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Completed

NCT Number: NCT05033080

A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)

The purpose of this study evaluates the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who were heterozygous for F508del and a minimal function mutation (F/MF participants).

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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

Royal Prince Alfred Hospital, Camperdown, Australia

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Who can participate

Healthy volunteers accepted: No

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

Key Inclusion Criteria:

  • Heterozygous for F508del and a minimal function mutation (F/MF genotype)
  • Forced expiratory volume in 1 second (FEV1) value >=40% and <=90% of predicted mean for age, sex, and height for participants currently receiving ELX/TEZ/IVA therapy; FEV1 >=40% and <=80% for participants not currently receiving ELX/TEZ/IVA

Key Exclusion Criteria:

  • History of solid organ or hematological transplantation
  • Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
  • Lung infection with organisms associated with a more rapid decline in pulmonary status
  • Pregnant or breast-feeding females

Other protocol defined Inclusion/Exclusion criteria may apply

Treatment and study plan

VX-121/TEZ/D-IVA

Drug

Fixed-dose combination tablets for oral administration.

Other names: VX-121/VX-661/CTP-656, VX-121/VX-661/VX-561, VX-121/tezacaftor/deutivacaftor

ELX/TEZ/IVA

Drug

Fixed-dose combination tablets for oral administration.

Other names: VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor

IVA

Drug

Tablet for oral administration.

Other names: VX-770, ivacaftor

Placebo (matched to VX-121/TEZ/D-IVA)

Drug

Placebo matched to VX-121/TEZ/D-IVA for oral administration.

Placebo (matched to ELX/TEZ/IVA)

Drug

Placebo matched to ELX/TEZ/IVA for oral administration.

Placebo (matched to IVA)

Drug

Placebo matched to IVA for oral administration.

Primary outcomes

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

    Time frame: From Baseline Through Week 24

    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 Through Week 24

    Sweat samples were collected using an approved collection device.

  2. Percentage of Participants With SwCl <60 mmol/L (Pooled With Data From Study VX20-121-103)

    Time frame: From Baseline Through Week 24

    Sweat samples were collected using an approved collection device.

  3. Percentage of Participants With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-103)

    Time frame: From Baseline Through Week 24

    Sweat samples were collected using an approved collection device.

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-121 Combination Therapy in Subjects With Cystic Fibrosis (CF) Who Are Heterozygous for F508del and a Minimal Function Mutation (F/MF)

Important dates

Study start
2021
Primary completion
2023
Study completion
2023
First posted
Sep 2, 2021
Registry last updated
Oct 3, 2024

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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