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Study of Cavosonstat (N91115) in CF Patients Who Are Heterozygous for F508del-CFTR and a Gating Mutation and Being Treated With Ivacaftor (SNO-7)

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ClinicalTrials.gov Identifier: NCT02724527
Recruitment Status : Unknown
Verified November 2016 by Nivalis Therapeutics, Inc..
Recruitment status was:  Active, not recruiting
First Posted : March 31, 2016
Last Update Posted : November 21, 2016
Sponsor:
Information provided by (Responsible Party):
Nivalis Therapeutics, Inc.

Brief Summary:
Cavosonstat (N91115) is being studied as a potential novel therapy for cystic fibrosis (CF), and this study assesses a target population of patients who are heterozygous for F508del-CFTR and a gating mutation that is approved for treatment with ivacaftor (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R).

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Cavosonstat Drug: Placebo Phase 2

Detailed Description:
Assess the effect of Cavosonstat (N91115) on lung function when added to preexisting treatment with ivacaftor in adult patients with CF who are heterozygous for F508del-CFTR and a gating mutation that is approved for treatment with ivacaftor (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R).

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
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 for Efficacy and Safety in Patients With CF Heterozygous for F508del-CFTR + Gating Mutation Being Treated With Ivacaftor
Study Start Date : April 2016
Estimated Primary Completion Date : April 2017
Estimated Study Completion Date : April 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

Arm Intervention/treatment
Placebo Comparator: Placebo
Matching capsule (BID administration Q12H)
Drug: Placebo
Matched Placebo capsule
Other Name: Control

Experimental: Cavosonstat (N91115) 400 mg
Cavosonstat (N91115) at 400 mg dose (100 mg x 4 capsules) (BID administration Q12H)
Drug: Cavosonstat
CFTR modulator that stabilizes CFTR
Other Name: N91115




Primary Outcome Measures :
  1. The absolute change in ppFEV1 in the N91115 treated group [ Time Frame: Baseline, week 4 and 8 assessments ]
    Forced Expiratory Volume (FEV) absolute measurements comparing baseline to after 4 and 8 weeks of N91115 treatment. FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. ppFEV1 (predicted for age, gender, and height) is calculated using the Hankinson method.


Secondary Outcome Measures :
  1. The relative change from study baseline within the active treatment group in ppFEV1 values [ Time Frame: Baseline, week 4 and 8 assessments ]
    Forced Expiratory Volume relative measurements comparing baseline to after 4 and 8 weeks of N91115 treatment. FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. ppFEV1 (predicted for age, gender, and height) is calculated using the Hankinson method.

  2. Absolute change from study baseline within the active treatment group in sweat chloride [ Time Frame: Baseline, week 4 and 8 assessments ]
    Sweat chloride concentration measured by pilocarpine iontophoresis, a standard clinical laboratory technique. Sweat collection accomplished with the Wescor Macroduct System.

  3. Changes in the respiratory domain of the Cystic Fibrosis Questionnaire - Revised, (CFQ-R) [ Time Frame: Baseline, week 4 and 8 assessments ]
    Patient questionnaires will compare baseline scores on their respiratory symptoms to weeks 4 and 8

  4. Absolute change from baseline within the active treatment group in Patient Global Impression of Change [ Time Frame: Baseline, week 4 and 8 assessments ]
    Patient questionnaires will compare baseline global impression of changes in health from baseline to weeks 4 and 8

  5. Safety as determined by adverse events assessment [ Time Frame: Baseline to 8 weeks treatment with a 28-day follow up period ]
    Assessments of clinical laboratory values, electrocardiogram (ECG), pulmonary exacerbations, and vital signs

  6. Pharmacokinetic Assessment of Maximum Plasma Concentration [Cmax] for N91115 & ivacaftor [ Time Frame: Weeks 1, 4 and 8 ]
    Plasma collection for assessment of N91115 and ivacaftor Cmax

  7. Pharmacokinetic Assessment of area under the plasma concentration verse time curve [AUC] for N91115 & ivacaftor [ Time Frame: Weeks 1, 4 and 8 ]
    Plasma collection for assessment of N91115 and ivacaftor AUC



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

Inclusion Criteria:

  • Confirmed diagnosis of CF, heterozygous for F508del-CFTR and a gating mutation that is approved for treatment with ivacaftor (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R)
  • Have been treated with chronic ivacaftor twice daily for at least 6 months prior to Screening (date of consent) and are currently being treated with commercially available Ivacaftor
  • Negative serum pregnancy test
  • Weight ≥ 40 kg at screening
  • Oxygen saturation by pulse oximetry ≥ 90% breathing ambient air, at screening

Exclusion Criteria:

  • Any acute infection, including acute upper or lower respiratory infections and pulmonary exacerbations that require treatment that has completed within 2 weeks of Study Day 1 or hospitalization discharge within 2 weeks of Study Day 1
  • Recent infection (per investigator discretion) with organisms associated with more rapid decline in pulmonary status, for example: 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
  • Blood hemoglobin < 10 g/dL at screening
  • Serum albumin < 2.5 g/dL at screening
  • Abnormal liver or renal function
  • 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 (> 450 msec for men; > 470 msec for women)
  • History of solid organ or hematological transplantation
  • History of alcohol abuse or drug abuse (including cannabis, cocaine, and opioids) in the year prior to screening
  • 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): NCT02724527


Locations
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United States, Colorado
National Jewish Health
Denver, Colorado, United States, 80206
United States, Maryland
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, Missouri
Washington University
St. Louis, Missouri, United States, 63110
United States, New York
Columbia University
New York, New York, United States, 10032
United States, Ohio
Cincinnati Children's Hospital
Cincinnati, Ohio, United States, 45229
Rainbow Babies and Children's Hospital - Case Medical Center
Cleveland, Ohio, United States, 44106
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Children's Hospital Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
United States, Utah
University of Utah
Salt Lake City, Utah, United States, 84132
United States, Wisconsin
Medical Center of Wisconsin
Madison, Wisconsin, United States, 53792
Sponsors and Collaborators
Nivalis Therapeutics, Inc.
Investigators
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Principal Investigator: James Chmiel, MD Rainbow Babies and Children's Hospital/ University

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Responsible Party: Nivalis Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT02724527     History of Changes
Other Study ID Numbers: N91115-2CF-06
First Posted: March 31, 2016    Key Record Dates
Last Update Posted: November 21, 2016
Last Verified: November 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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

Additional relevant MeSH terms:
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Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action