Cystic FibrosisPhase 2SNO-7NCT02724527
Study of Cavosonstat (N91115) in CF Patients Who Are Heterozygous for F508del-CFTR and a Gating Mutation and Being Treated With Ivacaftor
A Phase 2 study testing Cavosonstat in people with Cystic Fibrosis, run by Nivalis Therapeutics, Inc. at 12 sites in United States.
Trial 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… 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
- 19 participants
- Where
- 12 sitesUnited States
- Study dates
- Apr 2016to Apr 2017
- 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
- 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
Who cannot join
- 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
Show all 11Show fewer
- 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
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Johns Hopkins Hospital
Baltimore, Maryland, 21287
Boston Children's Hospital
Boston, Massachusetts, 02115
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
National Jewish Health
Denver, Colorado, 80206
Medical Center of Wisconsin
Madison, Wisconsin, 53792
Columbia University
New York, New York, 10032
Children's Hospital Pittsburgh
Pittsburgh, Pennsylvania, 15224
Oregon Health and Science University
Portland, Oregon, 97239
University of Utah
Salt Lake City, Utah, 84132
Washington University
St Louis, Missouri, 63110
About this study
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
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).
Read the 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).
- 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
CFTR modulator that stabilizes CFTR
PlaceboDrug
Also called Control
Matched Placebo capsule
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Matching capsule (BID administration Q12H) Receives: Drug: Placebo |
| Cavosonstat (N91115) 400 mg | Experimental | Cavosonstat (N91115) at 400 mg dose (100 mg x 4 capsules) (BID administration Q12H) Receives: Drug: Cavosonstat |
What is being measured
The primary outcome is the main question the study is designed to answer.
The absolute change in ppFEV1 in the N91115 treated group
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.
Measured: Baseline, week 4 and 8 assessments
Secondary outcomes (7)
The relative change from study baseline within the active treatment group in ppFEV1 values
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.
Measured: Baseline, week 4 and 8 assessments
Absolute change from study baseline within the active treatment group in sweat chloride
Sweat chloride concentration measured by pilocarpine iontophoresis, a standard clinical laboratory technique. Sweat collection accomplished with the Wescor Macroduct System.
Measured: Baseline, week 4 and 8 assessments
Changes in the respiratory domain of the Cystic Fibrosis Questionnaire - Revised, (CFQ-R)
Patient questionnaires will compare baseline scores on their respiratory symptoms to weeks 4 and 8
Measured: Baseline, week 4 and 8 assessments
Absolute change from baseline within the active treatment group in Patient Global Impression of Change
Patient questionnaires will compare baseline global impression of changes in health from baseline to weeks 4 and 8
Measured: Baseline, week 4 and 8 assessments
Safety as determined by adverse events assessment
Assessments of clinical laboratory values, electrocardiogram (ECG), pulmonary exacerbations, and vital signs
Measured: Baseline to 8 weeks treatment with a 28-day follow up period
Pharmacokinetic Assessment of Maximum Plasma Concentration [Cmax] for N91115 & ivacaftor
Plasma collection for assessment of N91115 and ivacaftor Cmax
Measured: Weeks 1, 4 and 8
Pharmacokinetic Assessment of area under the plasma concentration verse time curve [AUC] for N91115 & ivacaftor
Plasma collection for assessment of N91115 and ivacaftor AUC
Measured: Weeks 1, 4 and 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Mar 2016 (estimated)
- Study startApr 2016
- Primary completionApr 2017 (estimated)
- Study completionApr 2017 (estimated)
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
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nivalis Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- James Chmiel, MD, Principal investigator, Rainbow Babies and Children's Hospital/ University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02724527
- Sponsor's protocol ID
- N91115-2CF-06
- Acronym
- SNO-7
- Status last verified
- Nov 2016
- First submitted
- 11 Mar 2016
- Last updated
- 21 Nov 2016
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Nov 2016. 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 for Efficacy and Safety in Patients With CF Heterozygous for F508del-CFTR + Gating Mutation Being Treated With Ivacaftor
- ClinicalTrials.gov ID
- NCT02724527
- Acronym / sponsor ID
- SNO-7 / N91115-2CF-06
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, N91115, Cavosonstat
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Nivalis Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- Apr 2016
- Primary completion
- Apr 2017 (estimated)
- Study completion
- Apr 2017 (estimated)
- First posted
- 31 Mar 2016 (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.
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).
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboMatching capsule (BID administration Q12H) |
| Cavosonstat (N91115) 400 mg | Experimental | Drug: CavosonstatCavosonstat (N91115) at 400 mg dose (100 mg x 4 capsules) (BID administration Q12H) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (11 listed)
- 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
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: N91115CFTR modulator that stabilizes CFTR | Drug | Cavosonstat (N91115) 400 mg |
| PlaceboAlso: ControlMatched Placebo capsule | Drug | Placebo |
Primary outcome
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.
7 secondary outcomes
The relative change from study baseline within the active treatment group in ppFEV1 values
Time frame: Baseline, week 4 and 8 assessments
Absolute change from study baseline within the active treatment group in sweat chloride
Time frame: Baseline, week 4 and 8 assessments
Changes in the respiratory domain of the Cystic Fibrosis Questionnaire - Revised, (CFQ-R)
Time frame: Baseline, week 4 and 8 assessments
Absolute change from baseline within the active treatment group in Patient Global Impression of Change
Time frame: Baseline, week 4 and 8 assessments
Safety as determined by adverse events assessment
Time frame: Baseline to 8 weeks treatment with a 28-day follow up period
Pharmacokinetic Assessment of Maximum Plasma Concentration [Cmax] for N91115 & ivacaftor
Time frame: Weeks 1, 4 and 8
Pharmacokinetic Assessment of area under the plasma concentration verse time curve [AUC] for N91115 & ivacaftor
Time frame: Weeks 1, 4 and 8
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Nov 2016.
- Individual participant dataSharing planned: No
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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.
James Chmiel, MD principal investigator
Rainbow Babies and Children's Hospital/ University
12 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 12 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 11 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: status not confirmed, as of the sponsor's last update on 21 Nov 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 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?
Results are not available in the source record.
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?
The absolute change in ppFEV1 in the N91115 treated group (time frame: Baseline, week 4 and 8 assessments). 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 21 Nov 2016; AzTrials last refreshed its copy on 8 Nov 2026.