Cystic FibrosisPhase 4NCT02015663

Tobramycin Inhalation Powder (TIP) Administered Once Daily Continuously Versus TIP Administered BID in 28 Day on / 28 Day Off Cycles

A Phase 4 study testing Tobramycin Inhalation Powder in people with Cystic Fibrosis, run by Novartis Pharmaceuticals at 21 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

Search all trials

Trial summary

To provide efficacy and safety data comparing two dosing schedules of Tobramycin Inhalation Powder (TIP) for the treatment of pulmonary Pseudomonas aeruginosa in patients with cystic fibrosis. Read more about this study

Who is it for?: Ages 6 years and older
Conditions the study is for: Cystic Fibrosis
What is being tested?: Tobramycin Inhalation Powder
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 32 participants
Where: 21 sitesUnited States
Study dates: Jan 2014to Dec 2014
Phase: Phase 4
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 6 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Provide written informed consent, HIPPA (Health Insurance Portability and Accountability Act) authorization (where applicable), and assent (as appropriate) prior to the performance of any study-related procedure.
Confirmed diagnosis of CF
FEV1 at screening (Visit 1) ≥25% and ≤ 80% of normal predicted values for age, sex, and height
P. aeruginosa must be present within 6 months prior to screening and at screening
Able to comply with all protocol requirements
Clinically stable in the opinion of the investigator

Who cannot join

History of Burkholderia cenocepacia (Bcc) complex within 2 years prior to screening and/or Bcc complex at screening
Hemoptysis more than 60 cc at any time within 30 days prior to study drug administration
History of hearing loss or chronic tinnitus deemed clinically significant by the investigator
Serum creatinine 2 mg/dL or greater, BUN 40 mg/dL or greater, or an abnormal urinalysis defined as 2+ or greater proteinuria at screening
Known local or systemic hypersensitivity to aminoglycosides or inhaled antibiotics
Patients who are unable to discontinue previously received inhaled antibiotic regimen(s) (inhaled antibiotics are not allowed other than study drug)

Show all 19Show fewer

Use of inhaled aminoglycosides within 28 days prior to study drug administration (Visit 2)
Use of systemic anti-pseudomonal antibiotics within 28 days prior to study drug administration
Use of loop diuretics within 7 days prior to study drug administration
Administration of any investigational drug within 30 days prior to enrollment or 5 half-lives, whichever is longer
Signs and symptoms of acute pulmonary disease, e.g , pneumonia, pneumothorax
Hospitalization during the baseline visit
History of malignancy
Patients with clinically significant laboratory abnormalities (not associated with the study indication) at screening
Patients with other clinically significant conditions (not associated with the study indication) which might interfere with the assessment of this study
Patients or caregivers with a history of noncompliance to medical regimens and patients or caregivers who are considered potentially unreliable
Pregnant or nursing (lactating) women
Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, including women whose career, lifestyle, or sexual orientation precludes intercourse with a male partner and women whose partners have been sterilized by vasectomy or other means, UNLESS they are using two birth control methods.
Other protocol-defined inclusion/exclusion criteria may apply.

Trial locations and contacts

21 sites in 1 country.

City, postcode or hospital

21 sites in 1 country

United States21 sites

Novartis Investigative Site
Akron, Ohio, 44308
Novartis Investigative Site
Altamonte Springs, Florida, 32701
Novartis Investigative Site
Austin, Texas, 78723
Novartis Investigative Site
Glenview, Illinois, 60025
Novartis Investigative Site
Hershey, Pennsylvania, 17033-8050
Novartis Investigative Site
Indianapolis, Indiana, 46202
Novartis Investigative Site
Iowa City, Iowa, 52242
Novartis Investigative Site
Jacksonville, Florida, 32207
Novartis Investigative Site
Los Angeles, California, 90027
Novartis Investigative Site
Miami, Florida, 33136
Novartis Investigative Site
Mobile, Alabama, 36608-1128
Novartis Investigative Site
New Hyde Park, New York, 11040
Novartis Investigative Site
New York, New York, 10595
Novartis Investigative Site
Oklahoma City, Oklahoma, 73112
Novartis Investigative Site
Orlando, Florida, 32806
Novartis Investigative Site
Pensacola, Florida, 32504
Novartis Investigative Site
Portland, Oregon, 92772
Novartis Investigative Site
Sacramento, California, 95819
Novartis Investigative Site
Salt Lake City, Utah, 84132
Novartis Investigative Site
St Louis, Missouri, 63104
Novartis Investigative Site
Ventura, California, 93003

Other trials in United States

About this study

Official title: A 24-week, Open-label, Parallel-group, Interventional Phase IV Study Comparing Tobramycin Inhalation Powder (TIP) Administered Once Daily Continuously Versus TIP Administered BID in 28 Day on / 28 Day Off Cycles for the Treatment of Pulmonary Pseudomonas Aeruginosa in Patients With Cystic Fibrosis

To provide efficacy and safety data comparing two dosing schedules of Tobramycin Inhalation Powder (TIP) for the treatment of pulmonary Pseudomonas aeruginosa in patients with cystic fibrosis.

Conditions: Cystic Fibrosis
Being tested: Tobramycin Inhalation Powder
Keywords: Cystic Fibrosis, CF
Medical subject headings: Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Tobramycin Inhalation PowderDrug
Tobramycin Inhalation Powder 112 mg (four 28-mg capsules) taken via inhaler once or twice a day, depending on study arm

Study groups in NCT02015663
Study group	Role	What they receive
Arm 1	Experimental	Tobramycin Inhalation Powder (112 mg) once daily during 168 days Receives: Drug: Tobramycin Inhalation Powder
Arm 2	Active comparator	Tobramycin Inhalation Powder (112 mg) twice daily on days 1-28, days 57-84 and days 113-140 Receives: Drug: Tobramycin Inhalation Powder

What is being measured

The primary outcome is the main question the study is designed to answer.

Change From Baseline in Forced Expiratory Volume in 1 Second ( FEV1) Percent Predicted
The Forced Expiratory Volume in 1 second (FEV1) percent predicted expresses FEV1 as a percentage of the "predicted values" for participants of similar characteristics (height, age, sex, and sometimes race and weight). A positive change from baseline in FEV1 percent predicted indicates improvement in lung function.
Measured: Baseline and Day 168

Secondary outcomes (11)

Percent Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) Percent Predicted
The Forced Expiratory Volume in 1 second (FEV1) percent predicted expresses FEV1 as a percentage of the "predicted values" for participants of similar characteristics (height, age, sex, and sometimes race and weight). A positive change from baseline in FEV1 percent predicted indicates improvement in lung function.
Measured: Baseline and Day 168
Percent Change From Baseline in Forced Vital Capacity (FVC) Percent Predicted
Forced Vital Capacity (FVC) is the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible. FVC will be assessed via spirometry. A positive change from baseline in FVC indicates improvement in lung function.
Measured: Baseline and Day 168
Percent Change From Baseline in Forced Expiratory Flow (FEF) 25%-75% Predicted
The Forced Expiratory Flow (FEF) 25%-75% measurement describes the amount of air expelled from the lungs during the middle half (25% - 75%) of the forced vital capacity test and is measured using spirometry. A positive change from baseline in FEF indicates improvement in lung function. The predicted percent will be assessed.
Measured: Baseline and day 168
Change From Baseline in Pseudomonas Aeruginosa Sputum Density
Change from baseline in Pseudomonas aeruginosa sputum density will be measured by log10 colony forming units per gram of sputum.
Measured: Baseline and day 168
Time to First Hospitalization Due to Respiratory-related Events
Time to the first hospitalization due to respiratory-related events (number of days) per patient.
Measured: Day 1 to day 168
Percentage of Patients With Hospitalizations Due to Respiratory-related Events
Percentage of patients with hospitalization due to respiratory-related events
Measured: Day 1 to day 168
Length of Hospital Stay Due to Respiratory-related Events
The number of days in length of hospital stay per patient due to respiratory-related events will be measured.
Measured: Day 1 to day 168
Time to First Usage of Anti-pseudomonal Antibiotic
Time to first usage of anti-pseudomonal antibiotic per patient will be assessed by number of days
Measured: Day 1 to day 168
Percentage of Patients Who Use Anti-pseudomonal Antibiotic
Percentage of patients who use anti-pseudomonal antibiotic will be assessed.
Measured: Day 1 to day 168
Duration of Use of Anti-pseudomonal Antibiotic
Number of days of use of anti-pseudomonal antibiotic per patient will be assessed.
Measured: Day 1 to day 168
Change From Baseline in Tobramycin Minimal Inhibitory Concentration for Pseudomonas Aeruginosa
Change from baseline in tobramycin minimal inhibitory concentration for Pseudomonas aeruginosa will be measured by laboratory testing.
Measured: Baseline and day 168

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jan 2014Dec 2014

First posted19 Dec 2013 (estimated)
Study startJan 2014
Primary completionDec 2014
Study completionDec 2014

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Cystic FibrosisStudies of the same condition, open and under way.
More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
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 4
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 32 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02015663
Sponsor's protocol ID: CTBM100CUS03
Status last verified: Feb 2017
First submitted: 13 Dec 2013
Last updated: 29 Mar 2017
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
Active, not recruiting
Personalized Mobile Health Platform to Promote Physical Activity in Adolescents and Young Adults With Cystic Fibrosis
Cystic Fibrosis (CF)
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingPhase 1
Investigation of Pharmacokinetics, Safety of HSK31858 in Subjects With Renal Impairment
Non-cystic Fibrosis Bronchiectasis (NCFBE)

All Cystic Fibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 29 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A 24-week, Open-label, Parallel-group, Interventional Phase IV Study Comparing Tobramycin Inhalation Powder (TIP) Administered Once Daily Continuously Versus TIP Administered BID in 28 Day on / 28 Day Off Cycles for the Treatment of Pulmonary Pseudomonas Aeruginosa in Patients With Cystic Fibrosis
ClinicalTrials.gov ID: NCT02015663
Acronym / sponsor ID: CTBM100CUS03
Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis, CF
Phase: Phase 4
Study type: Interventional
Recruitment status: Terminated
Enrolment: 32 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: Jan 2014
Primary completion: Dec 2014 (actual)
Study completion: Dec 2014 (actual)
First posted: 19 Dec 2013 (estimated)
Results: Posted 29 Mar 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

To provide efficacy and safety data comparing two dosing schedules of Tobramycin Inhalation Powder (TIP) for the treatment of pulmonary Pseudomonas aeruginosa in patients with cystic fibrosis.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Arm 1	Experimental	Drug: Tobramycin Inhalation PowderTobramycin Inhalation Powder (112 mg) once daily during 168 days
Arm 2	Active comparator	Drug: Tobramycin Inhalation PowderTobramycin Inhalation Powder (112 mg) twice daily on days 1-28, days 57-84 and days 113-140

Who may qualify

Ages: 6 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (6 listed)

Provide written informed consent, HIPPA (Health Insurance Portability and Accountability Act) authorization (where applicable), and assent (as appropriate) prior to the performance of any study-related procedure.
Confirmed diagnosis of CF
FEV1 at screening (Visit 1) ≥25% and ≤ 80% of normal predicted values for age, sex, and height
P. aeruginosa must be present within 6 months prior to screening and at screening
Able to comply with all protocol requirements

Key exclusion criteria (19 listed)

History of Burkholderia cenocepacia (Bcc) complex within 2 years prior to screening and/or Bcc complex at screening
Hemoptysis more than 60 cc at any time within 30 days prior to study drug administration
History of hearing loss or chronic tinnitus deemed clinically significant by the investigator
Serum creatinine 2 mg/dL or greater, BUN 40 mg/dL or greater, or an abnormal urinalysis defined as 2+ or greater proteinuria at screening
Known local or systemic hypersensitivity to aminoglycosides or inhaled antibiotics

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
Tobramycin Inhalation PowderTobramycin Inhalation Powder 112 mg (four 28-mg capsules) taken via inhaler once or twice a day, depending on study arm	Drug	Arm 1, Arm 2

Primary outcome

Change From Baseline in Forced Expiratory Volume in 1 Second ( FEV1) Percent Predicted
Time frame: Baseline and Day 168
The Forced Expiratory Volume in 1 second (FEV1) percent predicted expresses FEV1 as a percentage of the "predicted values" for participants of similar characteristics (height, age, sex, and sometimes race and weight). A positive change from baseline in FEV1 percent predicted indicates improvement in lung function.

11 secondary outcomes

Percent Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) Percent Predicted
Time frame: Baseline and Day 168
Percent Change From Baseline in Forced Vital Capacity (FVC) Percent Predicted
Time frame: Baseline and Day 168
Percent Change From Baseline in Forced Expiratory Flow (FEF) 25%-75% Predicted
Time frame: Baseline and day 168
Change From Baseline in Pseudomonas Aeruginosa Sputum Density
Time frame: Baseline and day 168
Time to First Hospitalization Due to Respiratory-related Events
Time frame: Day 1 to day 168
Percentage of Patients With Hospitalizations Due to Respiratory-related Events
Time frame: Day 1 to day 168
Length of Hospital Stay Due to Respiratory-related Events
Time frame: Day 1 to day 168
Time to First Usage of Anti-pseudomonal Antibiotic
Time frame: Day 1 to day 168
Percentage of Patients Who Use Anti-pseudomonal Antibiotic
Time frame: Day 1 to day 168
Duration of Use of Anti-pseudomonal Antibiotic
Time frame: Day 1 to day 168
Change From Baseline in Tobramycin Minimal Inhibitory Concentration for Pseudomonas Aeruginosa
Time frame: Baseline and day 168

Results and safety

Results posted to the registry on 29 Mar 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Tobramycin Inhalation Powder Once Daily	16	1
Tobramycin Inhalation Powder Twice Daily	16	4

Primary outcome: Change From Baseline in Forced Expiratory Volume in 1 Second ( FEV1) Percent Predicted

Time frame: Baseline and Day 168

Population analysed: The study intended to randomize 200 patients within 18 months; however, after 9 months, only 32 patients were successfully randomized, with a screen fail rate of 50%. Study was terminated with only safety data analyzed. No data collected met the pre-specified powering of 200 patients needed for analysis (only 32 patients randomized)

11 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 3% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Tobramycin Inhalation Powder Once Daily	3 of 16 (18.8%)	13 of 16 (81.3%)	Not reported
Tobramycin Inhalation Powder Twice Daily	1 of 15 (6.7%)	12 of 15 (80%)	Not reported
Total Events	4 of 31 (12.9%)	25 of 31 (80.6%)	Not reported

Limitations and caveats (from the sponsor): Due to premature termination, summaries and analyses planned in the protocol were eliminated in the statistical analysis plan prior to database lock. No inferential analysis will be provided.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Cystic Fibrosis

RecruitingObservational study
Role of Genetic Factors in the Development of Lung Disease
Cystic Fibrosis, Pulmonary Fibrosis
Active, not recruiting
Personalized Mobile Health Platform to Promote Physical Activity in Adolescents and Young Adults With Cystic Fibrosis
Cystic Fibrosis (CF)
RecruitingPhase 2
Early Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators.
Cystic Fibrosis - Complete
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingPhase 1
Investigation of Pharmacokinetics, Safety of HSK31858 in Subjects With Renal Impairment
Non-cystic Fibrosis Bronchiectasis (NCFBE)

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.

Novartis Pharmaceuticals study director

Novartis Pharmaceuticals

21 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Tobramycin Inhalation Powder in people with Cystic Fibrosis, run by Novartis Pharmaceuticals at 21 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 6 inclusion and 19 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: stopped early, as of the sponsor's last update on 29 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 21 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 29 Mar 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Change From Baseline in Forced Expiratory Volume in 1 Second ( FEV1) Percent Predicted (time frame: Baseline and Day 168). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02015663.

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 29 Mar 2017.