A Study Evaluating the Safety and Efficacy of VX-440 Combination Therapy in Subjects With Cystic Fibrosis

Completed · Phase 2 · Has a placebo group

Conditions studied: Cystic Fibrosis

In brief

This is a Phase 2, randomized, double-blind, placebo- and active-controlled, parallel group, multicenter study to evaluate the safety, tolerability, and efficacy of VX-440 in dual and triple combination with tezacaftor (TEZ; VX-661) and ivacaftor (IVA; VX-770) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F508del/F508del), or who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ and/or IVA therapy (F508del/MF).

Key facts

Study ID

NCT02951182

Run by

Vertex Pharmaceuticals Incorporated

People needed

74

Starts

2016-10-01

Expected to finish

2017-08-01

Last updated by the study team

2020-08-28

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
• To prevent pregnancy, female participants of childbearing potential and their male partners will be required to use pre-specified, highly effective methods of non-hormonal contraception. Male participants with female partners of childbearing potential will be required to use a condom.
• Body weight ≥35 kg.
• Sweat chloride value ≥60 mmol/L from test results obtained during screening.
• Subjects must have an eligible CFTR genotype:
• Heterozygous for F508del and a minimal function (MF) mutation known or predicted not to be responsive to TEZ and/or IVA.
• Homozygous for F508del
• Subjects must have an FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height at the Screening Visit
• Stable CF disease as judged by the investigator.
• Willing to remain on a stable CF medication regimen through the planned end of treatment or, if applicable, the Safety Follow up Visit.

You may not qualify if…

• 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 subject.
• History of cirrhosis with portal hypertension.
• Risk factors for Torsade de Pointes
• History of hemolysis.
• Glucose-6-phosphate dehydrogenase (G6PD) deficiency assessed at Screening.
• Clinically significant abnormal laboratory values at screening
• An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before the first dose of study drug.
• Lung infection with organisms associated with a more rapid decline in pulmonary status
• An acute illness not related to CF within 14 days before the first dose of study drug
• A standard digital ECG demonstrating QTc >450 msec at screening.
• History of solid organ or hematological transplantation.
• History or evidence of cataract or lens opacity determined to be clinically significant by the ophthalmologist or optometrist based on the ophthalmologic examination during the Screening Period.
• History of alcohol or drug abuse in the past year, including but not limited to, cannabis, cocaine, and opiates, as deemed by the investigator.
• Ongoing or prior participation in an investigational drug study, with certain exceptions. (e.g., ongoing participation in NCT02565914)
• Use of commercially available CFTR modulator (e.g., Kalydeco, Orkambi) within 14 days before screening (applies only to the Heterozygous F508del/MF cohorts; does not apply to the Homozygous F508del/F508del Cohort).
• Pregnant or nursing females: Females of childbearing potential must have a negative pregnancy test at screening and Day 1.

Where it is running

• The Arizona Board of Regents on behalf of the University of Arizona — Tucson, Arizona, United States
• Stanford University — Palo Alto, California, United States
• University of California — San Francisco, California, United States
• National Jewish Health — Denver, Colorado, United States
• Joe Di Maggio Cystic Fibrosis & Pulmonary Center — Hollywood, Florida, United States
• Central Florida Pulmonary Group — Orlando, Florida, United States
• St. Luke's CF Center of Idaho — Boise, Idaho, United States
• Cystic Fibrosis Center of Chicago — Glenview, Illinois, United States
• The Johns Hopkins Hospital — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Massachusetts General Hospital Cystic Fibrosis Center — Boston, Massachusetts, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• New York Medical College — Hawthorne, New York, United States
• Long Island Jewish Medical Center — New Hyde Park, New York, United States
• Columbia University Medical Center — New York, New York, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Respiratory Diseases of Children and Adolescents — Oklahoma City, Oklahoma, United States
• UPMC OSPARS Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Sanford Children's Specialty Clinic Sanford Research USD — Sioux Falls, South Dakota, United States
• The University of Texas Southwestern Center — Dallas, Texas, United States
• Children's Hospital of the Kings Daughters — Norfolk, Virginia, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• Royal Adelaide Hospital — Adelaide, Australia
• Prince Charles Hospital — Chermside, Australia
• John Hunter Hospital & Hunter Medical Research Institute — New Lambton Heights, Australia

Full record on ClinicalTrials.gov

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