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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)

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: NCT02589236
Recruitment Status : Completed
First Posted : October 28, 2015
Last Update Posted : January 10, 2017
Sponsor:
Collaborator:
Medidata Solutions
Information provided by (Responsible Party):
Nivalis Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE October 26, 2015
First Posted Date  ICMJE October 28, 2015
Last Update Posted Date January 10, 2017
Study Start Date  ICMJE November 2015
Actual Primary Completion Date December 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 26, 2015)
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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 26, 2015)
  • 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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: October 26, 2015)
Number of pulmonary exacerbations [ Time Frame: baseline to 12 weeks ]
Assessment of number of pulmonary exacerbations at baseline compared through 12 weeks
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Study of Cavosonstat (N91115) in Patients With CF Homozygous for the F508del-CFTR Mutation
Official Title  ICMJE 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
Brief Summary 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™).
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
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • Drug: Cavosonstat
    GSNOR inhibitor
    Other Name: N91115
  • Drug: Placebo
    Control sample with only capsule excipients and fillers
    Other Name: control
Study Arms  ICMJE
  • Placebo Comparator: Placebo
    Placebo Capsule
    Intervention: Drug: Placebo
  • Experimental: Cavosonstat (N91115) 200 mg
    Cavosonstat (N91115) 200 mg twice daily (BID)
    Intervention: Drug: Cavosonstat
  • Experimental: Cavosonstat (N91115) 400 mg
    Cavosonstat (N91115) 400 mg BID
    Intervention: Drug: Cavosonstat
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  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 27, 2016)
138
Original Estimated Enrollment  ICMJE
 (submitted: October 26, 2015)
135
Actual Study Completion Date  ICMJE December 2016
Actual Primary Completion Date December 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02589236
Other Study ID Numbers  ICMJE N91115-2CF-05
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Nivalis Therapeutics, Inc.
Study Sponsor  ICMJE Nivalis Therapeutics, Inc.
Collaborators  ICMJE Medidata Solutions
Investigators  ICMJE
Principal Investigator: Scott Donaldson, MD University of North Carolina, Chapel Hill
PRS Account Nivalis Therapeutics, Inc.
Verification Date January 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP