Clinical trial NCT00006280: A Study of the Safety and Efficacy of Tobramycin for Inhalation in Young Children With Cystic Fibrosis
Official title: A Phase II Multicenter Randomized Trial of Tobramycin for Inhalation in Young Children With Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sponsor class: NIH
Collaborators: name: National Center for Research Resources (NCRR)
First posted: 2000-09-12
Enrollment: 98
Sex: ALL
Minimum age (years): 6
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Primary purpose: TREATMENT

Brief 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).


Eligibility 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford University/Lucille Packard Children's Health Services at Stanford
state: California
city: Denver
country: United States
facility: The Children's Hospital
state: Colorado
city: Baltimore
country: United States
facility: Johns Hopkins Hospital
state: Maryland
city: Boston
country: United States
facility: Children's Hospital
state: Massachusetts
city: Chapel Hill
country: United States
facility: University of North Carolina - Chapel Hill
state: North Carolina
city: Cincinnati
country: United States
facility: Children's Hospital Medical Center
state: Ohio
city: Cleveland
country: United States
facility: Rainbow Babies and Children's Hospital
state: Ohio
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Seattle
country: United States
facility: Children's Hospital and Regional Medical Center
state: Washington
Study officials:
affiliation: The Children's Hospital
name: Jeffrey Wagener, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Stanford University/Lucille Packard Children's Health Services at Stanford
name: Richard Moss, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital & Medical Center
name: Robert Wilmott, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Rainbow Babies and Children's Hospital
name: Michael Konstan, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Johns Hopkins University
name: Pamela Zeitlin, M.D., Ph.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital Medical Center, Cincinnati
name: David Waltz, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: University of North Carolina, Chapel Hill
name: George Retsch-Bogart, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Baylor College of Medicine
name: Peter Hiatt, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital Regional Medical Center
name: Ronald Gibson, M.D., Ph.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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. doi: 10.1056/NEJM199901073400104.