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Completed

NCT Number: NCT01225211

Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients 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 lumacaftor (VX-809) alone and when coadministered with ivacaftor (VX-770) in participants with cystic fibrosis, homozygous or heterozygous for the F508del-CFTR mutation.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Adelaide, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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 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 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)

Treatment and study plan

Lumacaftor

Drug

Tablet

Other names: VX-809, LUM

Ivacaftor

Drug

Tablet.

Other names: VX-770, IVA

Lumacaftor Placebo

Drug

Matching placebo tablet.

Ivacaftor Placebo

Drug

Matching placebo tablet.

Primary outcomes

  1. Cohort 1: Safety and Tolerability Based on Adverse Events (AEs)

    Time frame: Cohort 1: Day 1 up to 28 days after last dose (Last dose = Day 21)

    AE: any untoward medical occurrence in a participant during study; irrespective of relationship with treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent. AE includes serious AEs (SAEs) as well as Non-SAEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Number of participants with AEs and SAEs are reported. AE that started at/after initial dosing of study drug, or increased in severity after initial dosing of study drug is considered treatment-emergent. Results are reported separately for monotherapy period (Period 1: Day 1 to Day 14) and combination therapy period (Period 2: Day 15 to Day 21).

  2. Cohort 2 and 3: Safety and Tolerability Based on Adverse Events (AEs)

    Time frame: Cohort 2 and 3: Day 1 up to 28 days after last dose (Last dose = Day 56)

    Detailed description is provided in Outcome Measure 1. Results are reported separately for monotherapy period (Period 1: Day 1 to Day 28) and combination therapy period (Period 2: Day 29 to Day 56).

  3. Cohort 4: Safety and Tolerability Assessed by Number of Participants With AEs and SAEs

    Time frame: Cohort 4: Day 1 up to 28 days after last dose (Last dose = Day 56)

    AEs and SAEs are defined in Outcome Measure 1.

  4. Cohort 1: Absolute Change From Day 14 in Sweat Chloride at Day 21

    Time frame: Cohort 1: Day 14, Day 21

  5. Cohort 2 And 3: Absolute Change From Day 28 in Sweat Chloride at Day 56

    Time frame: Cohort 2 and 3: Day 28, Day 56

  6. Cohort 4: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 56

    Time frame: Cohort 4: Baseline, Day 56

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. ppFEV1 (predicted for age, gender, and height) was calculated using the Hankinson method.

Secondary outcomes

  1. Cohort 1: Absolute Change From Baseline in Sweat Chloride at Day 14

    Time frame: Cohort 1: Baseline, Day 14

  2. Cohort 2 And 3: Absolute Change From Baseline in Sweat Chloride at Day 14

    Time frame: Cohort 2: Baseline, Day 14

  3. Cohort 4: Absolute Change From Baseline in Sweat Chloride at Day 56

    Time frame: Cohort 4: Baseline, Day 56

  4. Cohort 1: Absolute Change From Day 14 in FEV1 at Day 21

    Time frame: Cohort 1: Day 14, Day 21

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

  5. Cohort 1: Absolute Change From Day 14 in ppFEV1 at Day 21

    Time frame: Cohort 1: Day 14, Day 21

    FEV1 and ppFEV1 are defined in Outcome Measure 6.

  6. Cohort 2 and 3: Absolute Change From Day 28 in ppFEV1 at Day 56

    Time frame: Cohort 2 and 3: Day 28, Day 56

    FEV1 and ppFEV1 are defined in Outcome Measure 6.

  7. Cohort 2 and 3: Relative Change From Day 28 in ppFEV1 at Day 56

    Time frame: Cohort 2 and 3: Day 28, Day 56

    FEV1 and ppFEV1 are defined in Outcome Measure 6.

  8. Cohort 2 and 3: Absolute Change From Baseline in ppFEV1 at Day 28 and 56

    Time frame: Cohort 2 and 3: Baseline, Day 28 and 56

    FEV1 and ppFEV1 are defined in Outcome Measure 6.

  9. Cohort 2 and 3: Relative Change From Baseline in FEV1 at Day 28 and 56

    Time frame: Cohort 2 and 3: Baseline, Day 28 and 56

    FEV1 and ppFEV1 are defined in Outcome Measure 6.

  10. Cohort 4: Relative Change From Baseline in Percent Predicted FEV1 at Day 56

    Time frame: Cohort 4: Baseline, Day 56

    FEV1 and ppFEV1 are defined in Outcome Measure 6.

  11. Cohort 2 and 3: Absolute Change From Day 28 in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Day 56

    Time frame: Cohort 2 and 3: Day 28, Day 56

    The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for subjects with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

  12. Cohort 4: Absolute Change From Baseline in CFQ-R Respiratory Domain Score at Day 56

    Time frame: Cohort 4: Baseline, Day 56

    CFQ-R respiratory domain is defined in Outcome Measure 17.

  13. Cohort 4: Absolute Change From Baseline in Body Mass Index (BMI) at Day 56

    Time frame: Cohort 4: Baseline, Day 56

    BMI was defined as weight in kilogram (kg) divided by height*height in square meter (m^2).

  14. Cohort 4: Absolute Change From Baseline in Weight at Day 56

    Time frame: Cohort 4: Baseline, Day 56

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 2, Multicenter, Double-Blinded, Placebo-Controlled, Multiple-Dose Study to Evaluate Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

Important dates

Study start
2010
Primary completion
2014
Study completion
2014
First posted
Oct 20, 2010
Registry last updated
Oct 5, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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