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Completed

NCT Number: NCT02589236

Study of Cavosonstat (N91115) in Patients With CF Homozygous for the F508del-CFTR Mutation

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™).

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University of Alabama, Birmingham, Alabama, United States

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About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Cavosonstat

Drug

GSNOR inhibitor

Other names: N91115

Placebo

Drug

Control sample with only capsule excipients and fillers

Other names: control

Primary outcomes

  1. 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 outcomes

  1. 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

  2. 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

  3. 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

  4. 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

  5. Absolute change from baseline in Patient Global Impression of Change (PGIC)

    Time frame: baseline to 12 weeks

    Patient reported outcome journal

  6. 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

  7. 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

  8. 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 outcomes

  1. Number of pulmonary exacerbations

    Time frame: baseline to 12 weeks

    Assessment of number of pulmonary exacerbations at baseline compared through 12 weeks

Sponsors and collaborators

Lead sponsor

Nivalis Therapeutics, Inc.

Industry

Collaborators

  • Medidata Solutions

Registry information

Official study 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

Acronym: SNO-6

Important dates

Study start
2015
Primary completion
2016
Study completion
2016
First posted
Oct 28, 2015
Registry last updated
Jan 10, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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