NCT00006280

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).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
98

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Feb 2000

Geographic Reach
1 country

9 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

February 1, 2000

Completed
7 months until next milestone

First Submitted

Initial submission to the registry

September 11, 2000

Completed
1 day until next milestone

First Posted

Study publicly available on registry

September 12, 2000

Completed
1.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2002

Completed
Last Updated

March 2, 2010

Status Verified

March 1, 2010

First QC Date

September 11, 2000

Last Update Submit

March 1, 2010

Conditions

Keywords

Cystic Fibrosis

Interventions

Eligibility Criteria

Age6 Months - 5 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

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

Palo Alto, California, 94304, United States

Location

The Children's Hospital

Denver, Colorado, 80218, United States

Location

Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

Location

Children's Hospital

Boston, Massachusetts, 02115, United States

Location

University of North Carolina - Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Rainbow Babies and Children's Hospital

Cleveland, Ohio, 44106, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Children's Hospital and Regional Medical Center

Seattle, Washington, 98105, United States

Location

Related 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. doi: 10.1056/NEJM199901073400104.

    PMID: 9878641BACKGROUND

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

TobramycinInhalation

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

NebramycinKanamycinAminoglycosidesGlycosidesCarbohydratesRespiratory MechanicsRespirationRespiratory Physiological PhenomenaCirculatory and Respiratory Physiological Phenomena

Study Officials

  • Jeffrey Wagener, M.D.

    The Children's Hospital

    PRINCIPAL INVESTIGATOR
  • Richard Moss, M.D.

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

    PRINCIPAL INVESTIGATOR
  • Robert Wilmott, M.D.

    Children's Hospital & Medical Center

    PRINCIPAL INVESTIGATOR
  • Michael Konstan, M.D.

    Rainbow Babies and Children's Hospital

    PRINCIPAL INVESTIGATOR
  • Pamela Zeitlin, M.D., Ph.D.

    Johns Hopkins University

    PRINCIPAL INVESTIGATOR
  • David Waltz, M.D.

    Children's Hospital Medical Center, Cincinnati

    PRINCIPAL INVESTIGATOR
  • George Retsch-Bogart, M.D.

    University of North Carolina, Chapel Hill

    PRINCIPAL INVESTIGATOR
  • Peter Hiatt, M.D.

    Baylor College of Medicine

    PRINCIPAL INVESTIGATOR
  • Ronald Gibson, M.D., Ph.D.

    Children's Hospital Regional Medical Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Purpose
TREATMENT
Sponsor Type
NIH

Study Record Dates

First Submitted

September 11, 2000

First Posted

September 12, 2000

Study Start

February 1, 2000

Study Completion

February 1, 2002

Last Updated

March 2, 2010

Record last verified: 2010-03

Locations