NCT01746784

Safety and Pharmacokinetic Study of N6022 in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation

A Phase 1b, Randomized, Double-Blind, Placebo-Controlled, Dose Escalation Study of N6022 to Evaluate Safety and Pharmacokinetics in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation (SNO1)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 66 (actual)
• Sponsor: Nivalis Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to investigate the safety, tolerability and pharmacokinetics of N6022, and to obtain descriptive information on the effect of N6022 on biomarkers of CFTR function and inflammation in adult cystic fibrosis subjects who are homozygous for the F508del-CFTR mutation.

Detailed description

This is a double-blind, randomized, placebo-controlled, multicenter, sequential dose-escalation study which will occur in two parts. All selection criteria, assessments and procedures described in this protocol will be applied to both parts. Up to 5 cohorts will be studied with a total of 67 patients at approximately 18 clinical sites in the United States.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	N6022	Intravenous solution of N6022 in normal saline administered by infusion pump over 1-8 minutes depending on the dose
DRUG	Normal saline	Intravenous solution of 0.9% (weight/volume) NaCl administered by infusion pump over 1-8 minutes depending on dose of active drug used in same cohort

Timeline

• Start date: 2014-02-01
• Primary completion: 2014-04-01
• Completion: 2014-05-01
• First posted: 2012-12-11
• Last updated: 2014-11-24
• Results posted: 2014-11-24

Locations

17 sites across 1 country: United States