Safety and Pharmacokinetic Study of N6022 in Subjects With Cystic Fibrosis
Homozygous for the F508del-CFTR Mutation
This study is currently recruiting participants.
Verified September 2013 by N30 Pharmaceuticals, Inc.
Sponsor:
N30 Pharmaceuticals, Inc.
Information provided by (Responsible Party):
N30 Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT01746784
First received: December 6, 2012
Last updated: September 16, 2013
Last verified: September 2013
History of Changes
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Tracking Information
First Received Date
Last Updated Date
Start Date
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December 6, 2012
September 16, 2013
January 2013
Estimated Primary
January 2014 (final data collection date for primary outcome
Completion Date
measure)
Current Primary Outcome
Safety and tolerability [ Time Frame: Over 7 treatment days and
Measures
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7 days of follow-up ] [ Designated as safety issue: No ]
(submitted: December 10,
2012)
Assessments are based on numbers of subjects with abnormal
clinical evaluations, abnormal laboratory assessments, and
adverse events.
Original Primary Outcome
Measures
Same as current
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Change History
Complete list of historical versions of study NCT01746784 on
ClinicalTrials.gov Archive Site
Current Secondary
Outcome Measures
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
Change in percent predicted forced expiratory volume in
1 second (FEV1) [ Time Frame: Change from baseline at
(submitted: December 10,
2012)
7 days ] [ Designated as safety issue: No ]

Change in biomarkers of CFTR function
[ Time Frame: Change from baseline at 7 days ]
[ Designated as safety issue: No ]
Original Secondary
Outcome Measures
Same as current
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Current Other Outcome
Measures ICMJE

Change in inflammatory biomarkers
[ Time Frame: Change from baseline at 7 days ]
(submitted: December 10,
[ Designated as safety issue: No ]
2012)
Measured in plasma, induced sputum and stool.

Changes in cystic fibrosis respiratory symptom diary
version 2 (CFRSD V2) [ Time Frame: Change from
baseline at 7 days ] [ Designated as safety issue: No ]

Patient global impression of change (PGIC)
[ Time Frame: Change from baseline at 7 days ]
[ Designated as safety issue: No ]
Original Other Outcome
Same as current
Measures ICMJE
Descriptive Information
Brief Title
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Safety and Pharmacokinetic Study of N6022 in Subjects With
Cystic Fibrosis Homozygous for the F508del-CFTR Mutation
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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
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.
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
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Study Phase
Study Design
Interventional
Phase 1
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Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator,
Outcomes Assessor)
Primary Purpose: Treatment
Condition
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Intervention
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Cystic Fibrosis

Drug: N6022
Intravenous solution of N6022 in normal saline
administered by infusion pump over 1-8 minutes
depending on the dose
Other Name: GSNORi

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
Study Arm (s)

Experimental: N6022
Subjects randomized to study drug will receive N6022 by
intravenous infusion once per day for 7 days
Intervention: Drug: N6022

Placebo Comparator: Normal saline
Subjects randomized to placebo will receive normal
saline administered intravenously using the same volume
as the active drug group
Intervention: Drug: Normal saline
Publications *
Not Provided
* Includes publications given by the data provider as well as publications
identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status
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Estimated Enrollment
Recruiting
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67
Estimated Completion Date
January 2014
Estimated Primary
January 2014 (final data collection date for primary outcome
Completion Date
measure)
Eligibility 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
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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.
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History, including the screening assessment, of ventricular
tachycardia or 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
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Required Use of continuous (24 hr/d) or nocturnal supplemental
oxygen.

Concomitant use of any inhibitors or inducers of CYP3A4.
Gender
Both
Ages
18 Years and older
Accepts Healthy
No
Volunteers
Contacts
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Contact:
720
Steven.Shoemaker@N30phar
Steven
-
ma.com
Shoemak
945
er, MD
770
0
ext
771
9
Location Countries
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United States
Administrative Information
NCT Number
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Other Study ID
Numbers
NCT01746784
N6022-1CF1-04
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Has Data Monitoring
Yes
Committee
Responsible Party
Study Sponsor
Collaborators
Investigators
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N30 Pharmaceuticals, Inc.
N30 Pharmaceuticals, Inc.
Not Provided
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Principal
Scott
University of
Investigator:
Donaldson,
North Carolina,
MD
Chapel Hill
Information Provided By
N30 Pharmaceuticals, Inc.
Verification Date
September 2013
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Data element required by the International Committee of Medical Journal
Editors and the World Health Organization ICTRP