NCT02709109

A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation

A Phase 2a, Randomized, Double-blind, Placebo-controlled, Incomplete Block, Crossover Study to Evaluate the Safety and Efficacy of VX-371 in Subjects Aged 12 Years or Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation, and Being Treated With Orkambi

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 147 (actual)
• Sponsor: Parion Sciences · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the safety and efficacy of treatment with VX-371 in hypertonic saline compared to hypertonic saline alone in subjects with cystic fibrosis (CF) who are ≥12 years of age, homozygous for the F508del-cystic fibrosis transmembrane conductance regulator (CFTR) mutation, and being treated with Orkambi

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	VX-371 + HS
DRUG	Hypertonic saline
DRUG	Placebo
DRUG	Orkambi
DRUG	VX-371

Timeline

• Start date: 2016-02-01
• Primary completion: 2017-09-01
• Completion: 2017-09-01
• First posted: 2016-03-15
• Last updated: 2021-07-29
• Results posted: 2021-07-29

Locations

40 sites across 4 countries: United States, France, Ireland, United Kingdom

Regulatory

• FDA-regulated drug study