Cystic FibrosisPhase 1NCT00004806
Phase I Study of Liposome-Mediated Gene Transfer in Patients With Cystic Fibrosis
A Phase 1 study testing Cystic fibrosis transmembrane conductance regulator in people with Cystic Fibrosis, run by University of Alabama at Birmingham.
Trial summary
OBJECTIVES: Evaluate the efficacy and safety of lipid-mediated transfer of the cystic fibrosis transmembrane conductance regulator gene to nasal epithelium in patients with cystic fibrosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Cystic fibrosis transmembrane conductance regulator
- How many people
- 9 participants
- Where
- No sites listed yet
- Study dates
- Jun 1995to Nov 2002
- Phase
- Phase 1
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics-- Cystic fibrosis (CF), i.e.: Sweat chloride greater than 60 mEq/L Clinical manifestations of CF Homozygous delta F508 mutation preferred Compound heterozygotes for F508 mutation with pancreatic insufficiency eligible FEV1 greater than 40% of predicted PO2 at least 60 mm Hg on room air No acute or recurrent sinusitis No obstructive nasal polyposis No pneumothorax or hemoptysis, e.g., more than 250 mL blood within 24-hour period, within past year No unstable lung disease with worsening pulmonary symptoms, arterial blood gas, or pulmonary function tests within 2 weeks prior to entry No pulmonary exacerbation within 4 weeks prior to entry --Prior/Concurrent Therapy-- At least 2 months since participation in any other clinical study At least 2 months since DNase At least 4 months since corticosteroids --Patient Characteristics-- Cardiac: No severe cardiac disease Other: No other severe organ system disease, e.g., juvenile-type diabetes mellitus No pregnant women
About this study
OBJECTIVES:
Evaluate the efficacy and safety of lipid-mediated transfer of the cystic fibrosis transmembrane conductance regulator gene to nasal epithelium in patients with cystic fibrosis.
Read the detailed description
PROTOCOL OUTLINE: Cystic fibrosis transmembrane conductance regulator (CFTR) gene complexed with lipid is administered intranasally to the right inferior turbinate. Lipid without CFTR is administered to the left nostril as a control.
Cohorts of 3 patients are given successively lower doses of CFTR, each dose complexed with 500 micrograms of lipid.
Patients are followed at days 2-12, 15, and 21, then every 3-4 days for 3 weeks, every 2-3 weeks for 10 weeks, and every 3 months thereafter.
- Conditions
- Cystic Fibrosis
- Being tested
- Cystic fibrosis transmembrane conductance regulator
- 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.
Cystic fibrosis transmembrane conductance regulatorGenetic
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 startJun 1995
- Primary completionNov 2002
- Study completionNov 2002
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.
- More trials run by University of Alabama at BirminghamThe sponsor's other registered studies.
- 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 1
- Primary purpose
- Treatment
- Enrolment
- 9 participants
Sponsor and investigators
- Lead sponsor
- University of Alabama at Birmingham
- Sponsor type
- University, hospital or other
- Investigators
- Eric J. Sorscher, Study chair, University of Alabama at Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004806
- Sponsor's protocol ID
- 199/11983
- Other IDs
- UAB-11983
- Status last verified
- Mar 2011
- First submitted
- 24 Feb 2000
- Last updated
- 30 Mar 2011
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Study of Liposome-Mediated Gene Transfer in Patients With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT00004806
- Acronym / sponsor ID
- 199/11983
- Conditions
- Cystic Fibrosis
- Keywords
- cardiovascular and respiratory diseases, cystic fibrosis, genetic diseases and dysmorphic syndromes, rare disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants
- Lead sponsor
- University of Alabama at BirminghamUniversity, hospital or other
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study start
- Jun 1995
- Primary completion
- Nov 2002 (actual)
- Study completion
- Nov 2002 (actual)
- 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:
Evaluate the efficacy and safety of lipid-mediated transfer of the cystic fibrosis transmembrane conductance regulator gene to nasal epithelium in patients with cystic fibrosis.
Detailed description
PROTOCOL OUTLINE: Cystic fibrosis transmembrane conductance regulator (CFTR) gene complexed with lipid is administered intranasally to the right inferior turbinate. Lipid without CFTR is administered to the left nostril as a control.
Cohorts of 3 patients are given successively lower doses of CFTR, each dose complexed with 500 micrograms of lipid.
Patients are followed at days 2-12, 15, and 21, then every 3-4 days for 3 weeks, every 2-3 weeks for 10 weeks, and every 3 months thereafter.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- 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 |
|---|---|---|
| Cystic fibrosis transmembrane conductance regulator | Genetic | – |
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 30 Mar 2011.
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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.
Eric J. Sorscher study chair
University of Alabama at Birmingham
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Cystic fibrosis transmembrane conductance regulator in people with Cystic Fibrosis, run by University of Alabama at Birmingham.
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 30 Mar 2011. 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 30 Mar 2011.