Treatment of Early Pulmonary Infections With P. Aeruginosa in Cystic Fibrosis PatientsPhase 3EARLYNCT01082367

Randomized, Controlled Study of CF Patients Between 3 Months and Less Than 7 Years

A Phase 3 study testing TOBI in people with Treatment of Early Pulmonary Infections With P. Aeruginosa in Cystic Fibrosis Patients, run by Novartis Pharmaceuticals at 19 sites in 10 countries.

Completed

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

Official record

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Trial summary

This study investigated the efficacy of inhaled TOBI treatment for early infections of P. aeruginosa in paediatric patients with cystic fibrosis. Read more about this study

Who is it for?: Ages 3 months to 6 years
Conditions the study is for: Treatment of Early Pulmonary Infections With P. Aeruginosa in Cystic Fibrosis Patients
What is being tested?: TOBI
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 50 participants
Where: 19 sitesin 10 countries
Study dates: Apr 2010to Jun 2015
Phase: Phase 3
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 3 months to 6 years
All sexes
No healthy volunteers
Child

Who may be able to join

Diagnosis of cystic fibrosis
Early lower respiratory tract infection with P. aeruginosa,

Who cannot join

Known local or systemic hypersensitivity to aminoglycosides or inhaled antibiotics.
Administration of loop diuretics within 7 days prior to study drug administration.
Other protocol-defined inclusion/exclusion criteria may apply

Trial locations and contacts

19 sites in 10 countries.

Country

City, postcode or hospital

19 sites in 10 countries

Hungary4 sites

Novartis Investigative Site
Debrecen, Hungary, 4032
Novartis Investigative Site
Debrecen, 4043
Novartis Investigative Site
Kaposvár, Hungary, 7400
Novartis Investigative Site
Szeged, Hungary, 6725

Other trials in Hungary

Greece3 sites

Novartis Investigative Site
Pátrai, Greece, 265 00
Novartis Investigative Site
Thessaloniki, GR, 546 29
Novartis Investigative Site
Thessaloniki, GR, 546 42

Other trials in Greece

Russia3 sites

Novartis Investigative Site
Kazan', 420012
Novartis Investigative Site
Voronezh, 394036
Novartis Investigative Site
Yaroslavl, 150003

Other trials in Russia

Canada2 sites

Novartis Investigative Site
Montreal, Quebec, H3T1C5
Novartis Investigative Site
Toronto, Ontario, M5G 1X8

Other trials in Canada

France2 sites

Novartis Investigative Site
Bordeaux, 33076
Novartis Investigative Site
Paris, 75019

Other trials in France

Egypt1 site

Novartis Investigative Site
Alexandria, 21131

Other trials in Egypt

Germany1 site

Novartis Investigative Site
Dresden, 01307

Other trials in Germany

Italy1 site

Novartis Investigative Site
Milan, MI, 20122

Other trials in Italy

Romania1 site

Novartis Investigative Site
Bucharest, 20395

Other trials in Romania

Switzerland1 site

Novartis Investigative Site
Zurich, Switzerland, 8032

Other trials in Switzerland

About this study

Official title: A Randomized, Double-Blind, Placebo-Controlled, Crossover Multi-Center Study to Assess the Efficacy and Safety of Inhaled Tobramycin Nebuliser Solution (TOBI®) for the Treatment of Early Infections of P. Aeruginosa in Cystic Fibrosis Subjects Aged From 3 Months to Less Than 7 Years

This study investigated the efficacy of inhaled TOBI treatment for early infections of P. aeruginosa in paediatric patients with cystic fibrosis.

Conditions: Treatment of Early Pulmonary Infections With P. Aeruginosa in Cystic Fibrosis Patients
Being tested: TOBI, Placebo
Keywords: Tobramycin Inhalation solution, 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.

TOBIDrug
TOBI (tobramycin inhaled solution)
PlaceboDrug
Placebo

Study groups in NCT01082367
Study group	Role	What they receive
TOBI (tobramycin inhaled solution)/Placebo	Experimental	Participants randomized to TOBI received the investigational treatment for 28 days twice daily (bi)d in the first treatment cycle. At the end of first treatment cycle, participants who were positive for P. aeruginosa entered the open label (OL) phase of the study and received TOBI for 28 days bid. Participants who were negative for P. aeruginosa at the end of first treatment cycle and agreed to participate in the cross-over treatment period received placebo for 28 days bid (second treatment cycle). Receives: Drug: TOBI; Drug: Placebo
Placebo/TOBI	Placebo comparator	Participants randomized to placebo group received 0.9 % saline (NaCl) for 28 days bid in the first treatment cycle. At the end of first treatment cycle, participants who were positive for P. aeruginosa entered the OL phase of the study and received TOBI for 28 days bid. Participants who were negative for P. aeruginosa at the end of first treatment cycle and agreed to participate in the cross-over treatment period received TOBI for 28 days bid (second treatment cycle). Receives: Drug: TOBI; Drug: Placebo

What is being measured

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

Percentage of Participants P Aeruginosa-free After Completion of the First Treatment Cycle
Sputum/throat swab cultures were assessed.
Measured: Day 29

Secondary outcomes (2)

Percentage of Participants Free From P. Aeruginosa 28 Days After Termination of the Second Treatment Cycle
Sputum/throat swab cultures were assessed.
Measured: Day 91
Percentage of Participants P Aeruginosa-free at Termination of the Double Blind Period
Sputum/throat swab cultures were assessed.
Measured: Day 91

Timeline

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

Apr 2010Jun 2015

First posted8 Mar 2010 (estimated)
Study startApr 2010
Primary completionJun 2015
Study completionJun 2015

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 Treatment of Early Pulmonary Infections With P. Aeruginosa in Cystic Fibrosis PatientsStudies 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 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 50 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01082367
Sponsor's protocol ID: CTBM100C2304
Other IDs: 2009-016590-15, 2009
Acronym: EARLY
Status last verified: Jun 2016
First submitted: 5 Mar 2010
Last updated: 15 Jul 2016
Results posted: Yes, on ClinicalTrials.gov

Publications

Ratjen F, Moeller A, McKinney ML, Asherova I, Alon N, Maykut R, Angyalosi G; EARLY study group. Eradication of early P. aeruginosa infection in children <7 years of age with cystic fibrosis: The early study. J Cyst Fibros. 2019 Jan;18(1):78-85. doi: 10.1016/j.jcf.2018.04.002. Epub 2018 Apr 21.
PubMed 29685813

Study at a glance

From the registry record, last updated by the sponsor on 15 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized, Double-Blind, Placebo-Controlled, Crossover Multi-Center Study to Assess the Efficacy and Safety of Inhaled Tobramycin Nebuliser Solution (TOBI®) for the Treatment of Early Infections of P. Aeruginosa in Cystic Fibrosis Subjects Aged From 3 Months to Less Than 7 Years
ClinicalTrials.gov ID: NCT01082367
Acronym / sponsor ID: EARLY / CTBM100C2304
Conditions: Treatment of Early Pulmonary Infections With P. Aeruginosa in Cystic Fibrosis Patients
Keywords: Tobramycin Inhalation solution, Cystic fibrosis, Lung disease, anti-bacterial agents
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 50 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: Apr 2010
Primary completion: Jun 2015 (actual)
Study completion: Jun 2015 (actual)
First posted: 8 Mar 2010 (estimated)
Results: Posted 27 Apr 2016

Clinical summary

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

This study investigated the efficacy of inhaled TOBI treatment for early infections of P. aeruginosa in paediatric patients with cystic fibrosis.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Crossover
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
TOBI (tobramycin inhaled solution)/Placebo	Experimental	Drug: TOBI, Drug: PlaceboParticipants randomized to TOBI received the investigational treatment for 28 days twice daily (bi)d in the first treatment cycle. At the end of first treatment cycle, participants who were positive for P. aeruginosa entered the open label (OL) phase of the study and received TOBI for 28 days bid. Participants who were negative for P. aeruginosa at the end of first treatment cycle and agreed to participate in the cross-over treatment period received placebo for 28 days bid (second treatment cycle).
Placebo/TOBI	Placebo comparator	Drug: TOBI, Drug: PlaceboParticipants randomized to placebo group received 0.9 % saline (NaCl) for 28 days bid in the first treatment cycle. At the end of first treatment cycle, participants who were positive for P. aeruginosa entered the OL phase of the study and received TOBI for 28 days bid. Participants who were negative for P. aeruginosa at the end of first treatment cycle and agreed to participate in the cross-over treatment period received TOBI for 28 days bid (second treatment cycle).

Who may qualify

Ages: 3 months to 6 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (2 listed)

Diagnosis of cystic fibrosis
Early lower respiratory tract infection with P. aeruginosa,

Key exclusion criteria (3 listed)

Known local or systemic hypersensitivity to aminoglycosides or inhaled antibiotics.
Administration of loop diuretics within 7 days prior to study drug administration.
Other protocol-defined inclusion/exclusion criteria may apply

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
TOBITOBI (tobramycin inhaled solution)	Drug	Placebo/TOBI, TOBI (tobramycin inhaled solution)/Placebo
PlaceboPlacebo	Drug	Placebo/TOBI, TOBI (tobramycin inhaled solution)/Placebo

Primary outcome

Percentage of Participants P Aeruginosa-free After Completion of the First Treatment Cycle
Time frame: Day 29
Sputum/throat swab cultures were assessed.

2 secondary outcomes

Percentage of Participants Free From P. Aeruginosa 28 Days After Termination of the Second Treatment Cycle
Time frame: Day 91
Percentage of Participants P Aeruginosa-free at Termination of the Double Blind Period
Time frame: Day 91

Results and safety

Results posted to the registry on 27 Apr 2016, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
TOBI (Tobramycin Inhaled Solution)/Placebo	26	21
Placebo/TOBI	25	12

Primary outcome: Percentage of Participants P Aeruginosa-free After Completion of the First Treatment Cycle

Time frame: Day 29. Number. Unit: Percentage of participants

Measure	TOBI (Tobramycin Inhaled Solution)/Placebo n = 26	Placebo/TOBI n = 25
Value	84.6	24.0

TOBI (Tobramycin Inhaled Solution)/Placebo vs Placebo/TOBI: Odds Ratio (OR): 21.55 (95% CI 4.67 to 99.52), p <0.001, Regression, Logistic

Population analysed: Intent-to-treat (ITT): The ITT included all randomized participants who received at least dose of study treatment.

2 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
DB TOBI	0 of 32 (0%)	12 of 32 (37.5%)	Not reported
DB Placebo	1 of 38 (2.6%)	18 of 38 (47.4%)	Not reported
OL TOBI (Core)	0 of 22 (0%)	10 of 22 (45.5%)	Not reported
Off-treatment (Core)	2 of 50 (4%)	13 of 50 (26%)	Not reported
OL TOBI (Follow-up)	0 of 10 (0%)	2 of 10 (20%)	Not reported
Off-treatment (Follow-up)	4 of 29 (13.8%)	23 of 29 (79.3%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 15 Jul 2016.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 29685813Ratjen F, Moeller A, McKinney ML, Asherova I, Alon N, Maykut R, Angyalosi G; EARLY study group. Eradication of early P. aeruginosa infection in children <7 years of age with cystic fibrosis: The early study. J Cyst Fibros. 2019 Jan;18(1):78-85. doi: 10.1016/j.jcf.2018.04.002. Epub 2018 Apr 21.

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

19 sites in 10 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing TOBI in people with Treatment of Early Pulmonary Infections With P. Aeruginosa in Cystic Fibrosis Patients, run by Novartis Pharmaceuticals at 19 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 2 inclusion and 3 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 15 Jul 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 19 sites in Hungary, Greece, Russia, Canada, France 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. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 27 Apr 2016). 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 P Aeruginosa-free After Completion of the First Treatment Cycle (time frame: Day 29). 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 NCT01082367, 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 15 Jul 2016.