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Completed

NCT Number: NCT03224351

A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis

This is a Phase 2, randomized, double-blind, placebo- and tezacaftor/ivacaftor (TEZ/IVA)-controlled, parallel-group, 3-part, multicenter study designed to evaluate the safety and efficacy of VX-659 in triple combination (TC) with TEZ and IVA in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F/F genotype), or who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ, IVA, or TEZ/IVA (F/MF genotypes).

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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

Cork University Hospital, Cork, Ireland

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Key Inclusion Criteria:

  • Body weight ≥35 kg.
  • Subjects must have an eligibleCFTR genotype.
  • Part 1 and Part 3: Heterozygous for F508del and an MF mutation (F/MF)
  • Part 2: Homozygous for F508del (F/F)
  • FEV1 value ≥40% and ≤90% of predicted mean for age, sex, and height

Key Exclusion Criteria:

  • History of clinically significant cirrhosis with or without portal hypertension.
  • Glucose-6-phosphate dehydrogenase (G6PD) deficiency
  • Lung infection with organisms associated with a more rapid decline in pulmonary status.
  • History of solid organ or hematological transplantation.

Other protocol defined Inclusion/Exclusion criteria may apply.

Treatment and study plan

VX-659

Drug

Tablet for oral administration.

TEZ/IVA

Drug

TEZ/IVA fixed-dose combination tablet for oral administration.

Other names: VX-661/VX-770, Tezacaftor/Ivacaftor

IVA

Drug

Tablet for oral administration.

Other names: VX-770, Ivacaftor

Placebo (matched to VX-659/TEZ/IVA)

Drug

Placebo matched to VX-659 and TEZ/IVA.

TEZ

Drug

Tablet for oral administration.

Other names: VX-661, Tezacaftor

VX-561

Drug

Tablet for oral administration.

Other names: CTP-656

Placebo (matched to VX-659/TEZ/VX-561)

Drug

Placebo matched to VX-659, TEZ and VX-561.

Primary outcomes

  1. Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    Time frame: Day 1 Through Safety Follow-up (up to Day 61 for Part 1, Day 85 for Part 2 and Day 57 for Part 3)

  2. Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

    Time frame: From Baseline Through Day 29

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

Secondary outcomes

  1. Absolute Change in Sweat Chloride Concentrations

    Time frame: From Baseline Through Day 29

    Sweat samples were collected using an approved collection device.

  2. Relative Change in ppFEV1

    Time frame: From Baseline Through Day 29

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

  3. Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score

    Time frame: From Baseline at Day 29

    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, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

  4. Observed Pre-dose Concentration (Ctrough) of VX-659, TEZ, M1-TEZ, IVA, M1-IVA, and VX-561

    Time frame: Pre-dose at Day 15 and Day 29

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-659 Combination Therapy in Subjects Aged 18 Years and Older With Cystic Fibrosis

Important dates

Study start
2017
Primary completion
2018
Study completion
2018
First posted
Jul 21, 2017
Registry last updated
Apr 22, 2021

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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