Study of Uridine Triphosphate (UTP) as an Aerosol Spray for Cystic Fibrosis
Study Details
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.
- Determine the acute safety and efficacy of aerosolized UTP in children with cystic fibrosis.
Condition or Disease | Intervention/Treatment | Phase |
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N/A |
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.
Study Design
Outcome Measures
Primary Outcome Measures
Eligibility Criteria
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
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- FDA Office of Orphan Products Development
- University of North Carolina
Investigators
- Study Chair: Michael R. Knowles, University of North Carolina
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 199/13446
- UNCCH-FDR001008