A Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F)

Vertex Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: IVA; Drug: Placebo; Drug: TEZ/IVA; Drug: VX-445/TEZ/IVA

Study type: Interventional

Funder types: Industry

Identifiers

NCT03525548

VX17-445-103

2018-000184-89 (EudraCT Number)

Details and patient eligibility

About

This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F)

Enrollment: 113 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Homozygous for the F508del mutation (F/F)
• Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height

Key Exclusion Criteria:

• Clinically significant cirrhosis with or without portal hypertension
• Lung infection with organisms associated with a more rapid decline in pulmonary status
• Solid organ or hematological transplantation

Other protocol defined Inclusion/Exclusion criteria may apply

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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113 participants in 2 patient groups

TEZ/IVA

Active Comparator group

Description:

Following a run-in period of 4 weeks with Tezacaftor (TEZ)/Ivacaftor (IVA), participants received TEZ 100 milligram (mg)/IVA 150 mg as fixed-dose combination (FDC) tablet in the morning and IVA 150 mg as mono tablet in the evening for 4 weeks in the triple combination (TC) treatment period.

Treatment:

Drug: TEZ/IVA

Drug: Placebo

Drug: Placebo

Drug: IVA

VX-445/TEZ/IVA TC

Experimental group

Description:

Following a run-in period of 4 weeks with TEZ/IVA, participants received VX-445 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA 150 mg as mono tablet in the evening for 4 weeks in the TC treatment period.

Treatment:

Drug: VX-445/TEZ/IVA

Drug: Placebo

Drug: Placebo

Drug: IVA