Chronic Lung Infection With Pseudomonas Aeruginosa in Cystic FibrosisPhase 4ELIXIRNCT02248922
Evaluation of Lung Clearance Index in Cystic Fibrosis (CF) Patients, Infected With P.Aeruginosa
A Phase 4 study testing TIS or TIP in people with Chronic Lung Infection With Pseudomonas Aeruginosa in Cystic Fibrosis, run by Novartis Pharmaceuticals at 6 sites in Germany.
Trial summary
The purpose of the study was to evaluate lung clearance index (LCI) by a standardized procedure in a well characterized study setting and to assess feasibility of LCI as a more sensitive method than forced expiratory volume at 1 second (FEV1) to measure effectiveness of antibiotic therapy in patients with CF aged 6… Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Chronic Lung Infection With Pseudomonas Aeruginosa in Cystic Fibrosis
- What is being tested?
- TIS or TIP
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 6 sitesGermany
- Study dates
- 27 Jan 2015to 10 Apr 2017
- 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 CF
- Patients with elevated LCI of ≥ 7.5 at screening
- Patients with FEV1 of ≥ 50% predicted at screening
- Use of inhaled Tobramycin in a 28 days on / off regimen in the past 3 months before screening
- chronic lung Infection with Pseudomonas aeruginosa
Who cannot join
- Patients who are regularly receiving more than one class of inhaled anti-pseudomonal antibiotic
- Patients who have used oral or intravenous anti-pseudomonal antibiotics within 28 days prior to on-phase of study drug
- Pregnant or nursing (lactating) women
- Change in dose, formulation or strength of the study drug in the past treatment cycle before screening
- History of hearing loss or chronic tinnitus
- Infection with Burkholderia cenocepacia complex
- Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
Germany6 sites
Novartis Investigative Site
Dresden, 01307
Novartis Investigative Site
Erlangen, 91054
Novartis Investigative Site
Essen, 45147
Novartis Investigative Site
Frankfurt, 60590
Novartis Investigative Site
Germering, 82110
Novartis Investigative Site
Jena, 07740
About this study
Official title: An 8 Week Open-label Interventional Multicenter Study to Evaluate the Lung Clearance Index as Endpoint for Clinical Trials in Cystic Fibrosis Patients ≥ 6 Years of Age, Chronically Infected With Pseudomonas Aeruginosa
The purpose of the study was to evaluate lung clearance index (LCI) by a standardized procedure in a well characterized study setting and to assess feasibility of LCI as a more sensitive method than forced expiratory volume at 1 second (FEV1) to measure effectiveness of antibiotic therapy in patients with CF aged 6 years and older with mild to moderate lung disease.
Read the detailed description
This study was terminated prematurely. The reason for trial termination was challenge with enrollment and patient recruitment. A significant decrease in the eligible patient population had been identified as main driver.
- Being tested
- TIS or TIP
- Keywords
- Cystic Fibrosis, Pseudomonas aeruginosa, Lung Clearance Index, Multiple Breath Washout, Inhaled Tobramycin, Inhaled Antibiotics.
- Medical subject headings
- Cystic Fibrosis, Pseudomonas Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TIS or TIPDrug
300mg nebulized Tobramycin (Tobramycin inhalation solution(TIS)) or TOBI Podhaler (Tobramycin inhalation powder(TIP), equivalent dry powder)
| Study group | Role | What they receive |
|---|---|---|
| Tobramycin ALL | Experimental | 300mg nebulized Tobramycin (Tobramycin inhalation solution(TIS)) or TOBI Podhaler (Tobramycin inhalation powder(TIP), equivalent dry powder)twice a day (BID) 28days on / 28 days off Receives: Drug: TIS or TIP |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Lung Clearance Index (LCI) After 4 Weeks Following Onset of Study
The Lung Clearance Index (LCI), measured by Multiple Breath Washout of a tracer gas reflects the obstruction of airways in the lung. Wash-out was completed by definition at the time point when the inhaled gas concentration declined to 2.5% of its concentration at baseline. Washout took longer in patients with more severe disease as gas was trapped in narrowed airways (leading to a higher LCI). A LCI of 7.5 and below is normal.
Measured: Baseline, week 4
Secondary outcomes (6)
Change From Baseline of Forced Expiratory Volume at 1 Second (FEV1) After 4 Weeks Following Onset of Study
Change of FEV1 (Forced expiry volume in the first second) measured by Spirometry
Measured: Baseline, week 4
Change From Baseline of Colony-forming Units (CFU) After 4 Weeks Following Onset of Study
Microbacterial density of Pseudomonas aeruginosa in Sputum-Samples in CFU (Colony Forming Units) per gram sputum.
Measured: Baseline, week 4
Change From Baseline in Lung Clearance Index (LCI) After 1 Week
The Lung Clearance Index (LCI), measured by Multiple Breath Washout of a tracer gas reflects the obstruction of airways in the lung. Wash-out was completed by definition at the time point when the inhaled gas concentration declined to 2.5% of its concentration at baseline. Washout took longer in patients with more severe disease as gas was trapped in narrowed airways (leading to a higher LCI). A LCI of 7.5 and below is normal.
Measured: Baseline, week 1
Change of Lung Clearance Index (LCI) Between Week 4 (End of Study Drug Inhalation in the Current Treatment Cycle) and Week 8 (Prior to Start of Study Drug Inhalation in the Following Treatment Cycle)
The Lung Clearance Index (LCI), measured by Multiple Breath Washout of a tracer gas reflects the obstruction of airways in the lung. Wash-out was completed by definition at the time point when the inhaled gas concentration declined to 2.5% of its concentration at baseline. Washout took longer in patients with more severe disease as gas was trapped in narrowed airways (leading to a higher LCI). A LCI of 7.5 and below is normal.
Measured: week 4, week 8
Change of Forced Expiratory Volume at 1 Second(FEV1) Between Week 4 (End of Study Drug Inhalation in the Current Treatment Cycle) and Week 8 (Prior to Start of Study Drug Inhalation in the Following Treatment Cycle)
Change of FEV1 (Forced expiry volume in the first second) measured by Spirometry
Measured: week 4, week 8
Change of Colony-forming Units (CFU) Between Week 4 (End of Study Drug Inhalation in the Current Treatment Cycle) and Week 8 (Prior to Start of Study Drug Inhalation in the Following Treatment Cycle)
Microbacterial density of Pseudomonas aeruginosa in Sputum-Samples in CFU (Colony Forming Units) per gram sputum.
Measured: week 4, week 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Sep 2014 (estimated)
- Study start27 Jan 2015
- Primary completion10 Apr 2017
- Study completion10 Apr 2017
Why it stopped: Reason for termination was challenge with enrollment and recruitment. A significant decrease in the eligible patient population was main driver.
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic Lung Infection With Pseudomonas Aeruginosa in 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
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jörg Ziegler, PhD, Study director, Novartis Pharma GmbH, Nürnberg, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02248922
- Sponsor's protocol ID
- CTBM100CDE02
- Acronym
- ELIXIR
- Status last verified
- Jan 2018
- First submitted
- 22 Sep 2014
- Last updated
- 9 Oct 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Publications
Sutharsan S, Naehrig S, Mellies U, Sieder C, Ziegler J. An 8 week open-label interventional multicenter study to explore the lung clearance index as endpoint for clinical trials in cystic fibrosis patients >/=8 years of age, chronically infected with Pseudomonas aeruginosa. BMC Pulm Med. 2020 Jun 12;20(1):167. doi: 10.1186/s12890-020-01201-y.
PubMed 32532226
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An 8 Week Open-label Interventional Multicenter Study to Evaluate the Lung Clearance Index as Endpoint for Clinical Trials in Cystic Fibrosis Patients ≥ 6 Years of Age, Chronically Infected With Pseudomonas Aeruginosa
- ClinicalTrials.gov ID
- NCT02248922
- Acronym / sponsor ID
- ELIXIR / CTBM100CDE02
- Keywords
- Cystic Fibrosis, Pseudomonas aeruginosa, Lung Clearance Index, Multiple Breath Washout, Inhaled Tobramycin, Inhaled Antibiotics.
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Reason for termination was challenge with enrollment and recruitment. A significant decrease in the eligible patient population was main driver.
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 27 Jan 2015 (actual)
- Primary completion
- 10 Apr 2017 (actual)
- Study completion
- 10 Apr 2017 (actual)
- First posted
- 25 Sep 2014 (estimated)
- Results
- Posted 9 Oct 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study was to evaluate lung clearance index (LCI) by a standardized procedure in a well characterized study setting and to assess feasibility of LCI as a more sensitive method than forced expiratory volume at 1 second (FEV1) to measure effectiveness of antibiotic therapy in patients with CF aged 6 years and older with mild to moderate lung disease.
Detailed description
This study was terminated prematurely. The reason for trial termination was challenge with enrollment and patient recruitment. A significant decrease in the eligible patient population had been identified as main driver.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tobramycin ALL | Experimental | Drug: TIS or TIP300mg nebulized Tobramycin (Tobramycin inhalation solution(TIS)) or TOBI Podhaler (Tobramycin inhalation powder(TIP), equivalent dry powder)twice a day (BID) 28days on / 28 days off |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Confirmed diagnosis of CF
- Patients with elevated LCI of ≥ 7.5 at screening
- Patients with FEV1 of ≥ 50% predicted at screening
- Use of inhaled Tobramycin in a 28 days on / off regimen in the past 3 months before screening
- chronic lung Infection with Pseudomonas aeruginosa
Key exclusion criteria (7 listed)
- Patients who are regularly receiving more than one class of inhaled anti-pseudomonal antibiotic
- Patients who have used oral or intravenous anti-pseudomonal antibiotics within 28 days prior to on-phase of study drug
- Pregnant or nursing (lactating) women
- Change in dose, formulation or strength of the study drug in the past treatment cycle before screening
- History of hearing loss or chronic tinnitus
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 |
|---|---|---|
| TIS or TIP300mg nebulized Tobramycin (Tobramycin inhalation solution(TIS)) or TOBI Podhaler (Tobramycin inhalation powder(TIP), equivalent dry powder) | Drug | Tobramycin ALL |
Primary outcome
Change From Baseline in Lung Clearance Index (LCI) After 4 Weeks Following Onset of Study
Time frame: Baseline, week 4
The Lung Clearance Index (LCI), measured by Multiple Breath Washout of a tracer gas reflects the obstruction of airways in the lung. Wash-out was completed by definition at the time point when the inhaled gas concentration declined to 2.5% of its concentration at baseline. Washout took longer in patients with more severe disease as gas was trapped in narrowed airways (leading to a higher LCI). A LCI of 7.5 and below is normal.
6 secondary outcomes
Change From Baseline of Forced Expiratory Volume at 1 Second (FEV1) After 4 Weeks Following Onset of Study
Time frame: Baseline, week 4
Change From Baseline of Colony-forming Units (CFU) After 4 Weeks Following Onset of Study
Time frame: Baseline, week 4
Change From Baseline in Lung Clearance Index (LCI) After 1 Week
Time frame: Baseline, week 1
Change of Lung Clearance Index (LCI) Between Week 4 (End of Study Drug Inhalation in the Current Treatment Cycle) and Week 8 (Prior to Start of Study Drug Inhalation in the Following Treatment Cycle)
Time frame: week 4, week 8
Change of Forced Expiratory Volume at 1 Second(FEV1) Between Week 4 (End of Study Drug Inhalation in the Current Treatment Cycle) and Week 8 (Prior to Start of Study Drug Inhalation in the Following Treatment Cycle)
Time frame: week 4, week 8
Change of Colony-forming Units (CFU) Between Week 4 (End of Study Drug Inhalation in the Current Treatment Cycle) and Week 8 (Prior to Start of Study Drug Inhalation in the Following Treatment Cycle)
Time frame: week 4, week 8
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 32532226Sutharsan S, Naehrig S, Mellies U, Sieder C, Ziegler J. An 8 week open-label interventional multicenter study to explore the lung clearance index as endpoint for clinical trials in cystic fibrosis patients >/=8 years of age, chronically infected with Pseudomonas aeruginosa. BMC Pulm Med. 2020 Jun 12;20(1):167. doi: 10.1186/s12890-020-01201-y.
- Individual participant dataSharing planned: Undecided. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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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.
Jörg Ziegler, PhD study director
Novartis Pharma GmbH, Nürnberg, Germany
6 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 TIS or TIP in people with Chronic Lung Infection With Pseudomonas Aeruginosa in Cystic Fibrosis, run by Novartis Pharmaceuticals at 6 sites in Germany.
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 7 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 9 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Germany. 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 9 Oct 2018). 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 Lung Clearance Index (LCI) After 4 Weeks Following Onset of Study (time frame: Baseline, week 4). 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 NCT02248922, 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 9 Oct 2018.