This study's primary goals are to test the safety and effectiveness of Tobramycin for Inhalation (TOBIr) in cystic fibrosis (CF) patients who are between 6 months and 6 years of age. This drug is an antibiotic that is inhaled into the lungs by the patient. It has already been studied and approved by the FDA for treatment of CF patients 6 years and older. Lung fluid will be examined for bacteria before and after the 28-day treatment. The amount of bacteria before and after treatment will be compared. This will indicate whether the antibiotic was effective in killing bacteria in the lungs. Once treatment begins, patients will be monitored every 2 weeks throughout the study (5 exams in 56 days). Half of the patients will receive TOBIr, half will receive a placebo (a substance that looks like TOBIr but contains no medication).

Condition	Treatment or Intervention	Phase
Cystic Fibrosis	Drug: Tobramycin for Inhalation	Phase II

Further Study Details: 

Expected Total Enrollment:  98

Ages Eligible for Study:  6 Months   -   5 Years,  Genders Eligible for Study:  Both

Criteria

Inclusion Criteria:

• Age at least 6 months and less than 6 years
• Diagnosis of cystic fibrosis with 2 clinical features consistent with CF and confirmed by either sweat chloride >= 60 mEq/L (by quantitative pilocarpine iontophoresis) or by genotype with 2 identifiable mutations consistent with CF.
• One throat or sputum microbiology culture positive for Pseudomonas aeruginosa (Pa) within 2 weeks to 12 months prior to screening.
• Informed consent by parent or legal guardian.

Exclusion Criteria:

• History of adverse reaction to anesthesia or sedation.
• History of aminoglycoside hypersensitivity.
• History of unresolved anemia (hematocrit < 30%) or thrombocytopenia (platelet count < 100,000/mm3).
• History of hemoptysis with 30 days prior to screening.
• History of abnormal renal function (serum creatinine > 1.5 times the upper limit of normal for age).
• History of clinically documented chronic hearing loss.
• Administration of any investigational drug within 30 days prior to screening.

California
      Stanford University/Lucille Packard Children's Health Services at Stanford, Palo Alto,  California,  94304,  United States
Colorado
      The Children's Hospital, Denver,  Colorado,  80218,  United States
Maryland
      Johns Hopkins Hospital, Baltimore,  Maryland,  21287,  United States
Massachusetts
      Children's Hospital, Boston,  Massachusetts,  02115,  United States
North Carolina
      University of North Carolina - Chapel Hill, Chapel Hill,  North Carolina,  27599,  United States
Ohio
      Children's Hospital Medical Center, Cincinnati,  Ohio,  45229,  United States
      Rainbow Babies and Children's Hospital, Cleveland,  Ohio,  44106,  United States
Texas
      Baylor College of Medicine, Houston,  Texas,  77030,  United States
Washington
      Children's Hospital and Regional Medical Center, Seattle,  Washington,  98105,  United States

Study chairs or principal investigators

Jeffrey Wagener, M.D.,  Principal Investigator,  The Children's Hospital   
Richard Moss, M.D.,  Principal Investigator,  Stanford University/Lucille Packard Children's Health Services at Stanford   
Robert Wilmott, M.D.,  Principal Investigator,  Children's Hospital Medical Center   
Michael Konstan, M.D.,  Principal Investigator,  Rainbow Babies and Children's Hospital   
Pamela Zeitlin, M.D., Ph.D.,  Principal Investigator,  Johns Hopkins University   
David Waltz, M.D.,  Principal Investigator,  Children's Hospital   
George Retsch-Bogart, M.D.,  Principal Investigator,  University of North Carolina   
Peter Hiatt, M.D.,  Principal Investigator,  Baylor College of Medicine   
Ronald Gibson, M.D., Ph.D.,  Principal Investigator,  Children's Hospital Regional Medical Center   

Study ID Numbers:  Inhaled Tobramycin in CF; 1-R01-DK57755-01; M01 RR 00037
Record last reviewed:  July 2003
Last Updated:  October 13, 2004
Record first received:  September 11, 2000
ClinicalTrials.gov Identifier:  NCT00006280
Health Authority: United States: Federal Government
ClinicalTrials.gov processed this record on 2005-04-08