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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 October 2017 |
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Main ID: |
NCT01746784 |
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Date of registration:
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06/12/2012 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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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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Scientific title:
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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) |
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Date of first enrolment:
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February 2014 |
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Target sample size:
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66 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01746784 |
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Study type:
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Interventional |
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Study design:
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Phase:
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Phase 1
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Countries of recruitment
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United States
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Contacts
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Name:
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Scott Donaldson, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of North Carolina, Chapel Hill |
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Key inclusion & exclusion criteria
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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.
Age minimum:
18 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Cystic Fibrosis
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Intervention(s)
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Drug: N6022
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Drug: Normal saline
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Primary Outcome(s)
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Safety and Tolerability
[Time Frame: Over 7 treatment days and 7 days of follow-up]
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Secondary Outcome(s)
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Change in Biomarkers of CFTR Function
[Time Frame: Change from baseline at Day 7]
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Change in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)
[Time Frame: Change from baseline at Day 7]
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Secondary ID(s)
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N6022-1CF1-04
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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