Cystic FibrosisPhase 1SNO3NCT02227888
PK Study of N91115 in Cystic Fibrosis Patients
A Phase 1 study testing N91115 in people with Cystic Fibrosis, run by Nivalis Therapeutics, Inc. at 3 sites in United States.
Trial summary
This Phase 1 study in F508del-CFTR homozygous CF patients is being conducted to assess the pharmacokinetics and absorption dynamics of N91115 compared with healthy subjects in order to identify an initial starting dose for Phase 2 trials. 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?
- N91115
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 3 sitesUnited States
- Study dates
- Oct 2014to Jan 2015
- Phase
- Phase 1
- 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, homozygous for the F508del-CFTR mutation
- Weight ≥ 40 kg at screening
- FEV1 ≥ 40% of predicted normal for age, gender, and height (Hankinson standards) pre or post-bronchodilator value, at screening
- Hematology, clinical chemistry, and urinalysis results with no clinically significant abnormalities that would interfere with the study assessments at screening
Who cannot join
- History of any illness or condition that in the opinion of the investigator could confound the results of the study or pose additional risk when administered IMP
- Any acute infection, including acute upper or lower respiratory infections and pulmonary exacerbations that require treatment or hospitalizations within 4 weeks of Study Day 1
- Any change in chronic therapies for CF lung disease (e.g., Ibuprofen, Pulmozyme®, hypertonic saline, Azithromycin, TOBI®, Cayston®) within 4 weeks of Study Day 1
- History, including the screening assessment, of ventricular tachycardia or ventricular arrhythmias
- History, including the screening assessment, of prolonged QT and/or QTcF (Fridericia's correction) interval (> 450 msec)
- History of solid organ or hematological transplantation
- History of alcohol abuse or drug addiction (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
3 sites in 1 country.
United States3 sites
Children's CO
Aurora, Colorado, 80045
University of Alabama @ Birmingham
Birmingham, Alabama, 35294
National Jewish Health
Denver, Colorado, 80206
About this study
Official title: A Phase 1, Open-Label Study Evaluating the Pharmacokinetics of N91115 in Cystic Fibrosis Patients
This Phase 1 study in F508del-CFTR homozygous CF patients is being conducted to assess the pharmacokinetics and absorption dynamics of N91115 compared with healthy subjects in order to identify an initial starting dose for Phase 2 trials.
Read the detailed description
This is an open-label pharmacokinetic (PK) study of twice daily doses of 50 mg of N91115 administered for 14 days in F508del-CFTR homozygous CF patients. Six patients are planned for enrollment. Each patient will undergo screening (Day 28 to Day 3) and, if eligible, will return to the clinical site on Day 1 or optionally on Day 1. Eligibility will be reconfirmed and the patient will be admitted to a clinical research unit (CRU). Patients will stay overnight from Day 1 to Day 2 and will be discharged on Day 2 after all procedures and assessments are completed. Patients will return to the CRU as outpatients on Days 4, 7, 11, 14, and 15 for PK sample collection and other assessments. They will receive an oral dose of investigational medicinal product (IMP), N91115, twice daily on Days 1 through 14 and will be followed for PK through Day 15. Telephone calls to assess safety and ensure compliance with dosing will be made on days that patients do not make clinic visits (Days 3, 5, 6, 8, 9, 10, 12, and 13). Follow-up safety calls will then be made on Day 21 (adverse events) and Day 28 (serious adverse events only). Participation of an individual patient may last approximately 56 days from the time of screening until the end-of-study (Day 28) follow-up call.
- Conditions
- Cystic Fibrosis
- Being tested
- N91115
- Keywords
- CF, F508Del, Cavosonstat, N91115
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
N91115Drug
Also called Cavosonstat
S nitrosoglutathione reductase inhibitor
| Study group | Role | What they receive |
|---|---|---|
| N91115 | Experimental | Every 12 hour oral dosing of 50 mg N91115 for 14 days Receives: Drug: N91115 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetics
To assess the pharmacokinetics of N91115 after twice daily (q 12 h) doses given for 14 days by measuring plasma and urine concentrations of N91115 and its primary metabolite.
Measured: 14 Days
Secondary outcomes (1)
Safety of dosing twice per day over 14 days
To assess the safety of 14 twice daily (q 12 h) doses of N91115 in F508del-CFTR homozygous CF patients by monitoring, 12 lead ECG, Adverse events, Clinical safety laboratory tests (hematology, clinical chemistry, and urinalysis), Concomitant medications, Abbreviated physical examinations, Hemoglobin oxygen saturation (O2 Sat) by pulse oximetry, Spirometry, Telemetry, Vital signs, Weight
Measured: 14 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2014 (estimated)
- Study startOct 2014
- Primary completionJan 2015
- Study completionJan 2015
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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nivalis Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Steven Shoemaker, MD, Study director, Nivalis Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02227888
- Sponsor's protocol ID
- N91115-1PK-02
- Acronym
- SNO3
- Status last verified
- Nov 2016
- First submitted
- 26 Aug 2014
- Last updated
- 7 Nov 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open-Label Study Evaluating the Pharmacokinetics of N91115 in Cystic Fibrosis Patients
- ClinicalTrials.gov ID
- NCT02227888
- Acronym / sponsor ID
- SNO3 / N91115-1PK-02
- Conditions
- Cystic Fibrosis
- Keywords
- CF, F508Del, Cavosonstat, N91115
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Nivalis Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- Oct 2014
- Primary completion
- Jan 2015 (actual)
- Study completion
- Jan 2015 (actual)
- First posted
- 28 Aug 2014 (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 Phase 1 study in F508del-CFTR homozygous CF patients is being conducted to assess the pharmacokinetics and absorption dynamics of N91115 compared with healthy subjects in order to identify an initial starting dose for Phase 2 trials.
Detailed description
This is an open-label pharmacokinetic (PK) study of twice daily doses of 50 mg of N91115 administered for 14 days in F508del-CFTR homozygous CF patients. Six patients are planned for enrollment. Each patient will undergo screening (Day 28 to Day 3) and, if eligible, will return to the clinical site on Day 1 or optionally on Day 1. Eligibility will be reconfirmed and the patient will be admitted to a clinical research unit (CRU). Patients will stay overnight from Day 1 to Day 2 and will be discharged on Day 2 after all procedures and assessments are completed. Patients will return to the CRU as outpatients on Days 4, 7, 11, 14, and 15 for PK sample collection and other assessments. They will receive an oral dose of investigational medicinal product (IMP), N91115, twice daily on Days 1 through 14 and will be followed for PK through Day 15. Telephone calls to assess safety and ensure compliance with dosing will be made on days that patients do not make clinic visits (Days 3, 5, 6, 8, 9, 10, 12, and 13). Follow-up safety calls will then be made on Day 21 (adverse events) and Day 28 (serious adverse events only). Participation of an individual patient may last approximately 56 days from the time of screening until the end-of-study (Day 28) follow-up call.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| N91115 | Experimental | Drug: N91115Every 12 hour oral dosing of 50 mg N91115 for 14 days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Confirmed diagnosis of CF, homozygous for the F508del-CFTR mutation
- Weight ≥ 40 kg at screening
- FEV1 ≥ 40% of predicted normal for age, gender, and height (Hankinson standards) pre or post-bronchodilator value, at screening
- Hematology, clinical chemistry, and urinalysis results with no clinically significant abnormalities that would interfere with the study assessments at screening
Key exclusion criteria (8 listed)
- History of any illness or condition that in the opinion of the investigator could confound the results of the study or pose additional risk when administered IMP
- Any acute infection, including acute upper or lower respiratory infections and pulmonary exacerbations that require treatment or hospitalizations within 4 weeks of Study Day 1
- Any change in chronic therapies for CF lung disease (e.g., Ibuprofen, Pulmozyme®, hypertonic saline, Azithromycin, TOBI®, Cayston®) within 4 weeks of Study Day 1
- History, including the screening assessment, of ventricular tachycardia or ventricular arrhythmias
- History, including the screening assessment, of prolonged QT and/or QTcF (Fridericia's correction) interval (> 450 msec)
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 |
|---|---|---|
| N91115Also: CavosonstatS nitrosoglutathione reductase inhibitor | Drug | N91115 |
Primary outcome
Pharmacokinetics
Time frame: 14 Days
To assess the pharmacokinetics of N91115 after twice daily (q 12 h) doses given for 14 days by measuring plasma and urine concentrations of N91115 and its primary metabolite.
1 secondary outcome
Safety of dosing twice per day over 14 days
Time frame: 14 Days
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 7 Nov 2016.
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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.
Steven Shoemaker, MD study director
Nivalis Therapeutics, Inc.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing N91115 in people with Cystic Fibrosis, run by Nivalis Therapeutics, Inc. at 3 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 4 inclusion and 8 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 7 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 3 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Pharmacokinetics (time frame: 14 Days). 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 7 Nov 2016.