Cystic Fibrosis Clinical Trial
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.
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.
;
Primary Purpose: Treatment
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