A Study of the Safety and Efficacy of Tobramycin for Inhalation in Young Children With Cystic Fibrosis

Cystic Fibrosis Clinical Trial

Official title:

A Phase II Multicenter Randomized Trial of Tobramycin for Inhalation in Young Children With Cystic Fibrosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00006280
• Other study ID #: Inhaled Tobramycin (completed)
• Secondary ID: 1R01DK057755-01M
• Status: Completed
• Phase: Phase 2
• First received: September 11, 2000
• Last updated: March 1, 2010
• Start date: February 2000
• Est. completion date: February 2002

Study information

• Verified date: March 2010
• Source: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial Summary

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 98
• Est. completion date: February 2002
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Months to 5 Years

Eligibility

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.

Study Design

Allocation: Randomized, Masking: Double-Blind, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• Tobramycin for Inhalation

Locations

Country	Name	City	State
United States	Johns Hopkins Hospital	Baltimore	Maryland
United States	Children's Hospital	Boston	Massachusetts
United States	University of North Carolina - Chapel Hill	Chapel Hill	North Carolina
United States	Children's Hospital Medical Center	Cincinnati	Ohio
United States	Rainbow Babies and Children's Hospital	Cleveland	Ohio
United States	The Children's Hospital	Denver	Colorado
United States	Baylor College of Medicine	Houston	Texas
United States	Stanford University/Lucille Packard Children's Health Services at Stanford	Palo Alto	California
United States	Children's Hospital and Regional Medical Center	Seattle	Washington

Sponsors (2)

Lead Sponsor	Collaborator
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	National Center for Research Resources (NCRR)

Country where clinical trial is conducted

United States, 

References & Publications (1)

Ramsey BW, Pepe MS, Quan JM, Otto KL, Montgomery AB, Williams-Warren J, Vasiljev-K M, Borowitz D, Bowman CM, Marshall BC, Marshall S, Smith AL. Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. Cystic Fibrosis Inhaled Tobramycin Study Group. N Engl J Med. 1999 Jan 7;340(1):23-30. — View Citation