Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis F508del-CFTR N6022 S-Nitrosoglutathione
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 66
Healthy volunteers: no

Brief 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.

Arms:
arm 1: Subjects randomized to study drug will receive N6022 by intravenous infusion once per day for 7 days arm 2: Subjects randomized to placebo will receive normal saline administered intravenously using the same volume as the active drug group

Interventions: intervention 1: N6022 intervention 2: Normal saline
Intervention descriptions: intervention 1: Intravenous solution of N6022 in normal saline administered by infusion pump over 1-8 minutes depending on the dose intervention 2: 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

Protocol document: