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Phase 4 N=157 Treatment

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

Pulmonary Infections · Pseudomonas Aeruginosa in Cystic Fibrosis

Enrolled (actual)
157
Serious AEs
31.2%
Results posted
Feb 2015
Primary outcome: Primary: Percentage of Participants With Treatment Emergent Adverse Events, Serious Adverse Events (SAEs) and Deaths — 85.4; 31.2; 0.0 Percentage of participants

Study Design & Population

Study type
Interventional
Phase
Phase 4
Interventions
TBM100 (Drug)
Age
Pediatric, Adult, Older Adult · 6+ yrs
Sex
All
Sponsor
Novartis Pharmaceuticals
Primary completion
Jan 2014

Outcome Measures

OutcomeResultp-value
PRIMARY
Percentage of Participants With Treatment Emergent Adverse Events, Serious Adverse Events (SAEs) and Deaths
85.4; 31.2; 0.0
SECONDARY
Relative Change From Baseline in Forced Expiratory Volume in One Second (FEV1) Percent Predicted
0.8; 0.0; 0.2; -0.2; -1.5; -1.9
SECONDARY
Relative Change From Baseline in FVC Percent Predicted
-2.5; -2.8; -2.1; -1.8; -3.5; -3.1
SECONDARY
Relative Change From Baseline in FEF Rate Over 25 to 75 Percent of Vital Capacity Predicted
10.3; 9.4; 5.5; 6.0; 2.9; 4.3
SECONDARY
Change From Baseline in Pseudomonas Aeruginosa Colony Forming Units in Sputum
-1.6; -1.1; -1.2; -1.1; -1.3; -1.2
SECONDARY
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
2; 2; 2; 2; 2; 2
SECONDARY
Percentage of Participants Hospitalized Due to Serious Respiratory-related Adverse Events
26.8
SECONDARY
Number of Hospitalization Days Due to Serious Respiratory-related Adverse Events
18.1
SECONDARY
Time to First Hospitalization Due to Serious Respiratory-related Adverse Events
NA
SECONDARY
Percentage of Participants Who Used New Anti-pseudomonal Antibiotics
65.6
SECONDARY
Number of Days of New Anti-pseudomonal Antibiotic Use
33.1
SECONDARY
Time to Use of New Anti-pseudomonal Antibiotic
136

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.

Eligibility Criteria

Inclusion Criteria

  • 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

Exclusion Criteria

  • 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
  • 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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01519661). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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