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Study of Uridine Triphosphate (UTP) as an Aerosol Spray for Cystic Fibrosis

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
amiloride
uridine
Sponsored by
FDA Office of Orphan Products Development
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis focused on measuring cardiovascular and respiratory diseases, cystic fibrosis, genetic diseases and dysmorphic syndromes, rare disease

Eligibility Criteria

4 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Diagnosis of mild to moderate cystic fibrosis Small production of daily airway secretions Stable pulmonary course --Prior/Concurrent Therapy-- Radiotherapy: No radiation within 12 months to cause patient to exceed annual limits Other: No chronic medication for reactive airways disease At least 12 hours since inhaled beta-adrenergic agonists At least 24 hours since systemic theophylline --Patient Characteristics-- FEV1 greater than 50% predicted Other: Not pregnant Must perform reproducible spirometry

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    February 24, 2000
    Last Updated
    March 24, 2015
    Sponsor
    FDA Office of Orphan Products Development
    Collaborators
    University of North Carolina
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00004705
    Brief Title
    Study of Uridine Triphosphate (UTP) as an Aerosol Spray for Cystic Fibrosis
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    March 1999
    Overall Recruitment Status
    Completed
    Study Start Date
    September 1996 (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    January 1999 (undefined)

    3. Sponsor/Collaborators

    Name of the Sponsor
    FDA Office of Orphan Products Development
    Collaborators
    University of North Carolina

    4. Oversight

    5. Study Description

    Brief Summary
    OBJECTIVES: I. Determine the stability of uridine triphosphate (UTP) and examine the metabolism of exogenous nucleotides on airway epithelial surfaces in patients with cystic fibrosis. II. Determine the acute safety and efficacy of aerosolized UTP in children with cystic fibrosis.
    Detailed Description
    PROTOCOL OUTLINE: Patients may be treated on any of three different regimens. Patients may be treated on more than one regimen, if they meet the eligibility requirements. Patients on regimen A are adults and receive uridine triphosphate (UTP) by inhalation, followed immediately by bronchoscopy. Patients on regimen B are children, aged 4 to 10 years. Patients receive up to 4 graded doses of UTP by inhalation on day 1. On day 2, patients receive a single dose of UTP. Patients receive amiloride followed by UTP by inhalation on day 3. Patients on regimen C are children, aged 4 to 18 years. Patients inhale a radiolabelled (technetium 99m) monodisperse iron oxide aerosol. Radiation deposited in the patient's lungs is monitored. Patients are randomized to receive one of 4 different aerosols (vehicle; UTP; amiloride; or UTP plus amiloride), which is inhaled for 20 minutes. Patients are followed 24 hours after aerosol exposure. Regimen D is a dose escalation study in which patients are aged 9 to 40 years. Patients receive either the vehicle or UTP by inhalation 3 times daily for 3 days. Cohorts of 4 patients each are entered at each dose level.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Cystic Fibrosis
    Keywords
    cardiovascular and respiratory diseases, cystic fibrosis, genetic diseases and dysmorphic syndromes, rare disease

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Not Applicable

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    amiloride
    Intervention Type
    Drug
    Intervention Name(s)
    uridine

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    4 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Diagnosis of mild to moderate cystic fibrosis Small production of daily airway secretions Stable pulmonary course --Prior/Concurrent Therapy-- Radiotherapy: No radiation within 12 months to cause patient to exceed annual limits Other: No chronic medication for reactive airways disease At least 12 hours since inhaled beta-adrenergic agonists At least 24 hours since systemic theophylline --Patient Characteristics-- FEV1 greater than 50% predicted Other: Not pregnant Must perform reproducible spirometry
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Michael R. Knowles
    Organizational Affiliation
    University of North Carolina
    Official's Role
    Study Chair

    12. IPD Sharing Statement

    Learn more about this trial

    Study of Uridine Triphosphate (UTP) as an Aerosol Spray for Cystic Fibrosis

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