A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

Vertex Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Cystic Fibrosis

Treatments: Drug: Tezacaftor/Ivacaftor matching placebo; Drug: Ivacaftor; Drug: Tezacaftor/Ivacaftor; Drug: Ivacaftor matching placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02508207

VX14-661-111

Details and patient eligibility

About

To evaluate the clinical mechanisms of action in lung and extrapulmonary systems of VX-661 (tezacaftor; TEZ) in combination with ivacaftor (IVA) (TEZ/IVA) in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.

Enrollment: 34 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male and female participants, homozygous for the F508del CFTR mutation
• Confirmed diagnosis of CF by sweat chloride testing
• Forced Expiratory Volume in 1 Second (FEV1) ≥40% and ≤90% of predicted normal for age, sex, and height at Screening Visit
• 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 subject.
• An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1
• History or evidence of clinically significant findings on ophthalmologic examination during the Screening Period.
• History of solid organ or hematological transplantation
• Pregnant or nursing females
• Participants who have had radiation exposure within 1 year before the first mucociliary clearance (MCC) procedure that would cause them to exceed federal regulations by participating in this study
• In the opinion of the investigator, unable to adequately perform inhalation maneuvers during the MCC procedures

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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34 participants in 2 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

Participants received placebo matched to tezacaftor (TEZ)/ivacaftor (IVA) fixed dose combination (FDC) tablet orally once daily in the morning followed by placebo matched to IVA tablet orally once daily in the evening for 29 days.

Treatment:

Drug: Ivacaftor matching placebo

Drug: Tezacaftor/Ivacaftor matching placebo

TEZ/IVA

Experimental group

Description:

Participants received 100 milligram (mg) TEZ/150 mg IVA FDC tablet orally once daily in the morning followed by 150 mg IVA tablet orally once daily in the evening for 29 days.

Treatment:

Drug: Tezacaftor/Ivacaftor

Drug: Ivacaftor