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Safety and Pharmacokinetic Study of N6022 in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation (SNO-1)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01746784
Recruitment Status : Completed
First Posted : December 11, 2012
Results First Posted : November 24, 2014
Last Update Posted : November 24, 2014
Information provided by (Responsible Party):
Nivalis Therapeutics, Inc.

Brief Summary:
The purpose of this study is to investigate the safety, tolerability and pharmacokinetics of N6022, and to obtain descriptive information on the effect of N6022 on biomarkers of CFTR function and inflammation in adult cystic fibrosis subjects who are homozygous for the F508del-CFTR mutation.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: N6022 Drug: Normal saline Phase 1

Detailed Description:
This is a double-blind, randomized, placebo-controlled, multicenter, sequential dose-escalation study which will occur in two parts. All selection criteria, assessments and procedures described in this protocol will be applied to both parts. Up to 5 cohorts will be studied with a total of 67 patients at approximately 18 clinical sites in the United States.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 66 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1b, Randomized, Double-Blind, Placebo-Controlled, Dose Escalation Study of N6022 to Evaluate Safety and Pharmacokinetics in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation (SNO1)
Study Start Date : February 2014
Primary Completion Date : April 2014
Study Completion Date : May 2014

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
U.S. FDA Resources

Arm Intervention/treatment
Experimental: N6022
Subjects randomized to study drug will receive N6022 by intravenous infusion once per day for 7 days
Drug: N6022
Intravenous solution of N6022 in normal saline administered by infusion pump over 1-8 minutes depending on the dose
Other Name: GSNORi
Placebo Comparator: Normal saline
Subjects randomized to placebo will receive normal saline administered intravenously using the same volume as the active drug group
Drug: Normal saline
Intravenous solution of 0.9% (weight/volume) NaCl administered by infusion pump over 1-8 minutes depending on dose of active drug used in same cohort
Other Name: Placebo

Primary Outcome Measures :
  1. Safety and Tolerability [ Time Frame: Over 7 treatment days and 7 days of follow-up ]
    Assessments are based on numbers of subjects with abnormal clinical evaluations, abnormal laboratory assessments, and adverse events.

Secondary Outcome Measures :
  1. Change in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) [ Time Frame: Change from baseline at Day 7 ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson standards were used to calculate percent predicted FEV1 (for age, sex, and height).

  2. Change in Biomarkers of CFTR Function [ Time Frame: Change from baseline at Day 7 ]
    Sweat chloride millequivalents/Liter (mEq/L)

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Homozygous for F508del-CFTR gene
  • Sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis
  • Body weight ≥ 40 kg
  • FEV1 ≥ 40% predicted
  • Oxygen saturation ≥ 90% breathing ambient air
  • Hematology and clinical chemistry of blood and urine results with no clinically significant abnormalities that would interfere with the study assessments
  • Negative pregnancy test for women of child bearing potential
  • Sexually active subjects of child bearing potential willing to follow contraception requirements

Exclusion Criteria:

  • Previous enrollment in another cohort for this study.
  • Any acute infection, including acute upper or lower respiratory infections and pulmonary exacerbations that require treatment within 4 weeks of Study Day 1.
  • Any change in chronic therapies for CF lung disease within 4 weeks of Study Day 1.
  • 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) in three or more of the following: AST, ALT, GGT, ALP, total bilirubin at screening.
  • History of abnormal renal function (creatinine clearance < 50 mL/min using Cockcroft-Gault equation) within a year at screening.
  • History, including the screening assessment, of ventricular tachycardia or other ventricular arrhythmias.
  • History, including the screening assessment, of prolonged QT and/or QTcF interval (> 450 msec).
  • History of solid organ or hematological transplantation.
  • Intranasal medication changes within 14 days prior to Study Day 1
  • Required Use of continuous (24 hr/d) or nocturnal supplemental oxygen.
  • Concomitant use of any inhibitors or inducers of CYP3A4.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01746784

United States, Alabama
University of Alabama
Birmingham, Alabama, United States, 35294
United States, Alaska
Providence Alaska Medical Center
Anchorage, Alaska, United States, 99508
United States, California
Stanford University
Palo Alto, California, United States, 94304
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
National Jewish Health
Denver, Colorado, United States, 80206
United States, Illinois
Northwestern University
Chicago, Illinois, United States, 60611
United States, Iowa
University of Iowa Children's Hospital
Iowa City, Iowa, United States, 52242
United States, Maryland
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
United States, Missouri
Washington University
St. Louis, Missouri, United States, 63110
United States, North Carolina
University of North Carolina
Chapel Hill, North Carolina, United States, 27599
United States, Ohio
Cincinnati Children's Hospital
Cincinnati, Ohio, United States, 45229
Rainbow Babies and Children's Hospital - Case Medical Center
Cleveland, Ohio, United States, 44106
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
Sponsors and Collaborators
Nivalis Therapeutics, Inc.
Principal Investigator: Scott Donaldson, MD University of North Carolina, Chapel Hill

Additional Information:
Responsible Party: Nivalis Therapeutics, Inc. Identifier: NCT01746784     History of Changes
Other Study ID Numbers: N6022-1CF1-04
First Posted: December 11, 2012    Key Record Dates
Results First Posted: November 24, 2014
Last Update Posted: November 24, 2014
Last Verified: November 2014

Keywords provided by Nivalis Therapeutics, Inc.:
Cystic Fibrosis

Additional relevant MeSH terms:
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases