Safety and Pharmacokinetic Study of N6022 in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation
This study is currently recruiting participants.
Verified June 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: June 4, 2013
Last verified: June 2013
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Purpose
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 | Intervention | Phase |
|---|---|---|
|
Cystic Fibrosis |
Drug: N6022 Drug: Normal saline |
Phase 1 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, 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 |
Resource links provided by NLM:
Genetics Home Reference related topics:
cystic fibrosis
MedlinePlus related topics:
Cystic Fibrosis
U.S. FDA Resources
Further study details as provided by N30 Pharmaceuticals, Inc.:
Primary Outcome Measures:
- Safety and tolerability [ Time Frame: Over 7 treatment days and 7 days of follow-up ] [ Designated as safety issue: No ]Assessments are based on numbers of subjects with abnormal clinical evaluations, abnormal laboratory assessments, and adverse events.
Secondary Outcome Measures:
- Change in percent predicted forced expiratory volume in 1 second (FEV1) [ Time Frame: Change from baseline at 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 ]
Other Outcome Measures:
- Change in inflammatory biomarkers [ Time Frame: Change from baseline at 7 days ] [ Designated as safety issue: No ]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 ]
| Estimated Enrollment: | 67 |
| Study Start Date: | January 2013 |
| Estimated Study Completion Date: | January 2014 |
| Estimated Primary Completion Date: | January 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
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
|
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.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
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 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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01746784
Contacts
| Contact: Steven Shoemaker, MD | 720-945-7700 ext 7719 | Steven.Shoemaker@N30pharma.com |
Locations
| United States, Alabama | |
| University of Alabama | Recruiting |
| Birmingham, Alabama, United States, 35294 | |
| Contact: Ginger Reeves, RRT 205-638-5970 greeves@peds.uab.edu | |
| Contact: Heather Hathorne, RRT 205-638-9568 hhathorne@peds.uab.edu | |
| Principal Investigator: Amit Gaggar, MD | |
| United States, Colorado | |
| Children's Hospital Colorado | Recruiting |
| Aurora, Colorado, United States, 80045 | |
| Contact: Churee Pardee, RN 720-777-6162 churee.pardee@childrenscolorado.org | |
| Contact: Carol Kopecky, RN 720-777-5416 carol.kopecky@childrenscolorado.org | |
| Principal Investigator: Edith Zemanick, MD | |
| National Jewish Health | Recruiting |
| Denver, Colorado, United States, 80206 | |
| Contact: Marion Jones, RN BSN 303-398-1265 jonesm@njhealth.org | |
| Contact: Connie St. Clair, RN BSN MS 303-270-2517 stclairc@njhealth.org | |
| Principal Investigator: Jennifer Taylor-Cousar, MD | |
| United States, Illinois | |
| Northwestern University | Recruiting |
| Chicago, Illinois, United States, 60611 | |
| Contact: Raquel Garcia-Cano, RN (312) 695-0415 raquel.garcia-cano@northwestern.edu | |
| Contact: Clare Marlin 312-695-2269 clare-murphy@northwestern.edu | |
| Principal Investigator: Manu Jain, MD, MSCI, FCCP | |
| United States, Maryland | |
| Johns Hopkins Hospital | Recruiting |
| Baltimore, Maryland, United States, 21287 | |
| Contact: Carolyn Chapman, RN 410-955-1167 cchapma7@jhmi.edu | |
| Contact: Karen Callahan, RN 410-955-1167 kcallah1@jhmi.edu | |
| Principal Investigator: Pamela Zeitlin, MD, PhD | |
| United States, Massachusetts | |
| Boston Children's Hospital | Recruiting |
| Boston, Massachusetts, United States, 02115 | |
| Contact: Amanda McGeachey, RN 617-355-6665 Amanda.mcgeachey@childrens.harvard.edu | |
| Contact: Robert Fowler 617-355-1834 Robert.fowler@childrens.harvard.edu | |
| Principal Investigator: Alicia Casey, MD | |
| United States, Minnesota | |
| University of Minnesota | Recruiting |
| Minneapolis, Minnesota, United States, 55455 | |
| Contact: Patricia Grover 612-626-7425 Grove026@umn.edu | |
| Contact: Denise Stacklie 612-626-9491 stack046@umn.edu | |
| Principal Investigator: Joanne Billings, MD | |
| United States, Missouri | |
| Washington University | Recruiting |
| St. Louis, Missouri, United States, 63110 | |
| Contact: Patty Burks, RN CCRA 314-454-5175 Burks_p@kids.wustl.edu | |
| Contact: Denise Rodgers 314-286-1176 rodgers@kids.wustl.edu | |
| Principal Investigator: Daniel Rosenbluth, MD | |
| United States, North Carolina | |
| University of North Carolina | Recruiting |
| Chapel Hill, North Carolina, United States, 27599 | |
| Contact: Nadia Bendahmane-Shive, RC 919-966-9198 nadia_bendahmane@med.unc.edu | |
| Contact: Claudia Salazar, RC (919) 966-9198 Claudia_salazar@med.unc.edu | |
| Principal Investigator: Scott Donaldson, MD | |
| United States, Ohio | |
| Cincinnati Children's Hospital | Recruiting |
| Cincinnati, Ohio, United States, 45229 | |
| Contact: Megan Bushman, RN 513-803-4325 Megan.Bushman@cchmc.org | |
| Contact: Delana Terrill, RRT 513-636-9780 Delana.terril@cchmc.org | |
| Principal Investigator: John P Clancy, MD | |
| Rainbow Babies and Children's Hospital - Case Medical Center | Recruiting |
| Cleveland, Ohio, United States, 44106 | |
| Contact: Bobbi Ksenich, RN CCRC 216-844-5733 Roberta.Ksenich@UHHospitals.org | |
| Contact: Dave Weaver 216-983-0469 David.Weaver@uhhospitals.org | |
| Principal Investigator: James Chmiel, MD | |
| Nationwide Children's Hospital | Recruiting |
| Columbus, Ohio, United States, 43205 | |
| Contact: Terri Johnson, RN BSN 614-722-4766 Terri.Johnson@nationwidechildrens.org | |
| Contact: Patty Olson, RC 614-722-4766 patti.olson@nationwidechildrens.org | |
| Principal Investigator: Karen McCoy, MD | |
Sponsors and Collaborators
N30 Pharmaceuticals, Inc.
Investigators
| Principal Investigator: | Scott Donaldson, MD | University of North Carolina, Chapel Hill |
More Information
Additional Information:
No publications provided
| Responsible Party: | N30 Pharmaceuticals, Inc. |
| ClinicalTrials.gov Identifier: | NCT01746784 History of Changes |
| Other Study ID Numbers: | N6022-1CF1-04 |
| Study First Received: | December 6, 2012 |
| Last Updated: | June 4, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by N30 Pharmaceuticals, Inc.:
|
Cystic Fibrosis F508del-CFTR N6022 S-Nitrosoglutathione |
Additional relevant MeSH terms:
|
Cystic Fibrosis Fibrosis Pancreatic Diseases Digestive System Diseases Lung Diseases |
Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Pathologic Processes |
ClinicalTrials.gov processed this record on June 18, 2013