A Study Evaluating the Safety of VX-152 Combination Therapy in Adults With Cystic Fibrosis

• Status: Completed
• Trial end date: 2018-01-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a Phase 2, randomized, double blind, placebo and active-controlled, parallel group, multicenter study designed to evaluate the safety and tolerability of VX-152 in Triple Combination (TC) with tezacaftor (TEZ; VX-661) and ivacaftor (IVA; VX-770) in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation and a minimal function (MF) CFTR mutation not likely to respond to TEZ and/or IVA therapy (F508del/MF), or who are homozygous for the F508del mutation of the CF transmembrane conductance regulator (CFTR) gene (F508del/F508del).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated
• Treatments: Ivacaftor

Criteria

Inclusion Criteria:

• Willing and able to comply with scheduled visits, treatment pan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.

• Body weight ≥35 kg.

• Sweat chloride value ≥ 60 mmol/L from test results obtained during screening.

• Subjects must have an eligible CFTR genotype:

• Cohorts 1A, 1B, 1C: Heterozygous for F508del and a minimal function mutation known or predicted not to respond to TEZ and/or IVA.

• Cohorts 2A, 2B: Homozygous for F508del.

• Subjects must have an FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height at the Screening Visit.

• Stable CF disease as judged by the investigator.

• Willing to remain on a stable CF medication regimen through the planned end of treatment or if applicable the Safety Follow-up Visit.

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.

• History of cirrhosis with portal hypertension.

• Risk factors for Torsade de Pointes.

• History of hemolysis.

• Glucose-6-phosphate dehydrogenase (G6PD) deficiency assessed at Screening.

• Clinically significant abnormal laboratory values at screening.

• An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before the first dose of study drug.

• Lung infection with organisms associated with a more rapid decline in pulmonary status.

• An acute illness not related to CF within 14 days before the first dose of study drug.

• A standard digital ECG demonstrating QTc >450 msec at screening.

• History of solid organ or hematological transplantation.

• History or evidence of cataract or lens opacity determined to be clinically significant by the ophthalmologist or optometrist, based on the ophthalmologic examination during the Screening Period.

• History of alcohol or drug abuse in the past year, including but not limited to, cannabis, cocaine, and opiates, as deemed by the investigator.

• Ongoing or prior participation in an investigational drug study with certain exceptions.

• Use of commercially available CFTR modulator within 14 days before screening (applies only to Cohorts 1A, 1B, and 1C).

• Pregnant or nursing females: Females of childbearing potential must have a negative pregnancy test at screening and Day 1.