AzTrials
AzTrials

Pulmonary InfectionsPhase 4NCT01519661

Long Term Safety of Tobramycin Inhalation Powder in Patients With Cystic Fibrosis

A Phase 4 study testing TBM100 in people with Pulmonary Infections and Pseudomonas Aeruginosa in Cystic Fibrosis, run by Novartis Pharmaceuticals at 49 sites in 10 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This study assessed the long term safety data for the use of tobramycin inhalation powder in patients suffering from cystic fibrosis who have a chronic pulmonary infection with Pseudomonas aeruginosa. Read more about this study

Who is it for?
Ages 6 years and older
What is being tested?
TBM100
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
157 participants
Where
49 sitesin 10 countries
Study dates
Jan 2012to Jan 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

  • Confirmed diagnosis of Cystic Fibrosis
  • FEV1 at screening must be between 25 and 75 percent of normal predicted values for age, sex and height based on the Knudson equation
  • Pseudomonas aeruginosa must be present in a sputum / deep cough throat swab culture or bronchoalveolar lavage within 6 months prior to screening and in the sputum/deep-throat cough swab culture at screening

Who cannot join

  • History of sputum culture or deep cough throat swab culture yielding Burkholderia cenocepacia complex within 2 years prior to screening and /or sputum culture yielding Burkholderia cenocepacia at screening
  • Hemoptysis more than 60mL at any time within 30 days prior to study drug administration
  • History of hearing loss or chronic tinnitus deemed clinically significant
  • Serum creatinine 2mg/dl or more, BUN 40mg/dl or more, 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 regularly receiving more than 1 class of inhaled anti-pseudomonal antibiotic
Show all 9Show fewer
  • Any use of inhaled or systemic anti-pseudomonal antibiotic within 28 days prior to study drug administration
  • Use of loop diuretics within 7 days prior to study drug administration
  • Other protocol-defined inclusion/exclusion criteria may apply.

Trial locations and contacts

49 sites in 10 countries.

49 sites in 10 countries

United States20 sites

  • Novartis Investigative Site

    Akron, Ohio, 44308

  • Novartis Investigative Site

    Atlanta, Georgia, 30322

  • Novartis Investigative Site

    Charleston, South Carolina, 29425

  • Novartis Investigative Site

    Cleveland, Ohio, 44106

  • Novartis Investigative Site

    Dallas, Texas, 75230

  • Novartis Investigative Site

    Denver, Colorado, 80206

  • Novartis Investigative Site

    Fort Worth, Texas, 76104

  • Novartis Investigative Site

    Houston, Texas, 77030

  • Novartis Investigative Site

    Jacksonville, Florida, 32207

  • Novartis Investigative Site

    Las Vegas, Nevada, 89107

  • Novartis Investigative Site

    Little Rock, Arkansas, 72205

  • Novartis Investigative Site

    Madison, Wisconsin, 53792-1615

  • Novartis Investigative Site

    Milwaukee, Wisconsin, 53226

  • Novartis Investigative Site

    Morristown, New Jersey, 07962

  • Novartis Investigative Site

    Oklahoma City, Oklahoma, 73104

  • Novartis Investigative Site

    Oklahoma City, Oklahoma, 73112

  • Novartis Investigative Site

    Omaha, Nebraska, 68198

  • Novartis Investigative Site

    San Antonio, Texas, 78212

  • Novartis Investigative Site

    St Louis, Missouri, 63110

  • Novartis Investigative Site

    Tacoma, Washington, 98405

Other trials in United States

Italy7 sites

  • Novartis Investigative Site

    Florence, FI, 50139

  • Novartis Investigative Site

    Genova, GE, 16147

  • Novartis Investigative Site

    Messina, ME, 98125

  • Novartis Investigative Site

    Naples, 80131

  • Novartis Investigative Site

    Palermo, Italy, 90100

  • Novartis Investigative Site

    Roma, 00161

  • Novartis Investigative Site

    Verona, VR, 37126

Other trials in Italy

France5 sites

  • Novartis Investigative Site

    Giens, 83406

  • Novartis Investigative Site

    Montpellier, 34059

  • Novartis Investigative Site

    Paris, 75006

  • Novartis Investigative Site

    Reims, 51092

  • Novartis Investigative Site

    Roscoff, 29684

Other trials in France

Argentina4 sites

  • Novartis Investigative Site

    Caba, Buenos Aires, C1425DTG

  • Novartis Investigative Site

    Capital Federal, Buenos Aires, C1425EFD

  • Novartis Investigative Site

    Córdoba, Córdoba Province, X5014AKN

  • Novartis Investigative Site

    Paraná, Entre Ríos Province, E3100FKA

Other trials in Argentina

Australia3 sites

  • Novartis Investigative Site

    Clayton, Victoria, 3168

  • Novartis Investigative Site

    New Lambton Heights, New South Wales, 2305

  • Novartis Investigative Site

    Parkville, Victoria, 3052

Other trials in Australia

Canada3 sites

  • Novartis Investigative Site

    Calgary, Alberta, T2N 4n1

  • Novartis Investigative Site

    Edmonton, Alberta, T6G 2B7

  • Novartis Investigative Site

    Montreal, Quebec, H3T1C5

Other trials in Canada

Germany2 sites

  • Novartis Investigative Site

    Essen, 45147

  • Novartis Investigative Site

    Frankfurt, 60590

Other trials in Germany

Mexico2 sites

  • Novartis Investigative Site

    Mexico City, Mexico City, 06720

  • Novartis Investigative Site

    Monterrey, Nuevo León, 64460

Other trials in Mexico

Spain2 sites

  • Novartis Investigative Site

    Barcelona, Catalonia

  • Novartis Investigative Site

    Valencia, Valencia, 46026

Other trials in Spain

Hungary1 site

  • Novartis Investigative Site

    Budapest, 1121

Other trials in Hungary

About this study

Official title: A Single Arm, Open-label, Multicenter, Phase IV Trial to Assess Long Term Safety of Tobramycin Inhalation Powder (TIP) in Patients With Cystic Fibrosis

This study assessed the long term safety data for the use of tobramycin inhalation powder in patients suffering from cystic fibrosis who have a chronic pulmonary infection with Pseudomonas aeruginosa.

Being tested
TBM100
Keywords
Tobramycin Inhalation powder, Cystic fibrosis, Lung disease, Anti-bacterial agents
Medical subject headings
Cystic Fibrosis, Lung Diseases

What participants receive

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

  • TBM100Drug

    Tobramycin inhalation powder was assigned as four capsules at 28mg dosage strength. It was inhaled b.i.d in the morning and in the evening via the T-326 Inhaler.

Study groups in NCT01519661
Study groupRoleWhat they receive
Tobramycin Inhalation Powder (TIP)ExperimentalEligible patients were assigned to four capsules of TIP at 28mg dosage strength, inhaled b.i.d. in the morning and in the evening via the T-326 inhaler, for 28 days (on treatment), followed by 28 days of no study treatment (off treatment). Each treatment therefore consisted of 112mg tobramycin (4 capsules of 28mg each) with the total daily dose = 224mg tobramycin (112mg b.i.d.). These 56 days represented 1 cycle of therapy.
Receives: Drug: TBM100

What is being measured

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

  1. Percentage of Participants With Treatment Emergent Adverse Events, Serious Adverse Events (SAEs) and Deaths

    Adverse events were deemed treatment-emergent if the onset date/time was on or after the date and time of first study drug. All adverse events were included after this time during both on and off-treatment periods.

    Measured: 337 days

Secondary outcomes (11)

  1. Relative Change From Baseline in Forced Expiratory Volume in One Second (FEV1) Percent Predicted

    Spirometry was performed at each visit. FEV1, FVC, and FEF25-75 were recorded at all visits according to American Thoracic Society (ATS) guidelines. FEV1 = the volume of air expired in 1 second. FEV1 % predicted is a normalized value of FEV1 calculated using the Knudsen equation, based upon participant's age, gender and height. FVC (forced vital capacity) = the maximal volume of air exhaled with maximally forced effort from a position of maximal inspiration. FEF25-75 = forced expiratory flow from 25% to 75% of the FVC. Relative change in FEV1 % predicted from baseline to pre-dose day X = ((pre-dose day X FEV1 % predicted - baseline FEV1 % predicted) / baseline FEV1 % predicted) • 100.

    Measured: Baseline, day 29, day 85, day 141, day 197, day 253, day 309, day 337. All study visits except baseline and day 337 occurred at the end of a 28-day on-treatment period of a cycle. Day 337 was the end of the final 28-day off treatment period.

  2. Relative Change From Baseline in FVC Percent Predicted

    Spirometry was performed at each visit. FEV1, FVC, and FEF25-75 were recorded at all visits according to American Thoracic Society (ATS) guidelines. FEV1 = the volume of air expired in 1 second. FEV1 % predicted is a normalized value of FEV1 calculated using the Knudsen equation, based upon participant's age, gender and height. FVC (forced vital capacity) = the maximal volume of air exhaled with maximally forced effort from a position of maximal inspiration. FEF25-75 = forced expiratory flow from 25% to 75% of the FVC. Relative change in FVC % predicted from baseline to pre-dose day X = ((pre-dose day X FVC % predicted - baseline FVC % predicted) / baseline FVC % predicted) • 100.

    Measured: Baseline, day 29, day 85, day 141, day 197, day 253, day 309, day 337. All study visits except baseline and day 337 occurred at the end of a 28-day on-treatment period of a cycle. Day 337 was the end of the final 28-day off treatment period.

  3. Relative Change From Baseline in FEF Rate Over 25 to 75 Percent of Vital Capacity Predicted

    Spirometry was performed at each visit. FEV1, FVC, and FEF25-75 were recored at all visits according to American Thoracic Society (ATS) guidelines. FEV1 = the volume of air expired in 1 second. FEV1 % predicted is a normalized value of FEV1 calculated using the Knudsen equation, based upon participant's age, gender and height. FVC (forced vital capacity) = the maximal volume of air exhaled with maximally forced effort from a position of maximal inspiration. FEF25-75 = forced expiratory flow from 25% to 75% of the FVC. Relative change in FEEF25-75 from baseline to pre-dose day X = ((pre-dose day X FEF25-75 - baseline FEF25-75) / baseline FEF25-75) • 100.

    Measured: Baseline, day 29, day 85, day 141, day 197, day 253, day 309, day 337. All study visits except baseline and day 337 occurred at the end of a 28-day on-treatment period of a cycle. Day 337 was the end of the final 28-day off treatment period.

  4. Change From Baseline in Pseudomonas Aeruginosa Colony Forming Units in Sputum

    Sputum was collected in sterile containers and cultured for Pseudomonas aeruginosa (Pa.) (quantitative test) and other typical Cystic Fibrosis respiratory pathogens. The Pa. biotypes measured were mucoid, dry and small colony variant. Results are presented for the sum of all biotypes of Pa, with data transformed using a base 10 logarithm.

    Measured: Baseline, day 1, day 29, day 85, day 141, day 197, day 253, day 309, day 337

  5. Tobramycin MIC 50 and MIC 90 Values Over All Isolates for the Sum of All Biotypes (Mucoid, Dry and Small Colony Variant) of Pseudomonas Aeruginosa

    Tobramycin MIC 50 and MIC 90 values were defined as the lowest concentration of tobramycin required to inhibit 50% and 90%, respectively, of the P. aeruginosa strains tested.

    Measured: Baseline, day 29, day 85, day 141, day 197, day 253, day 309, day 337

  6. Percentage of Participants Hospitalized Due to Serious Respiratory-related Adverse Events

    Measured: Day 337

  7. Number of Hospitalization Days Due to Serious Respiratory-related Adverse Events

    The total number of hospitalization days due to serious respiratory-related adverse events was analyzed.

    Measured: Day 337

  8. Time to First Hospitalization Due to Serious Respiratory-related Adverse Events

    The day of first hospitalization due to serious respiratory-related adverse events was analyzed.

    Measured: Day 337

  9. Percentage of Participants Who Used New Anti-pseudomonal Antibiotics

    Measured: Day 337

  10. Number of Days of New Anti-pseudomonal Antibiotic Use

    The total number of days of new anti-pseudomonal antibiotic use was analyzed.

    Measured: Day 337

  11. Time to Use of New Anti-pseudomonal Antibiotic

    Time to first use of new anti-pseudomonal antibiotic was analyzed.

    Measured: Day 337

Timeline

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

  1. First posted27 Jan 2012 (estimated)
  2. Study startJan 2012
  3. Primary completionJan 2014
  4. Study completionJan 2014

Resources

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
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
157 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01519661
Sponsor's protocol ID
CTBM100C2401
Other IDs
2011-002000-32
Status last verified
Feb 2015
First submitted
24 Jan 2012
Last updated
10 Feb 2015

Publications

  • Sommerwerck U, Virella-Lowell I, Angyalosi G, Viegas A, Cao W, Debonnett L. Long-term safety of tobramycin inhalation powder in patients with cystic fibrosis: phase IV (ETOILES) study. Curr Med Res Opin. 2016 Nov;32(11):1789-1795. doi: 10.1080/03007995.2016.1211516. Epub 2016 Sep 9.

    PubMed 27435882

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing TBM100 in people with Pulmonary Infections and Pseudomonas Aeruginosa in Cystic Fibrosis, run by Novartis Pharmaceuticals at 49 sites in 10 countries.

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 3 inclusion and 9 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 Feb 2015. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 49 sites in United States, Italy, France, Argentina, Australia and 5 more countries. 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 10 Feb 2015). 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?

Percentage of Participants With Treatment Emergent Adverse Events, Serious Adverse Events (SAEs) and Deaths (time frame: 337 days). 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 NCT01519661, which also lists 1 linked publication.

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 Feb 2015.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in