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

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ClinicalTrials.gov 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.

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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
Actual Primary Completion Date : April 2014
Actual Study Completion Date : May 2014

Resource links provided by the National Library of Medicine

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, Older Adult)
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 ClinicalTrials.gov identifier (NCT number): NCT01746784

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Sponsors and Collaborators
Nivalis Therapeutics, Inc.
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Principal Investigator: Scott Donaldson, MD University of North Carolina, Chapel Hill
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Nivalis Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT01746784    
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:
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Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases