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Study of N91115 in Patients With Cystic Fibrosis Homozygous F508del-CFTR Mutation (SNO4)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02275936
Recruitment Status : Completed
First Posted : October 27, 2014
Last Update Posted : November 6, 2016
Sponsor:
Information provided by (Responsible Party):
Nivalis Therapeutics, Inc.

Brief Summary:
This Phase 1b study in F508del-CFTR homozygous CF patients is being conducted to assess the safety of N91115 as the sole cystic fibrosis transmembrane conductance regulator (CFTR) modulator at doses near the expected therapeutic exposure level in preparation for Phase 2 studies of N91115 added to the CFTR modulator combination lumacaftor/ivacaftor when launched.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: N91115 Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 51 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1b, Randomized, Double-Blind, Placebo-Controlled, Parallel, Group Study of N91115 to Evaluate Safety and Pharmacokinetics in Patients With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation
Study Start Date : February 2015
Actual Primary Completion Date : July 2015
Actual Study Completion Date : July 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: Group 1 - 50 mg
Every 12 hour oral dosing of N91115 for 28 days
Drug: N91115
S Nitrosoglutathione Reductase Inhibitor
Other Name: Cavosonstat

Experimental: Group 2 - 100 mg
Every 12 hour oral dosing of N91115 for 28 days
Drug: N91115
S Nitrosoglutathione Reductase Inhibitor
Other Name: Cavosonstat

Experimental: Group 3 - 200 mg
Every 12 hour oral dosing of N91115 for 28 days
Drug: N91115
S Nitrosoglutathione Reductase Inhibitor
Other Name: Cavosonstat

Placebo Comparator: Group 4 - Placebo
Every 12 hour oral dosing of placebo comparator for 28 days
Drug: N91115
S Nitrosoglutathione Reductase Inhibitor
Other Name: Cavosonstat




Primary Outcome Measures :
  1. Safety assessments based on clinical evaluations, laboratory assessments, and adverse events. [ Time Frame: 28 Days ]

Secondary Outcome Measures :
  1. Pharmacokinetic (PK) parameters of N91115 and its glucuronide metabolite in plasma [ Time Frame: 28 Days ]
    Area under the curve(AUC) assessments

  2. Pharmacokinetic (PK) parameters of N91115 and its glucuronide metabolite in plasma [ Time Frame: 28 Days ]
    Maximum plasma concentration (Cmax) determinations

  3. Pharmacokinetic (PK) parameters of N91115 and its glucuronide metabolite in plasma [ Time Frame: 28 Days ]
    Ratio of parent:glucuronide metabolite



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male or female, age ≥ 18 years with confirmed diagnosis of CF, homozygous for the F508del-CFTR mutation based on historical results generated by Ambry Genetics within the past two years or if unavailable, confirmed by testing done within the past 28 days
  2. Sweat chloride ≥ 60 (milliequivalents) mEq/L, by quantitative pilocarpine iontophoresis test (QPIT) at screening
  3. Weight ≥ 40 kg at screening
  4. Forced expiratory volume (FEV1) ≥ 40% of predicted normal for age, gender, and height (Hankinson standards) pre- or post-bronchodilator value, at screening
  5. Oxygen saturation by pulse oximetry ≥ 90% breathing ambient air, at screening
  6. Hematology, clinical chemistry and urinalysis results with no clinically significant abnormalities that would interfere with the study assessments at screening

Exclusion Criteria:

  1. Any acute infection, including acute upper or lower respiratory infections and pulmonary exacerbations that require treatment or hospitalization within 2 weeks of Study Day 1
  2. Any change in chronic therapies for CF lung disease (e.g., Ibuprofen, Pulmozyme®, hypertonic saline, Azithromycin, Tobi®, Cayston®) within 4 weeks of Study Day 1
  3. Blood hemoglobin < 10 g/dL at screening
  4. Serum albumin < 2.5 g/dL at screening
  5. Abnormal liver function defined as ≥ 3 x upper limit of normal (ULN) in 3 or more of the following: aspartate aminotransferase (AST), alanine aminotransferase (ALT), g-glutamyl transferase (GGT), alkaline phosphatase (ALP), or total bilirubin at screening
  6. History of abnormal renal function (creatinine clearance < 50 mL/min using Cockcroft-Gault equation) within a year of screening
  7. History, including the screening assessment, of ventricular tachycardia or other ventricular arrhythmias
  8. History, including the screening assessment, of prolonged cardiac QT interval and/or QTcF (QT with Fridericia's correction) interval (> 450 msec)
  9. History of solid organ or hematological transplantation
  10. History of alcohol abuse or drug abuse (including cannabis, cocaine, and opioids) in the year prior to screening
  11. Use of continuous (24 hr/day) or nocturnal supplemental oxygen

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02275936


Locations
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Sponsors and Collaborators
Nivalis Therapeutics, Inc.
Investigators
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Principal Investigator: Scott Donaldson, MD University of North Carolina
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Nivalis Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT02275936    
Other Study ID Numbers: N91115-1CF-03
First Posted: October 27, 2014    Key Record Dates
Last Update Posted: November 6, 2016
Last Verified: November 2016
Keywords provided by Nivalis Therapeutics, Inc.:
N91115
Cavosonstat
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases