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

NCT ID: NCT02589236

Last Updated: 2017-01-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

138 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-11-30

Study Completion Date

2016-12-31

Brief Summary

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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™).

Detailed Description

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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

Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Placebo

Placebo Capsule

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Control sample with only capsule excipients and fillers

Cavosonstat (N91115) 200 mg

Cavosonstat (N91115) 200 mg twice daily (BID)

Group Type EXPERIMENTAL

Cavosonstat

Intervention Type DRUG

GSNOR inhibitor

Cavosonstat (N91115) 400 mg

Cavosonstat (N91115) 400 mg BID

Group Type EXPERIMENTAL

Cavosonstat

Intervention Type DRUG

GSNOR inhibitor

Interventions

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Cavosonstat

GSNOR inhibitor

Intervention Type DRUG

Placebo

Control sample with only capsule excipients and fillers

Intervention Type DRUG

Other Intervention Names

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N91115 control

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Medidata Solutions

INDUSTRY

Sponsor Role collaborator

Nivalis Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Scott Donaldson, MD

Role: PRINCIPAL_INVESTIGATOR

University of North Carolina, Chapel Hill

Locations

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University of Alabama

Birmingham, Alabama, United States

Site Status

Banner University of Arizona Medical Center

Tucson, Arizona, United States

Site Status

Children's Hospital Los Angeles

Los Angeles, California, United States

Site Status

Stanford University

Palo Alto, California, United States

Site Status

UC Davis Medical Center

Sacramento, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

National Jewish Health

Denver, Colorado, United States

Site Status

Yale University

New Haven, Connecticut, United States

Site Status

Tampa General Hospital

Tampa, Florida, United States

Site Status

St. Luke's CF Center of Idaho

Boise, Idaho, United States

Site Status

Northwestern University

Chicago, Illinois, United States

Site Status

Saint Francis Medical Center

Peoria, Illinois, United States

Site Status

Indiana University

Indianapolis, Indiana, United States

Site Status

University of Iowa Children's Hospital

Iowa City, Iowa, United States

Site Status

Via Christi Research

Wichita, Kansas, United States

Site Status

University of Kentucky

Lexington, Kentucky, United States

Site Status

Maine Medical Center

Portland, Maine, United States

Site Status

Johns Hopkins Hospital

Baltimore, Maryland, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

University of Massachusetts Medical School

Worcester, Massachusetts, United States

Site Status

Wayne State University-Harper

Detroit, Michigan, United States

Site Status

Spectrum Health Butterworth Hospital

Grand Rapids, Michigan, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

St. Louis University

St Louis, Missouri, United States

Site Status

Washington University

St Louis, Missouri, United States

Site Status

University of Nebraska Medical Center

Omaha, Nebraska, United States

Site Status

Morristown Medical Center

Morristown, New Jersey, United States

Site Status

Columbia University

New York, New York, United States

Site Status

University of North Carolina Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Wake Forest University School of Medicine

Winston-Salem, North Carolina, United States

Site Status

Children's Hospital Medical Center of Akron

Akron, Ohio, United States

Site Status

Cincinnati Children's Hospital

Cincinnati, Ohio, United States

Site Status

Rainbow Babies and Children's Hospital - Case Medical Center

Cleveland, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

ProMedica Toledo Children's Hospital

Toledo, Ohio, United States

Site Status

Oregon Health and Science University

Portland, Oregon, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Dell Children's Hospital | Austin Children's Chest Associates

Austin, Texas, United States

Site Status

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status

Cook Children's Medical Center

Fort Worth, Texas, United States

Site Status

Texas Children's Hospital

Houston, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

Medical Center of Wisconsin

Madison, Wisconsin, United States

Site Status

Countries

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United States

Other Identifiers

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N91115-2CF-05

Identifier Type: -

Identifier Source: org_study_id

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