Study of Cavosonstat (N91115) in Patients With CF Homozygous for the F508del-CFTR Mutation
SNO-6
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
1 other identifier
interventional
138
1 country
46
Brief 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™).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Nov 2015
Shorter than P25 for phase_2
46 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 26, 2015
CompletedFirst Posted
Study publicly available on registry
October 28, 2015
CompletedStudy Start
First participant enrolled
November 1, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2016
CompletedJanuary 10, 2017
January 1, 2017
1.1 years
October 26, 2015
January 6, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Absolute change from baseline in percent predicted FEV1 (ppFEV1)
Forced Expiratory Volume in one second (FEV1) from before study (Baseline) to after 12 weeks of N91115 treatment
From baseline to 12 weeks
Secondary Outcomes (8)
Relative change from baseline in ppFEV1
baseline to 12 weeks
Absolute change from baseline in sweat chloride
baseline to 12 weeks
Absolute change from baseline in Cystic Fibrosis Questionnaire -Revised CFQ-R (respiratory symptom scale)
baseline to 16 weeks
Absolute change from baseline in body mass index (BMI)
baseline to 12 weeks
Absolute change from baseline in Patient Global Impression of Change (PGIC)
baseline to 12 weeks
- +3 more secondary outcomes
Other Outcomes (1)
Number of pulmonary exacerbations
baseline to 12 weeks
Study Arms (3)
Placebo
PLACEBO COMPARATORPlacebo Capsule
Cavosonstat (N91115) 200 mg
EXPERIMENTALCavosonstat (N91115) 200 mg twice daily (BID)
Cavosonstat (N91115) 400 mg
EXPERIMENTALCavosonstat (N91115) 400 mg BID
Interventions
GSNOR inhibitor
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Nivalis Therapeutics, Inc.lead
- Medidata Solutionscollaborator
Study Sites (46)
University of Alabama
Birmingham, Alabama, 35294, United States
Banner University of Arizona Medical Center
Tucson, Arizona, 85724, United States
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
Stanford University
Palo Alto, California, 94304, United States
UC Davis Medical Center
Sacramento, California, 95817, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
National Jewish Health
Denver, Colorado, 80206, United States
Yale University
New Haven, Connecticut, 06510, United States
Tampa General Hospital
Tampa, Florida, 33606, United States
St. Luke's CF Center of Idaho
Boise, Idaho, 83712, United States
Northwestern University
Chicago, Illinois, 60611, United States
Saint Francis Medical Center
Peoria, Illinois, 61637, United States
Indiana University
Indianapolis, Indiana, 46202, United States
University of Iowa Children's Hospital
Iowa City, Iowa, 52242, United States
Via Christi Research
Wichita, Kansas, 67214, United States
University of Kentucky
Lexington, Kentucky, 40536, United States
Maine Medical Center
Portland, Maine, 04102, United States
Johns Hopkins Hospital
Baltimore, Maryland, 21287, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
University of Massachusetts Medical School
Worcester, Massachusetts, 01655, United States
Wayne State University-Harper
Detroit, Michigan, 48202, United States
Spectrum Health Butterworth Hospital
Grand Rapids, Michigan, 49503, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
St. Louis University
St Louis, Missouri, 63104, United States
Washington University
St Louis, Missouri, 63110, United States
University of Nebraska Medical Center
Omaha, Nebraska, 68198, United States
Morristown Medical Center
Morristown, New Jersey, 07960, United States
Columbia University
New York, New York, 10032, United States
University of North Carolina Chapel Hill
Chapel Hill, North Carolina, 27599, United States
Wake Forest University School of Medicine
Winston-Salem, North Carolina, 27103, United States
Children's Hospital Medical Center of Akron
Akron, Ohio, 44308, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
Rainbow Babies and Children's Hospital - Case Medical Center
Cleveland, Ohio, 44106, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
ProMedica Toledo Children's Hospital
Toledo, Ohio, 43606, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Dell Children's Hospital | Austin Children's Chest Associates
Austin, Texas, 78723, United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
University of Utah
Salt Lake City, Utah, 84132, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Medical Center of Wisconsin
Madison, Wisconsin, 53792, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Scott Donaldson, MD
University of North Carolina, Chapel Hill
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 26, 2015
First Posted
October 28, 2015
Study Start
November 1, 2015
Primary Completion
December 1, 2016
Study Completion
December 1, 2016
Last Updated
January 10, 2017
Record last verified: 2017-01