Overview

This trial is active, not recruiting.

Condition cystic fibrosis
Treatments cavosonstat, placebo
Phase phase 2
Sponsor Nivalis Therapeutics, Inc.
Collaborator Medidata Solutions
Start date November 2015
End date December 2016
Trial size 138 participants
Trial identifier NCT02589236, N91115-2CF-05

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™).

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator)
Primary purpose treatment
Arm
(Placebo Comparator)
Placebo Capsule
placebo control
Control sample with only capsule excipients and fillers
(Experimental)
Cavosonstat (N91115) 200 mg twice daily (BID)
cavosonstat N91115
GSNOR inhibitor
(Experimental)
Cavosonstat (N91115) 400 mg BID
cavosonstat N91115
GSNOR inhibitor

Primary Outcomes

Measure
Absolute change from baseline in percent predicted FEV1 (ppFEV1)
time frame: From baseline to 12 weeks

Secondary Outcomes

Measure
Relative change from baseline in ppFEV1
time frame: baseline to 12 weeks
Absolute change from baseline in sweat chloride
time frame: baseline to 12 weeks
Absolute change from baseline in Cystic Fibrosis Questionnaire -Revised CFQ-R (respiratory symptom scale)
time frame: baseline to 16 weeks
Absolute change from baseline in body mass index (BMI)
time frame: baseline to 12 weeks
Absolute change from baseline in Patient Global Impression of Change (PGIC)
time frame: baseline to 12 weeks
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability])
time frame: baseline to 16 weeks
Pharmacokinetic Measurements of Maximum Plasma Concentration [Cmax], of N91115, lumacaftor, and ivacaftor
time frame: baseline to 12 weeks
Pharmacokinetic Measurements of Area Under the Curve (AUC) for N91115, Ivacaftor and lumacaftor
time frame: baseline to 12 weeks

Eligibility Criteria

Male or female participants at least 18 years old.

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

Additional Information

Official title A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of N91115 to Evaluate Efficacy and Safety in Patients With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation Treated With Lumacaftor/Ivacaftor
Principal investigator Scott Donaldson, MD
Description Primary Objective: - Assess the efficacy of N91115 at 12 weeks when added to preexisting treatment with lumacaftor/ivacaftor in adult patients with CF who are homozygous for the F508del-CFTR mutation Secondary Objectives: - Assess the effect of N91115 added to lumacaftor/ivacaftor on safety - Assess the effect of lumacaftor/ivacaftor added to N91115 on the pharmacokinetics of N91115, lumacaftor, and ivacaftor
Trial information was received from ClinicalTrials.gov and was last updated in November 2016.
Information provided to ClinicalTrials.gov by Nivalis Therapeutics, Inc..