Cystic FibrosisNCT00004705
Study of Uridine Triphosphate (UTP) as an Aerosol Spray for Cystic Fibrosis
A study testing amiloride and uridine in people with Cystic Fibrosis, run by FDA Office of Orphan Products Development.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- amiloride and uridine
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Sep 1996to Jan 1999
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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
About this study
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.
Read the 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.
- Conditions
- Cystic Fibrosis
- Being tested
- amiloride, uridine
- Keywords
- cardiovascular and respiratory diseases, cystic fibrosis, genetic diseases and dysmorphic syndromes, rare disease
- Medical subject headings
- Cystic Fibrosis, Respiratory Tract Diseases, Genetic Diseases, Inborn, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
amilorideDrug
uridineDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2000 (estimated)
- Study startSep 1996
- Study completionJan 1999
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
Sponsor and investigators
- Lead sponsor
- FDA Office of Orphan Products Development
- Sponsor type
- US federal agency
- Collaborators
- University of North Carolina
- Investigators
- Michael R. Knowles, Study chair, University of North Carolina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004705
- Sponsor's protocol ID
- 199/13446
- Other IDs
- UNCCH-FDR001008
- Status last verified
- Mar 1999
- First submitted
- 24 Feb 2000
- Last updated
- 25 Mar 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Uridine Triphosphate (UTP) as an Aerosol Spray for Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT00004705
- Acronym / sponsor ID
- 199/13446
- Conditions
- Cystic Fibrosis
- Keywords
- cardiovascular and respiratory diseases, cystic fibrosis, genetic diseases and dysmorphic syndromes, rare disease
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- FDA Office of Orphan Products DevelopmentUS federal agency
- Collaborators
- University of North Carolina
- Study start
- Sep 1996
- Study completion
- Jan 1999
- First posted
- 25 Feb 2000 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| amiloride | Drug | – |
| uridine | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael R. Knowles study chair
University of North Carolina
Questions about this trial
What is this clinical trial studying?
A study testing amiloride and uridine in people with Cystic Fibrosis, run by FDA Office of Orphan Products Development.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2015.