Safety and Pharmacokinetic Study of N6022 in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation
NCT ID: NCT01746784
Last Updated: 2014-11-24
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE1
66 participants
INTERVENTIONAL
2014-02-28
2014-05-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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N6022
Subjects randomized to study drug will receive N6022 by intravenous infusion once per day for 7 days
N6022
Intravenous solution of N6022 in normal saline administered by infusion pump over 1-8 minutes depending on the dose
Normal saline
Subjects randomized to placebo will receive normal saline administered intravenously using the same volume as the active drug group
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
Interventions
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N6022
Intravenous solution of N6022 in normal saline administered by infusion pump over 1-8 minutes depending on the dose
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 Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
18 Years
ALL
No
Sponsors
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Nivalis Therapeutics, Inc.
INDUSTRY
Responsible Party
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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
Providence Alaska Medical Center
Anchorage, Alaska, United States
Stanford University
Palo Alto, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
National Jewish Health
Denver, Colorado, United States
Northwestern University
Chicago, Illinois, United States
University of Iowa Children's Hospital
Iowa City, Iowa, United States
Johns Hopkins Hospital
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Minnesota
Minneapolis, Minnesota, United States
Washington University
St Louis, Missouri, United States
University of North Carolina
Chapel Hill, North Carolina, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, United States
Rainbow Babies and Children's Hospital - Case Medical Center
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Countries
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References
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Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
Related Links
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Home page of Sponsor
Other Identifiers
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N6022-1CF1-04
Identifier Type: -
Identifier Source: org_study_id