Study of Cavosonstat (N91115) in Patients With CF Homozygous for the F508del-CFTR Mutation (SNO-6)

January 6, 2017 updated by: Nivalis Therapeutics, Inc.

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

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™).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed 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

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
138

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • University of Alabama
    • Arizona
      • Tucson, Arizona, United States, 85724
        • Banner University of Arizona Medical Center
    • California
      • Los Angeles, California, United States, 90027
        • Children's Hospital Los Angeles
      • Palo Alto, California, United States, 94304
        • Stanford University
      • Sacramento, California, United States, 95817
        • UC Davis Medical Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
      • Denver, Colorado, United States, 80206
        • National Jewish Health
    • Connecticut
      • New Haven, Connecticut, United States, 06510
        • Yale University
    • Florida
      • Tampa, Florida, United States, 33606
        • Tampa General Hospital
    • Idaho
      • Boise, Idaho, United States, 83712
        • St. Luke's CF Center of Idaho
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern University
      • Peoria, Illinois, United States, 61637
        • Saint Francis Medical Center
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Children's Hospital
    • Kansas
      • Wichita, Kansas, United States, 67214
        • Via Christi Research
    • Kentucky
      • Lexington, Kentucky, United States, 40536
        • University of Kentucky
    • Maine
      • Portland, Maine, United States, 04102
        • Maine Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Johns Hopkins Hospital
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
      • Worcester, Massachusetts, United States, 01655
        • University of Massachusetts Medical School
    • Michigan
      • Detroit, Michigan, United States, 48202
        • Wayne State University-Harper
      • Grand Rapids, Michigan, United States, 49503
        • Spectrum Health Butterworth Hospital
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota
    • Missouri
      • St. Louis, Missouri, United States, 63110
        • Washington University
      • St. Louis, Missouri, United States, 63104
        • St. Louis University
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • University of Nebraska Medical Center
    • New Jersey
      • Morristown, New Jersey, United States, 07960
        • Morristown Medical Center
    • New York
      • New York, New York, United States, 10032
        • Columbia University
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • University of North Carolina Chapel Hill
      • Winston-Salem, North Carolina, United States, 27103
        • Wake Forest University School of Medicine
    • Ohio
      • Akron, Ohio, United States, 44308
        • Children's Hospital Medical Center of Akron
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital
      • Cleveland, Ohio, United States, 44106
        • Rainbow Babies and Children's Hospital - Case Medical Center
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
      • Toledo, Ohio, United States, 43606
        • ProMedica Toledo Children's Hospital
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children's Hospital of Pittsburgh of UPMC
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina
    • Texas
      • Austin, Texas, United States, 78723
        • Dell Children's Hospital | Austin Children's Chest Associates
      • Dallas, Texas, United States, 75390
        • University of Texas Southwestern Medical Center
      • Fort Worth, Texas, United States, 76104
        • Cook Children's Medical Center
      • Houston, Texas, United States, 77030
        • Texas Children's Hospital
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • University of Utah
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • Medical Center of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: TRIPLE

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
PLACEBO_COMPARATOR: Placebo
Placebo Capsule
Drug: Placebo
Control sample with only capsule excipients and fillers
Other Names:
  • control
EXPERIMENTAL: Cavosonstat (N91115) 200 mg
Cavosonstat (N91115) 200 mg twice daily (BID)
Drug: Cavosonstat
GSNOR inhibitor
Other Names:
  • N91115
EXPERIMENTAL: Cavosonstat (N91115) 400 mg
Cavosonstat (N91115) 400 mg BID
Drug: Cavosonstat
GSNOR inhibitor
Other Names:
  • N91115

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Absolute change from baseline in percent predicted FEV1 (ppFEV1)
Time Frame: From baseline to 12 weeks
Forced Expiratory Volume in one second (FEV1) from before study (Baseline) to after 12 weeks of N91115 treatment
From baseline to 12 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Relative change from baseline in ppFEV1
Time Frame: baseline to 12 weeks
Forced Expiratory Volume in one second (FEV1) from before study (Baseline) to after 12 weeks of N91115 treatment
baseline to 12 weeks
Absolute change from baseline in sweat chloride
Time Frame: baseline to 12 weeks
A sweat chloride measurement on the skin at study start and after 12 weeks of N91115
baseline to 12 weeks
Absolute change from baseline in Cystic Fibrosis Questionnaire -Revised CFQ-R (respiratory symptom scale)
Time Frame: baseline to 16 weeks
Comparison of the Questionnaire from study start to 16 weeks
baseline to 16 weeks
Absolute change from baseline in body mass index (BMI)
Time Frame: baseline to 12 weeks
Assessment of change in body mass index from study start to after 12 weeks of N91115
baseline to 12 weeks
Absolute change from baseline in Patient Global Impression of Change (PGIC)
Time Frame: baseline to 12 weeks
Patient reported outcome journal
baseline to 12 weeks
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability])
Time Frame: baseline to 16 weeks
Any adverse events assessment including clinical laboratory values, electrocardiogram (ECG), pulmonary exacerbations, or vital sign changes
baseline to 16 weeks
Pharmacokinetic Measurements of Maximum Plasma Concentration [Cmax], of N91115, lumacaftor, and ivacaftor
Time Frame: baseline to 12 weeks
Maximum Plasma Concentration [Cmax] measurements of N91115, lumacaftor and ivacaftor
baseline to 12 weeks
Pharmacokinetic Measurements of Area Under the Curve (AUC) for N91115, Ivacaftor and lumacaftor
Time Frame: baseline to 12 weeks
AUC measurements of N91115, lumacaftor and ivacaftor
baseline to 12 weeks

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Number of pulmonary exacerbations
Time Frame: baseline to 12 weeks
Assessment of number of pulmonary exacerbations at baseline compared through 12 weeks
baseline to 12 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Nivalis Therapeutics, Inc.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Medidata Solutions

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 1, 2015

Primary Completion (ACTUAL)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2016

Study Completion (ACTUAL)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2016

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 26, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 26, 2015

First Posted (ESTIMATE)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 28, 2015

Study Record Updates

Last Update Posted (ESTIMATE)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 10, 2017

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 6, 2017

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • N91115-2CF-05

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Cystic Fibrosis

Clinical Trials on Placebo

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings