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Completed

NCT Number: NCT02392234

A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation

The purpose of this study is to evaluate the efficacy and safety of VX-661 in combination with ivacaftor (IVA, VX-770) and IVA monotherapy in participants with Cystic Fibrosis (CF) who are heterozygous for F508del-CFTR allele and a second allele with a CFTR mutation predicted to have residual function.

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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

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

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

  • Heterozygous for F508del-CFTR and a second allele with a CFTR mutation predicted to have residual function
  • Forced Expiratory Volume in 1 Second (FEV1) greater than or equal to (≥) 40 percent (%) and less than or equal to (≤) 90% of predicted normal for age, sex, and height during screening
  • Sweat chloride value ≥60 millimole per liter (mmol/L) during screening OR as documented in the participant's medical record
  • 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
  • An acute upper or lower respiratory infection, pulmonary exacerbation
  • History of solid organ or hematological transplantation
  • Ongoing or prior participation in an investigational drug study (including studies investigating VX-661, lumacaftor [VX-809], and/or ivacaftor) within 30 days of screening
  • Pregnant and nursing females
  • Sexually active participants of reproductive potential who are not willing to follow the contraception requirements

Treatment and study plan

VX-661/Ivacaftor

Drug

Fixed dose combination tablet, oral use

Other names: VX-661+VX-770

Ivacaftor

Drug

Tablet, oral use

Other names: IVA, VX-770

Placebo matched to VX-661/ ivacaftor

Drug

Fixed dose combination tablet, oral use

Placebo matched to Ivacaftor

Drug

Tablet, oral use

Primary outcomes

  1. Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Average of Week 4 and Week 8

    Time frame: Baseline, Week 4 and Week 8 of each treatment period

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

Secondary outcomes

  1. Absolute Change From Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Average of Week 4 and Week 8

    Time frame: Baseline, Week 4 and Week 8 of each treatment period

    The CFQ-R assessed respiratory symptoms on a scale with scores ranging from 0 to 100; where higher scores indicated fewer symptoms and better health-related quality of life.

  2. Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: Day 1 up to Week 28

  3. Relative Change From Study Baseline in ppFEV1 at Average of Week 4 and Week 8

    Time frame: Baseline, Week 4 and Week 8 of each treatment period

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

  4. Absolute Change From Study Baseline in Sweat Chloride at Average of Week 4 and Week 8

    Time frame: Baseline, Week 4 and Week 8 of each treatment period

  5. Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1 VX-661), IVA and IVA Metabolite (M1 IVA) After Administration of VX-661/IVA Combination Therapy

    Time frame: Pre-morning dose on Week 8 of each treatment period

  6. Ctrough of IVA and IVA Metabolite (M1 IVA) After Administration of IVA Monotherapy

    Time frame: Pre-morning dose on Week 8 of each treatment period

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation, and a Second Allele With a CFTR Mutation Predicted to Have Residual Function

Important dates

Study start
2015
Primary completion
2017
Study completion
2017
First posted
Mar 18, 2015
Registry last updated
Jun 12, 2018

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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