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Trial record 1 of 1 for:    NCT02589236
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Study of Cavosonstat (N91115) in Patients With CF Homozygous for the F508del-CFTR Mutation (SNO-6)

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02589236
First Posted: October 28, 2015
Last Update Posted: January 10, 2017
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.
Collaborator:
Medidata Solutions
Information provided by (Responsible Party):
Nivalis Therapeutics, Inc.
  Purpose
This will be a double-blind, randomized, placebo-controlled, parallel group study. The purpose of this study is to investigate the efficacy and safety of Cavosonstat (N91115) in adult patients with CF who are homozygous for the F508del-CFTR mutation and being treated with lumacaftor/ivacaftor (Orkambi™).

Condition Intervention Phase
Cystic Fibrosis Drug: Cavosonstat Drug: Placebo Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of N91115 to Evaluate Efficacy and Safety in Patients With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation Treated With Lumacaftor/Ivacaftor

Resource links provided by NLM:


Further study details as provided by Nivalis Therapeutics, Inc.:

Primary Outcome Measures:
  • Absolute change from baseline in percent predicted FEV1 (ppFEV1) [ Time Frame: From baseline to 12 weeks ]
    Forced Expiratory Volume in one second (FEV1) from before study (Baseline) to after 12 weeks of N91115 treatment


Secondary Outcome Measures:
  • Relative change from baseline in ppFEV1 [ Time Frame: baseline to 12 weeks ]
    Forced Expiratory Volume in one second (FEV1) from before study (Baseline) to after 12 weeks of N91115 treatment

  • Absolute change from baseline in sweat chloride [ Time Frame: baseline to 12 weeks ]
    A sweat chloride measurement on the skin at study start and after 12 weeks of N91115

  • Absolute change from baseline in Cystic Fibrosis Questionnaire -Revised CFQ-R (respiratory symptom scale) [ Time Frame: baseline to 16 weeks ]
    Comparison of the Questionnaire from study start to 16 weeks

  • Absolute change from baseline in body mass index (BMI) [ Time Frame: baseline to 12 weeks ]
    Assessment of change in body mass index from study start to after 12 weeks of N91115

  • Absolute change from baseline in Patient Global Impression of Change (PGIC) [ Time Frame: baseline to 12 weeks ]
    Patient reported outcome journal

  • Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]) [ Time Frame: baseline to 16 weeks ]
    Any adverse events assessment including clinical laboratory values, electrocardiogram (ECG), pulmonary exacerbations, or vital sign changes

  • Pharmacokinetic Measurements of Maximum Plasma Concentration [Cmax], of N91115, lumacaftor, and ivacaftor [ Time Frame: baseline to 12 weeks ]
    Maximum Plasma Concentration [Cmax] measurements of N91115, lumacaftor and ivacaftor

  • Pharmacokinetic Measurements of Area Under the Curve (AUC) for N91115, Ivacaftor and lumacaftor [ Time Frame: baseline to 12 weeks ]
    AUC measurements of N91115, lumacaftor and ivacaftor


Other Outcome Measures:
  • Number of pulmonary exacerbations [ Time Frame: baseline to 12 weeks ]
    Assessment of number of pulmonary exacerbations at baseline compared through 12 weeks


Enrollment: 138
Study Start Date: November 2015
Study Completion Date: December 2016
Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Placebo
Placebo Capsule
Drug: Placebo
Control sample with only capsule excipients and fillers
Other Name: control
Experimental: Cavosonstat (N91115) 200 mg
Cavosonstat (N91115) 200 mg twice daily (BID)
Drug: Cavosonstat
GSNOR inhibitor
Other Name: N91115
Experimental: Cavosonstat (N91115) 400 mg
Cavosonstat (N91115) 400 mg BID
Drug: Cavosonstat
GSNOR inhibitor
Other Name: N91115

Detailed Description:

Primary Objective:

  • Assess the efficacy of N91115 at 12 weeks when added to preexisting treatment with lumacaftor/ivacaftor in adult patients with CF who are homozygous for the F508del-CFTR mutation

Secondary Objectives:

  • Assess the effect of N91115 added to lumacaftor/ivacaftor on safety
  • Assess the effect of lumacaftor/ivacaftor added to N91115 on the pharmacokinetics of N91115, lumacaftor, and ivacaftor
  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients must have been treated with lumacaftor/ivacaftor for at least 8 weeks prior to Day 1 (start of dosing)
  • A history of Sweat Chloride (SC) ≥ 60 mEq/L by quantitative pilocarpine iontophoresis test (QPIT) (either before or after starting lumacaftor/ivacaftor treatment)
  • Body weight ≥ 40 kg
  • ppFEV1 40 - 85 % predicted (inclusive) at screening
  • Oxygen saturation ≥ 90% breathing ambient air at screening

Exclusion Criteria:

  • Any acute infection that requires treatment or hospitalization within 2 weeks of Study Day 1
  • Colonization with organisms associated with more rapid decline in pulmonary status, such as Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus
  • Any change in the regimen for chronic therapies for CF lung disease (e.g., Pulmozyme®, hypertonic saline, Azithromycin, TOBI®, Cayston®) within 4 weeks of Study Day 1
  • Are pregnant, planning a pregnancy, or breast-feeding at screening
  • 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)
  • History of abnormal renal function within 3 months of screening
  • History of ventricular tachycardia or other clinically significant ventricular arrhythmias
  • History, including the screening assessment, of prolonged QT and/or QTcF (Fridericia's correction) interval
  • History of solid organ or hematological transplantation
  • History of alcohol abuse or drug abuse
  • Ongoing participation in another therapeutic clinical trial
  • Use of continuous (24 hr/day) or nocturnal supplemental oxygen
  Contacts and Locations
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): NCT02589236


  Show 46 Study Locations
Sponsors and Collaborators
Nivalis Therapeutics, Inc.
Medidata Solutions
Investigators
Principal Investigator: Scott Donaldson, MD University of North Carolina, Chapel Hill
  More Information

Responsible Party: Nivalis Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT02589236     History of Changes
Other Study ID Numbers: N91115-2CF-05
First Submitted: October 26, 2015
First Posted: October 28, 2015
Last Update Posted: January 10, 2017
Last Verified: January 2017

Keywords provided by Nivalis Therapeutics, Inc.:
Cystic Fibrosis
N91115
Cavosonstat

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases