Safety and Pharmacokinetic Study of N6022 in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation
SNO-1
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)
1 other identifier
interventional
66
1 country
17
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_1
Started Feb 2014
Shorter than P25 for phase_1
17 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 6, 2012
CompletedFirst Posted
Study publicly available on registry
December 11, 2012
CompletedStudy Start
First participant enrolled
February 1, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
May 1, 2014
CompletedResults Posted
Study results publicly available
November 24, 2014
CompletedNovember 24, 2014
November 1, 2014
2 months
December 6, 2012
November 17, 2014
November 21, 2014
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Safety and Tolerability
Assessments are based on numbers of subjects with abnormal clinical evaluations, abnormal laboratory assessments, and adverse events.
Over 7 treatment days and 7 days of follow-up
Secondary Outcomes (2)
Change in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)
Change from baseline at Day 7
Change in Biomarkers of CFTR Function
Change from baseline at Day 7
Study Arms (2)
N6022
EXPERIMENTALSubjects randomized to study drug will receive N6022 by intravenous infusion once per day for 7 days
Normal saline
PLACEBO COMPARATORSubjects randomized to placebo will receive normal saline administered intravenously using the same volume as the active drug group
Interventions
Intravenous solution of N6022 in normal saline administered by infusion pump over 1-8 minutes depending on the dose
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
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (17)
University of Alabama
Birmingham, Alabama, 35294, United States
Providence Alaska Medical Center
Anchorage, Alaska, 99508, United States
Stanford University
Palo Alto, California, 94304, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
National Jewish Health
Denver, Colorado, 80206, United States
Northwestern University
Chicago, Illinois, 60611, United States
University of Iowa Children's Hospital
Iowa City, Iowa, 52242, United States
Johns Hopkins Hospital
Baltimore, Maryland, 21287, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Washington University
St Louis, Missouri, 63110, United States
University of North Carolina
Chapel Hill, North Carolina, 27599, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
Rainbow Babies and Children's Hospital - Case Medical Center
Cleveland, Ohio, 44106, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Related Publications (2)
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.
PMID: 37983082DERIVEDSouthern 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.
PMID: 33331662DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Dr. Steven Shoemaker
- Organization
- N30 Pharmaceuticals
Study Officials
- PRINCIPAL INVESTIGATOR
Scott Donaldson, MD
University of North Carolina, Chapel Hill
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- GT60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 6, 2012
First Posted
December 11, 2012
Study Start
February 1, 2014
Primary Completion
April 1, 2014
Study Completion
May 1, 2014
Last Updated
November 24, 2014
Results First Posted
November 24, 2014
Record last verified: 2014-11