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

NCT ID: NCT00006280

Last Updated: 2010-03-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

98 participants

Study Classification

INTERVENTIONAL

Study Start Date

2000-02-29

Study Completion Date

2002-02-28

Brief Summary

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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).

Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Interventions

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Tobramycin for Inhalation

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

6 Months

Maximum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Center for Research Resources (NCRR)

NIH

Sponsor Role collaborator

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

NIH

Sponsor Role lead

Principal Investigators

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Jeffrey Wagener, M.D.

Role: PRINCIPAL_INVESTIGATOR

The Children's Hospital

Richard Moss, M.D.

Role: PRINCIPAL_INVESTIGATOR

Stanford University/Lucille Packard Children's Health Services at Stanford

Robert Wilmott, M.D.

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital & Medical Center

Michael Konstan, M.D.

Role: PRINCIPAL_INVESTIGATOR

Rainbow Babies and Children's Hospital

Pamela Zeitlin, M.D., Ph.D.

Role: PRINCIPAL_INVESTIGATOR

Johns Hopkins University

David Waltz, M.D.

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Medical Center, Cincinnati

George Retsch-Bogart, M.D.

Role: PRINCIPAL_INVESTIGATOR

University of North Carolina, Chapel Hill

Peter Hiatt, M.D.

Role: PRINCIPAL_INVESTIGATOR

Baylor College of Medicine

Ronald Gibson, M.D., Ph.D.

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Regional Medical Center

Locations

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Stanford University/Lucille Packard Children's Health Services at Stanford

Palo Alto, California, United States

Site Status

The Children's Hospital

Denver, Colorado, United States

Site Status

Johns Hopkins Hospital

Baltimore, Maryland, United States

Site Status

Children's Hospital

Boston, Massachusetts, United States

Site Status

University of North Carolina - Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Rainbow Babies and Children's Hospital

Cleveland, Ohio, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

Children's Hospital and Regional Medical Center

Seattle, Washington, United States

Site Status

Countries

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United States

References

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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.

Reference Type BACKGROUND
PMID: 9878641 (View on PubMed)

Other Identifiers

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1R01DK057755-01

Identifier Type: NIH

Identifier Source: secondary_id

View Link

M01RR000037

Identifier Type: NIH

Identifier Source: secondary_id

View Link

Inhaled Tobramycin (completed)

Identifier Type: -

Identifier Source: org_study_id

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