Efficacy and Safety of 28 or 56 Day Treatment for Pseudomonas Aeruginosa in Children With Cystic Fibrosis - Study Results

Efficacy and Safety of 28 or 56 Day Treatment for Pseudomonas Aeruginosa in Children With Cystic Fibrosis (ELITE)

This study has been completed.

Study NCT00391976 Information provided by Novartis

First Received on October 19, 2006. Last Updated on July 29, 2011 History of Changes

Results First Received: May 18, 2011

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Parallel Assignment; Masking: Open Label; Primary Purpose: Treatment
Condition:	Cystic Fibrosis
Intervention:	Drug: Tobramycin solution for inhalation 300 mg

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
123 patients were enrolled and received tobramycin 300 mg twice a day for 28 days. Patients who received treatment but tested positive for antibodies to any of 3 Pseudomonas aeruginosa exoenzymes in a blood sample collected at baseline were not randomized into one of the two treatment groups but were followed-up during routine clinic visits.

Reporting Groups

	Description
Tobramycin 300 mg for 28 Days	Patients inhaled tobramycin 300 mg twice a day for 28 days using the PARI LC PLUS™ jet nebulizer and a suitable compressor. The 2 daily doses were taken approximately 12 hours apart and no less than 6 hours apart.
Tobramycin 300 mg for 56 Days	Patients inhaled tobramycin 300 mg twice a day for 56 days using the PARI LC PLUS™ jet nebulizer and a suitable compressor. The 2 daily doses were taken approximately 12 hours apart and no less than 6 hours apart.
Non-randomized Patients	Patients who started the study and received tobramycin 300 mg twice a day for 28 days, but tested positive for antibodies to any of 3 Pseudomonas aeruginosa exoenzymes in a blood sample collected at baseline were not randomized into the treatment groups. These patients were not included in the efficacy analyses.

Participant Flow: Overall Study

	Tobramycin 300 mg for 28 Days	Tobramycin 300 mg for 56 Days	Non-randomized Patients
STARTED	45 ^[1]	43	35
Randomized	45 ^[2]	43	0
Safety Population	44 ^[3]	43	35
COMPLETED	18	18	0
NOT COMPLETED	27	25	35
Adverse Event	0	0	1
Withdrawal of consent	0	1	1
Lost to Follow-up	1	2	0
Inappropriate enrollment	1	1	0
Protocol deviation/violation	4	2	1
Recurrence/no eradication of infection	21	19	0
Unable to classify	0	0	1
Positive P. aeruginosa antibody test	0	0	31

[1]	Started indicates all patients who were enrolled at Visit 1
[2]	Randomization occurred after 28 days of treatment & negative antibody test aeruginosa antibody test
[3]	Patients who were enrolled and received at least one dose of study medication.

Baseline Characteristics

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Reporting Groups

	Description
Tobramycin 300 mg for 28 Days	Patients inhaled tobramycin 300 mg twice a day for 28 days using the PARI LC PLUS™ jet nebulizer and a suitable compressor. The 2 daily doses were taken approximately 12 hours apart and no less than 6 hours apart.
Tobramycin 300 mg for 56 Days	Patients inhaled tobramycin 300 mg twice a day for 56 days using the PARI LC PLUS™ jet nebulizer and a suitable compressor. The 2 daily doses were taken approximately 12 hours apart and no less than 6 hours apart.
Non-randomized Patients	Patients who started the study and received tobramycin 300 mg twice a day for 28 days, but tested positive for antibodies to any of 3 Pseudomonas aeruginosa exoenzymes in a blood sample collected at baseline were not randomized into the treatment groups. These patients were not included in the efficacy analyses.

Baseline Measures

	Tobramycin 300 mg for 28 Days	Tobramycin 300 mg for 56 Days	Non-randomized Patients	Total
Number of Participants [units: participants]	45	43	35	123
Age [units: years] Mean ± Standard Deviation	8.70 ± 7.22	8.65 ± 10.54	9.09 ± 5.76	8.79 ± 8.14
Age, Customized [units: participants]
6 months - < 6 years	19	18	11	48
6 years - < 18 years	20	21	21	62
≥ 18 years	6	4	3	13
Gender [units: participants]
Female	19	21	19	59
Male	26	22	16	64

Outcome Measures

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1. Primary:

Time to Recurrence of Pseudomonas (P.) Aeruginosa (Any Genotype) in Sputum or Deep Throat Cough Swab [ Time Frame: From 1 month after the end of treatment (Day 56 for the 28-day treatment group and Month 3 for the 56-day treatment group) until the end of the study (Month 27) ]

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2. Secondary:

Percentage of Patients With Pseudomonas (P.) Aeruginosa Eradicated From Deep Throat Cough Swab or Sputum [ Time Frame: From 1 month after the end of treatment until the end of the study (Month 27) ]

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3. Secondary:

Time to Recurrence of Pseudomonas (P.) Aeruginosa (New or Same Genotype) in Sputum or Deep Throat Cough Swab Based on Confirmatory Assessment by the Central Laboratory [ Time Frame: From 1 month after the end of treatment until the end of the study (Month 27) ]

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4. Secondary:

Percentage of Patients With Pseudomonas (P.) Aeruginosa Having an Increased, Decreased, or Unchanged Tobramycin Minimum Inhibitory Concentration (MIC) Value at the Final Visit Compared to Baseline [ Time Frame: From Baseline to the final visit (end of the study, Month 27) ]

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5. Secondary:

Number of Participants Hospitalized for Pulmonary Exacerbations [ Time Frame: From Baseline to end of study (27 months) ]

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Serious Adverse Events

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Other Adverse Events

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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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Results Point of Contact:

Name/Title: Study Director
Organization: Novartis Pharmaceuticals
phone: 862 778-8300

No publications provided by Novartis

Publications automatically indexed to this study:

Ratjen F, Munck A, Kho P, Angyalosi G; ELITE Study Group. Treatment of early Pseudomonas aeruginosa infection in patients with cystic fibrosis: the ELITE trial. Thorax. 2010 Apr;65(4):286-91. Epub 2009 Dec 8.

Responsible Party:	External Affairs, Novartis
ClinicalTrials.gov Identifier:	NCT00391976 History of Changes
Other Study ID Numbers:	CTBM100B2301
Study First Received:	October 19, 2006
Results First Received:	May 18, 2011
Last Updated:	July 29, 2011
Health Authority:	Austria: Agency for Health and Food Safety; Germany: Federal Institute for Drugs and Medical Devices; Spain: Spanish Agency of Medicines; France: Afssaps - French Health Products Safety Agency; United Kingdom: Medicines and Healthcare Products Regulatory Agency; Italy: National Institute of Health; Netherlands: Medicines Evaluation Board (MEB); Portugal: National Pharmacy and Medicines Institute