issue: No ]
    Assessments will be measured based on adverse events, plasma samples (hematology, clinical chemistry, coagulation), urinalysis, electrocardiograms, and vital signs
Complete list of historical versions of study NCT01225211 on ClinicalTrials.gov Archive Site
  • Change in percent predicted Forced expiratory volume in 1 second (FEV1) [ Time Frame: Through Day 21 (Cohort 1); Day 56 (Cohort 2 and Cohort 3) ] [ Designated as safety issue: No ]
  • Change in sweat chloride of increasing doses of VX-809 administered alone [ Time Frame: From Baseline to Day 14 (Cohort 1); From baseline to Day 28 (Cohort 2 and Cohort 3) ] [ Designated as safety issue: No ]
  • PK parameters, including exposure, concentration and half-life, of VX-809 and metabolite in plasma in the presence and absence of VX-770 [ Time Frame: Through Day 21 (Cohort 1); Day 56 (Cohort 2 and Cohort 3) ] [ Designated as safety issue: No ]
    Blood samples drawn during the study will be analyzed to measure the PK parameters, such as concentration, exposure and half-life of VX-809 and its metabolite.
  • PK parameters, including exposure, concentration and half-life, of VX-770 and metabolites in plasma in the presence of VX-809 [ Time Frame: Through Day 21 (Cohort 1); Day 56 (Cohort 2 and Cohort 3) ] [ Designated as safety issue: No ]
    Blood samples drawn during the study will be analyzed to measure the PK parameters, such as concentration, exposure and half-life of VX-770 and its metabolites.
  • Cystic Fibrosis Questionnaire (CFQ-R) Score [ Time Frame: Through Day 56 (Cohort 2 and Cohort 3) ] [ Designated as safety issue: No ]
  • Change in percent predicted Forced expiratory volume in 1 second (FEV1) [ Time Frame: Through Day 28 ] [ Designated as safety issue: No ]
  • Change in sweat chloride from baseline to Day 14 at increasing doses of VX-809 administered alone [ Time Frame: From Baseline to Day 14 ] [ Designated as safety issue: No ]
  • PK parameters, including exposure, concentration and half-life, of VX-809 and metabolite in plasma in the presence and absence of VX-770 [ Time Frame: Through Day 28 ] [ Designated as safety issue: No ]
    Blood samples drawn during the study will be analyzed to measure the PK parameters, such as concentration, exposure and half-life of VX-809 and its metabolite.
  • PK parameters, including exposure, concentration and half-life, of VX-770 and metabolites in plasma in the presence of VX-809 [ Time Frame: Through Day 28 ] [ Designated as safety issue: No ]
    Blood samples drawn during the study will be analyzed to measure the PK parameters, such as concentration, exposure and half-life of VX-770 and its metabolites.
Not Provided
Not Provided
 
Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
A Phase 2, Multicenter, Double-Blinded, Placebo-Controlled, Multiple-Dose Study to Evaluate Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-809 Alone and in Combination With VX-770 in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

The purpose of this study is to evaluate of the safety, efficacy, pharmacokinetics (PK) and pharmacodynamic (PD) effects of VX-809 alone and when coadministered with VX-770. This is the first study to assess the combination of VX-809 and VX-770 in CF subjects homozygous or heterozygous for the F508del-CFTR mutation.

This is a Phase 2, randomized, double-blind, placebo-controlled, multiple-dose study of orally administered VX-809 and VX-770 in CF subjects homozygous or heterozygous for the F508del-CFTR mutation. The primary objectives of the study are to evaluate the safety and tolerability when VX 809 is administered alone and when VX-809 is coadministered with VX-770 and to evaluate the effect of VX-809 administered alone and in combination VX-770 on sweat chloride

Enrollment is planned at clinical sites in the United States, Germany, Belgium, Australia and New Zealand. Up to 240 subjects may be enrolled. The study will be separated into 3 Cohorts. Cohort 1 will enroll 60 subjects. Cohorts 2 will enroll 100 subjects. Cohort 3 evaluating doses higher than Cohort 2, will enroll up to 80 subjects.
Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Cystic Fibrosis
  • Drug: VX-809
    tablet, taken once daily (qd) (Period 1 and 2)
  • Drug: VX-770
    tablet, taken every 12 hours (q12h) (Period 2)
  • Drug: VX-809 placebo
    tablet, taken once daily (qd) (Period 1 and 2)
  • Drug: VX-770 placebo
    tablet, taken every 12 hours (q12h) (Period 2)
  • Drug: VX-809 (Cohort 3)
    tablet, taken every 12 hours (q12h) or every 8 hours (q8h) (Period 1 and 2)
  • Drug: VX-770 (Cohort 3)
    tablet, taken every 12 hours (q12h) or every 8 hours (q8h) (Period 2)
  • Drug: VX-809 placebo
    tablet, taken every 12 hours (q12h) or every 8 hours (q8h) (Period 1 and 2)
  • Drug: VX-770 placebo
    tablet, taken every 12 hours (q12h) or every 8 hours (q8h) (Period 2)
  • Experimental: Treatment Arm (Cohort 1)
    Subjects randomized to study drug will take VX-809 once daily for 14 days. Beginning on Day 15, subjects will take both VX-809 and VX-770 through Day 21.
    Interventions:
    • Drug: VX-809
    • Drug: VX-770
  • Placebo Comparator: Placebo Arm (Cohort 1)
    Subjects randomized to placebo will remain on placebo from Day 1 through Day 21. Subjects will be given tablets that match both VX-809 and VX-770 and will follow the same dosing regimen as subjects receiving study drug.
    Interventions:
    • Drug: VX-809 placebo
    • Drug: VX-770 placebo
    • Drug: VX-809 placebo
    • Drug: VX-770 placebo
  • Experimental: Treatment Arm (Cohort 2)
    Subjects randomized to study drug will take VX-809 once daily for 28 days (Period 1). Beginning on Day 29, subjects will take both VX-809 and VX-770 through Day 56 (Period 2).
    Interventions:
    • Drug: VX-809
    • Drug: VX-770
  • Placebo Comparator: Placebo Arm (Cohort 2)
    Subjects randomized to placebo will remain on placebo from Day 1 through Day 56 (Period 1). Subjects will be given tablets that match both VX-809 and VX-770 and will follow the same dosing regimen as subjects receiving study drug (Period 2).
    Interventions:
    • Drug: VX-809 placebo
    • Drug: VX-770 placebo
    • Drug: VX-809 placebo
    • Drug: VX-770 placebo
  • Experimental: Treatment Arm (Cohort 3)
    Subjects randomized to study drug will take VX-809 for 28 days (Period 1). Beginning on Day 29, subjects will take both VX-809 and VX-770 through Day 56 (Period 2).
    Interventions:
    • Drug: VX-809 (Cohort 3)
    • Drug: VX-770 (Cohort 3)
  • Placebo Comparator: Placebo Arm (Cohort 3)
    Subjects randomized to placebo will remain on placebo from Day 1 through Day 56 (Period 1). Subjects will be given tablets that match both VX-809 and VX-770 and will follow the same dosing regiment as subjects receiving study drug (Period 2).
    Interventions:
    • Drug: VX-809 placebo
    • Drug: VX-770 placebo
    • Drug: VX-809 placebo
    • Drug: VX-770 placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
240
April 2013
April 2013   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Male or female subjects with confirmed diagnosis of CF
  • Must have the F508del-CFTR mutation on at least 1 allele.
  • FEV1 >= 40% of predicted normal for age, gender, and height (Knudson standards)
  • Subjects of child-bearing potential and who are sexually active must meet the contraception requirements

Exclusion Criteria:

  • 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 subject (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 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
  • Subjects enrolled in Cohort 1 or Cohort 2 will not be eligible for Cohort 3.
Both
18 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States,   Australia,   Belgium,   Germany,   New Zealand
 
NCT01225211
VX09-809-102, 2010-020413-90
Yes
Vertex Pharmaceuticals Incorporated
Vertex Pharmaceuticals Incorporated
Not Provided
Study Chair: Naimish Patel, MD Vertex Pharmaceuticals Incorporated
Vertex Pharmaceuticals Incorporated
October 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP