A Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis

• Status: Completed
• Trial end date: 2018-03-27
• Target enrollment: 0
• Participant gender: All

Summary

This is a first-in-human and proof-of-concept study of VX-445. The study includes 6 parts. Parts A, B, and C will be conducted in healthy subjects. Parts D, E, and F will be conducted in subjects with Cystic Fibrosis (CF) who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F/F genotype), or who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ, IVA, or TEZ/IVA (F/MF genotypes).

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated
• Treatments: Ivacaftor

Criteria

Key Inclusion Criteria:

Parts A, B, and C:

• Female subjects must be of non-childbearing potential.

• Between the ages of 18 and 55 years, inclusive.

• Body mass index (BMI) of 18.0 to 32.0 kg/m2, inclusive, and a total body weight >50 kg

Parts D, E, and F:

• Body weight ≥35 kg.

• Subjects must have an eligible CFTR genotype:

• Parts D and F: Heterozygous for F508del and an MF mutation (F/MF)

• Part E: Homozygous for F508del (F/F)

• FEV1 value ≥40% and ≤90% of predicted mean for age, sex, and height.

Key Exclusion Criteria:

Parts A, B, and C:

• Any condition possibly affecting drug absorption.

• History of febrile illness within 14 days before the first study drug dose.

• Glucose-6-phosphate dehydrogenase (G6PD) deficiency.

Parts D, E, and F:

• History of 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.

• History of solid organ or hematological transplantation.

Other protocol defined Inclusion/Exclusion criteria may apply.