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Completed

NCT Number: NCT02070744

Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion

The objective of this study was to evaluate the safety and efficacy of VX-661in combination with ivacaftor in participants with cystic fibrosis (CF) who are homozygous for F508del cystic fibrosis transmembrane conductance regulator (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

Birmingham, Alabama, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Homozygous for the F508del CFTR mutation
  • FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height
  • Stable CF disease as judged by the investigator

Exclusion criteria

  • History of any comorbidity 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
  • Pregnant and nursing females: Females of childbearing potential must have a negative pregnancy test at screening and Day 1 of the PC Phase and Day -7 or Day 1 of the OLE Phase (whichever was applicable)
  • Sexually active participants of reproductive potential who are not willing to follow the contraception requirements
  • The participant or a close relative of the participant is the investigator or sub investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study.

Treatment and study plan

VX-661

Drug

Tablet, oral use

Ivacaftor

Drug

Film coated tablet, oral use

Placebo matched to VX-661

Drug

Tablet, oral use

Placebo matched to Ivacaftor

Drug

Film coated tablet, oral use

Primary outcomes

  1. PC Phase: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: Baseline (PC Phase) up to 112 days

    AE: Any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation considered treatment-emergent. Baseline was defined as Day 1 of PC Phase.

  2. OLE Phase: Number of Participants With Treatment-Emergent AEs and SAEs

    Time frame: Baseline (OLE Phase) up to 364 days

    AE: Any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation considered treatment-emergent. Baseline was defined as Day 1 of the OLE Phase.

Secondary outcomes

  1. PC Phase: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 12

    Time frame: Baseline (PC Phase), Through Week 12

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of PC Phase.

  2. OLE Phase: Absolute Change From Baseline in Percent Predicted FEV1 Through Week 40

    Time frame: Baseline (OLE Phase), Through Week 40

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of the OLE Phase.

  3. PC Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 12

    Time frame: Baseline (PC Phase), Through Week 12

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of PC Phase.

  4. OLE Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 40

    Time frame: Baseline (OLE Phase), Through Week 40

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of the OLE Phase.

  5. PC Phase: Absolute Change From Baseline in Sweat Chloride Through Week 12

    Time frame: Baseline (PC Phase), Through Week 12

    Sweat samples were collected using an approved collection device. Baseline was defined as Day 1 of PC Phase.

  6. OLE Phase: Absolute Change From Baseline in Sweat Chloride Through Week 40

    Time frame: Baseline (OLE Phase), Through Week 40

    Sweat samples were collected using an approved collection device. Baseline was defined as Day 1 of the OLE Phase.

  7. PC Phase: Absolute Change From Baseline in Body Weight at Week 12

    Time frame: Baseline (PC Phase), Week 12

    Baseline was defined as Day 1 of PC Phase.

  8. OLE Phase: Absolute Change From Baseline in Body Weight at Week 40

    Time frame: Baseline (OLE Phase), Week 40

    Baseline was defined as Day 1 of the OLE Phase.

  9. PC Phase: Absolute Change From Baseline Body Mass Index (BMI) at Week 12

    Time frame: Baseline (PC Phase), Week 12

    BMI was calculated using following formula: BMI = Weight in kg/height in square meter (m^2). Baseline was defined as Day 1 of PC Phase.

  10. OLE Phase: Absolute Change From Baseline BMI at Week 40

    Time frame: Baseline (OLE Phase), Week 40

    BMI was calculated using following formula: BMI = Weight in kg/height in m^2. Baseline was defined as Day 1 of the OLE Phase.

  11. PC Phase: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 12

    Time frame: Baseline (PC Phase), Through Week 12

    The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants 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. Baseline was defined as Day 1 of PC Phase.

  12. OLE Phase: Absolute Change From Baseline in CFQ-R Respiratory Domain Score Through Week 40

    Time frame: Baseline (OLE Phase), Through Week 40

    The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants 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. Baseline was defined as Day 1 of the OLE Phase.

  13. PC Phase: Maximum Plasma Concentration (Cmax) of VX-661 and IVA

    Time frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85

  14. PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24h) of VX-661

    Time frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85

    Pharmacokinetic (PK) sampling was performed up to 12 hours post-dose on Day 85. For Arm VX-661 50 mg q12h + IVA 150 mg q12h (VX-661 q12h regimen), area under the concentration versus time curve from time 0 to 12 hours (AUC0-12h) was multiplied by 2 to obtain AUC0-24h.

  15. PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12h) of IVA

    Time frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85

  16. PC Phase: Time to Reach Cmax (Tmax) of VX-661 and IVA

    Time frame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 2, Randomized, Multicenter, Double Blind, Placebo Controlled Study to Evaluate Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-661 in Combination With Ivacaftor for 12 Weeks in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation With an Open-Label Extension

Important dates

Study start
2014
Primary completion
2016
Study completion
2016
First posted
Feb 25, 2014
Registry last updated
Sep 24, 2025

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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