Summary

for people ages 18 years and up (full criteria)
at San Francisco, California and other locations
study started
estimated completion

Description

Summary

The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.

Official Title

A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis

Keywords

Cystic Fibrosis Fibrosis Ivacaftor IVA

Eligibility

For people ages 18 years and up

Key Inclusion Criteria:

  • Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
  • On ivacaftor therapy
  • FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height

Key Exclusion Criteria:

  • History of clinically significant cirrhosis with or without portal hypertension
  • History of solid organ or hematological transplantation
  • Lung infection with organisms associated with a more rapid decline in pulmonary status

Other protocol defined Inclusion/Exclusion criteria may apply

Locations

  • UCSF Gateway Medical Center accepting new patients
    San Francisco California 94143 United States
  • Miller Children's Hospital / Long Beach Memorial accepting new patients
    Long Beach California 90806 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Vertex Pharmaceuticals Incorporated
ID
NCT03911713
Phase
Phase 2
Study Type
Interventional
Last Updated