Cystic FibrosisPhase 2SNO-6NCT02589236
Study of Cavosonstat (N91115) in Patients With CF Homozygous for the F508del-CFTR Mutation
A Phase 2 study testing Cavosonstat in people with Cystic Fibrosis, run by Nivalis Therapeutics, Inc. at 46 sites in United States.
Trial 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™). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Cavosonstat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 138 participants
- Where
- 46 sitesUnited States
- Study dates
- Nov 2015to Dec 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 14Show fewer
- 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
Trial locations and contacts
46 sites in 1 country.
46 sites in 1 country
United States46 sites
Children's Hospital Medical Center of Akron
Akron, Ohio, 44308
Children's Hospital Colorado
Aurora, Colorado, 80045
Dell Children's Hospital | Austin Children's Chest Associates
Austin, Texas, 78723
Johns Hopkins Hospital
Baltimore, Maryland, 21287
University of Alabama
Birmingham, Alabama, 35294
St. Luke's CF Center of Idaho
Boise, Idaho, 83712
Boston Children's Hospital
Boston, Massachusetts, 02115
University of North Carolina Chapel Hill
Chapel Hill, North Carolina, 27599
Medical University of South Carolina
Charleston, South Carolina, 29425
Northwestern University
Chicago, Illinois, 60611
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
Rainbow Babies and Children's Hospital - Case Medical Center
Cleveland, Ohio, 44106
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
National Jewish Health
Denver, Colorado, 80206
Wayne State University-Harper
Detroit, Michigan, 48202
Cook Children's Medical Center
Fort Worth, Texas, 76104
Spectrum Health Butterworth Hospital
Grand Rapids, Michigan, 49503
Texas Children's Hospital
Houston, Texas, 77030
Indiana University
Indianapolis, Indiana, 46202
University of Iowa Children's Hospital
Iowa City, Iowa, 52242
University of Kentucky
Lexington, Kentucky, 40536
Children's Hospital Los Angeles
Los Angeles, California, 90027
Medical Center of Wisconsin
Madison, Wisconsin, 53792
University of Minnesota
Minneapolis, Minnesota, 55455
Morristown Medical Center
Morristown, New Jersey, 07960
Yale University
New Haven, Connecticut, 06510
Columbia University
New York, New York, 10032
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Stanford University
Palo Alto, California, 94304
Saint Francis Medical Center
Peoria, Illinois, 61637
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Maine Medical Center
Portland, Maine, 04102
Oregon Health and Science University
Portland, Oregon, 97239
UC Davis Medical Center
Sacramento, California, 95817
University of Utah
Salt Lake City, Utah, 84132
Seattle Children's Hospital
Seattle, Washington, 98105
St. Louis University
St Louis, Missouri, 63104
Washington University
St Louis, Missouri, 63110
Tampa General Hospital
Tampa, Florida, 33606
ProMedica Toledo Children's Hospital
Toledo, Ohio, 43606
Banner University of Arizona Medical Center
Tucson, Arizona, 85724
Via Christi Research
Wichita, Kansas, 67214
Wake Forest University School of Medicine
Winston-Salem, North Carolina, 27103
University of Massachusetts Medical School
Worcester, Massachusetts, 01655
About this study
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
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™).
Read the 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
- Conditions
- Cystic Fibrosis
- Being tested
- Cavosonstat, Placebo
- Keywords
- Cystic Fibrosis, N91115, Cavosonstat
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CavosonstatDrug
Also called N91115
GSNOR inhibitor
PlaceboDrug
Also called control
Control sample with only capsule excipients and fillers
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Placebo Capsule Receives: Drug: Placebo |
| Cavosonstat (N91115) 200 mg | Experimental | Cavosonstat (N91115) 200 mg twice daily (BID) Receives: Drug: Cavosonstat |
| Cavosonstat (N91115) 400 mg | Experimental | Cavosonstat (N91115) 400 mg BID Receives: Drug: Cavosonstat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absolute change from baseline in percent predicted FEV1 (ppFEV1)
Forced Expiratory Volume in one second (FEV1) from before study (Baseline) to after 12 weeks of N91115 treatment
Measured: From baseline to 12 weeks
Secondary outcomes (8)
Relative change from baseline in ppFEV1
Forced Expiratory Volume in one second (FEV1) from before study (Baseline) to after 12 weeks of N91115 treatment
Measured: baseline to 12 weeks
Absolute change from baseline in sweat chloride
A sweat chloride measurement on the skin at study start and after 12 weeks of N91115
Measured: baseline to 12 weeks
Absolute change from baseline in Cystic Fibrosis Questionnaire -Revised CFQ-R (respiratory symptom scale)
Comparison of the Questionnaire from study start to 16 weeks
Measured: baseline to 16 weeks
Absolute change from baseline in body mass index (BMI)
Assessment of change in body mass index from study start to after 12 weeks of N91115
Measured: baseline to 12 weeks
Absolute change from baseline in Patient Global Impression of Change (PGIC)
Patient reported outcome journal
Measured: baseline to 12 weeks
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability])
Any adverse events assessment including clinical laboratory values, electrocardiogram (ECG), pulmonary exacerbations, or vital sign changes
Measured: baseline to 16 weeks
Pharmacokinetic Measurements of Maximum Plasma Concentration [Cmax], of N91115, lumacaftor, and ivacaftor
Maximum Plasma Concentration [Cmax] measurements of N91115, lumacaftor and ivacaftor
Measured: baseline to 12 weeks
Pharmacokinetic Measurements of Area Under the Curve (AUC) for N91115, Ivacaftor and lumacaftor
AUC measurements of N91115, lumacaftor and ivacaftor
Measured: baseline to 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Oct 2015 (estimated)
- Study startNov 2015
- Primary completionDec 2016
- Study completionDec 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 138 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nivalis Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Medidata Solutions
- Responsible party
- Sponsor
- Investigators
- Scott Donaldson, MD, Principal investigator, University of North Carolina, Chapel Hill
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02589236
- Sponsor's protocol ID
- N91115-2CF-05
- Acronym
- SNO-6
- Status last verified
- Jan 2017
- First submitted
- 26 Oct 2015
- Last updated
- 10 Jan 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 10 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02589236
- Acronym / sponsor ID
- SNO-6 / N91115-2CF-05
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, N91115, Cavosonstat
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 138 participants (actual)
- Lead sponsor
- Nivalis Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Medidata Solutions
- Study start
- Nov 2015
- Primary completion
- Dec 2016 (actual)
- Study completion
- Dec 2016 (actual)
- First posted
- 28 Oct 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboPlacebo Capsule |
| Cavosonstat (N91115) 200 mg | Experimental | Drug: CavosonstatCavosonstat (N91115) 200 mg twice daily (BID) |
| Cavosonstat (N91115) 400 mg | Experimental | Drug: CavosonstatCavosonstat (N91115) 400 mg BID |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (14 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CavosonstatAlso: N91115GSNOR inhibitor | Drug | Cavosonstat (N91115) 200 mg, Cavosonstat (N91115) 400 mg |
| PlaceboAlso: controlControl sample with only capsule excipients and fillers | Drug | Placebo |
Primary outcome
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
8 secondary outcomes
Relative change from baseline in ppFEV1
Time frame: baseline to 12 weeks
Absolute change from baseline in sweat chloride
Time frame: baseline to 12 weeks
Absolute change from baseline in Cystic Fibrosis Questionnaire -Revised CFQ-R (respiratory symptom scale)
Time frame: baseline to 16 weeks
Absolute change from baseline in body mass index (BMI)
Time frame: baseline to 12 weeks
Absolute change from baseline in Patient Global Impression of Change (PGIC)
Time frame: baseline to 12 weeks
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability])
Time frame: baseline to 16 weeks
Pharmacokinetic Measurements of Maximum Plasma Concentration [Cmax], of N91115, lumacaftor, and ivacaftor
Time frame: baseline to 12 weeks
Pharmacokinetic Measurements of Area Under the Curve (AUC) for N91115, Ivacaftor and lumacaftor
Time frame: baseline to 12 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jan 2017.
Related trials for Cystic Fibrosis
RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
Non-cystic Fibrosis Bronchiectasis
Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Scott Donaldson, MD principal investigator
University of North Carolina, Chapel Hill
46 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cavosonstat in people with Cystic Fibrosis, run by Nivalis Therapeutics, Inc. at 46 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 46 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Absolute change from baseline in percent predicted FEV1 (ppFEV1) (time frame: From baseline to 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jan 2017.