Study of Cavosonstat (N91115) in Patients With CF Homozygous for the F508del-CFTR Mutation

• Status: Completed
• Trial end date: 2016-12-01
• Target enrollment: 0
• Participant gender: All

Summary

This will be a double-blind, randomized, placebo-controlled, parallel group study. The purpose of this study is to investigate the efficacy and safety of Cavosonstat (N91115) in adult patients with CF who are homozygous for the F508del-CFTR mutation and being treated with lumacaftor/ivacaftor (Orkambi™).

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

Details

• Lead Sponsor: Nivalis Therapeutics, Inc.
• Collaborator: Medidata Solutions

Criteria

Inclusion Criteria:

• Patients must have been treated with lumacaftor/ivacaftor for at least 8 weeks prior to Day 1 (start of dosing)

• A history of Sweat Chloride (SC) ≥ 60 mEq/L by quantitative pilocarpine iontophoresis test (QPIT) (either before or after starting lumacaftor/ivacaftor treatment)

• Body weight ≥ 40 kg

• ppFEV1 40 - 85 % predicted (inclusive) at screening

• Oxygen saturation ≥ 90% breathing ambient air at screening

Exclusion Criteria:

• Any acute infection that requires treatment or hospitalization within 2 weeks of Study Day 1

• Colonization with organisms associated with more rapid decline in pulmonary status, such as Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus

• Any change in the regimen for chronic therapies for CF lung disease (e.g., Pulmozyme®, hypertonic saline, Azithromycin, TOBI®, Cayston®) within 4 weeks of Study Day 1

• Are pregnant, planning a pregnancy, or breast-feeding at screening

• Blood hemoglobin < 10 g/dL at screening

• Serum albumin < 2.5 g/dL at screening

• Abnormal liver function defined as ≥ 3 x upper limit of normal (ULN)

• History of abnormal renal function within 3 months of screening

• History of ventricular tachycardia or other clinically significant ventricular arrhythmias

• History, including the screening assessment, of prolonged QT and/or QTcF (Fridericia's correction) interval

• History of solid organ or hematological transplantation

• History of alcohol abuse or drug abuse

• Ongoing participation in another therapeutic clinical trial

• Use of continuous (24 hr/day) or nocturnal supplemental oxygen