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Completed

NCT Number: NCT03460990

A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)

This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F).

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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

The Alfred Hospital, Melbourne, Victoria, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Key Inclusion Criteria:

  • Homozygous for the F508del mutation (F/F)
  • Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height

Key Exclusion Criteria:

  • Clinically significant cirrhosis with or without portal hypertension
  • Lung infection with organisms associated with a more rapid decline in pulmonary status
  • Solid organ or hematological transplantation

Other protocol defined Inclusion/Exclusion criteria may apply

Treatment and study plan

VX-659/TEZ/IVA

Drug

Participants received VX-659/TEZ/IVA orally once daily in the morning.

Other names: VX-659/VX-661/VX-770, VX-659/tezacaftor/ivacaftor

TEZ/IVA

Drug

Participants received TEZ/IVA orally once daily in the morning.

Other names: VX-661/VX-770, tezacaftor/ivacaftor

IVA

Drug

Participants received IVA orally once daily in the evening.

Other names: VX-770, ivacaftor

Placebo

Drug

Participants received placebo matched TEZ/IVA orally once daily in the morning.

Primary outcomes

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

    Time frame: From Baseline at Week 4

    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 (SwCl)

    Time frame: From Baseline at Week 4

    Sweat samples were collected using an approved collection device.

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

    Time frame: From Baseline at Week 4

    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.

  3. Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to Week 8)

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

    Time frame: From Day 1 and Week 4

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F)

Important dates

Study start
2018
Primary completion
2018
Study completion
2018
First posted
Mar 9, 2018
Registry last updated
Oct 17, 2019

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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