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

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).

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

6 month–5 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Stanford University/Lucille Packard Children's Health Services at Stanford, Palo Alto, California, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Tobramycin for Inhalation

Drug

Sponsors and collaborators

Lead sponsor

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Nih

Collaborators

National Center for Research Resources (NCRR)

Registry information

Official study title

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

Important dates

Study start: 2000
Study completion: 2002
First posted: Sep 12, 2000
Registry last updated: Mar 2, 2010

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

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