A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

Vertex Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Cystic Fibrosis

Treatments: Drug: Placebo; Drug: Ivacaftor; Drug: Tezacaftor/Ivacaftor

Study type: Interventional

Funder types: Industry

Identifiers

NCT02730208

VX15-661-112

Details and patient eligibility

About

The primary purpose of study is to evaluate the treatment effect of tezacaftor in combination with ivacaftor (TEZ/IVA) on chest imaging endpoints using low-dose computed tomography (LDCT) at Week 72, and to evaluate the safety of TEZ/IVA through Week 72.

Enrollment: 41 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Homozygous for the F508del CFTR mutation
• Confirmed diagnosis of CF
• Percent predicted forced expiratory volume (ppFEV1) ≥70% of predicted normal for age, sex, and height during screening.
• 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 (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug)
• Pregnant or nursing females.
• Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
• Any contraindication to undergoing chest imaging, as per the site's institutional guidelines

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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41 participants in 2 patient groups, including a placebo group

TEZ/IVA

Experimental group

Description:

Participants received TEZ 100 milligram (mg)/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks.

Treatment:

Drug: Tezacaftor/Ivacaftor

Drug: Ivacaftor

Placebo

Placebo Comparator group

Description:

Participants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks.

Treatment:

Drug: Placebo

Drug: Placebo