Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion

Vertex Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Cystic Fibrosis

Treatments: Drug: Placebo matched to Ivacaftor; Drug: Ivacaftor; Drug: VX-661; Drug: Placebo matched to VX-661

Study type: Interventional

Funder types: Industry

Identifiers

NCT02070744

VX13-661-103

Details and patient eligibility

About

The objective of this study was to evaluate the safety and efficacy of VX-661in combination with ivacaftor in participants with cystic fibrosis (CF) who are homozygous for F508del cystic fibrosis transmembrane conductance regulator (CFTR) mutation

Enrollment: 40 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Homozygous for the F508del CFTR mutation
• FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height
• 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 participant
• Pregnant and nursing females: Females of childbearing potential must have a negative pregnancy test at screening and Day 1 of the PC Phase and Day -7 or Day 1 of the OLE Phase (whichever was applicable)
• Sexually active participants of reproductive potential who are not willing to follow the contraception requirements
• The participant or a close relative of the participant is the investigator or sub investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

---

40 participants in 5 patient groups, including a placebo group

PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h

Experimental group

Description:

Participants received VX-661 50 milligram (mg) tablet plus Ivacaftor (IVA) 150 mg tablet every 12 hours (q12h) for 12 weeks.

Treatment:

Drug: VX-661

Drug: Ivacaftor

PC Phase: VX 661 placebo q12h + IVA placebo q12h

Placebo Comparator group

Description:

Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks.

Treatment:

Drug: Placebo matched to VX-661

Drug: Placebo matched to Ivacaftor

PC Phase: VX-661 100 mg qd + IVA 150 mg q12h

Experimental group

Description:

Participants received two VX-661 50 mg tablets once daily (qd) plus IVA 150 mg tablet q12h for 12 weeks.

Treatment:

Drug: VX-661

Drug: Ivacaftor

PC Phase: VX -661 placebo qd + IVA placebo q12h

Placebo Comparator group

Description:

Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks.

Treatment:

Drug: Placebo matched to VX-661

Drug: Placebo matched to Ivacaftor

OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h

Experimental group

Description:

Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase.

Treatment:

Drug: VX-661

Drug: Ivacaftor