Cystic Fibrosis

A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

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 heterozygous for
F508del and a minimal function mutation (F/MF subjects).

Eligibility Criteria

Key Inclusion Criteria:

- Heterozygous for the F508del mutation (F/MF).

- 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

- Glucose-6-phosphate dehydrogenase (G6PD) deficiency

- 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.

Principal Investigator

Deepika Polineni, MD

Study Contact

Adam Schooley 913-588-1572

Estimated Completion Date

Thursday, August 1, 2019

ClinicalTrials.gov #

NCT03525444
06/25/2018