Cystic FibrosisPhase 1NCT00004471
Phase I Pilot Study of Gene Therapy for Cystic Fibrosis Using Cationic Liposome Mediated Gene Transfer
A Phase 1 study testing pGT-1 gene lipid complex in people with Cystic Fibrosis, run by University of Alabama at Birmingham.
Trial summary
OBJECTIVES: Determine whether copies of the cystic fibrosis gene (pGT-1) can be delivered to the cells lining the nose of cystic fibrosis patients using cationic liposome (DMRIE/DOPE) mediated gene transfer. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- pGT-1 gene lipid complex
- How many people
- 9 participants
- Where
- No sites listed yet
- Study dates
- Aug 1995to May 2001
- 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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
- Cystic fibrosis patients
- Not pregnant Fertile patients must use effective contraception
About this study
OBJECTIVES:
Determine whether copies of the cystic fibrosis gene (pGT-1) can be delivered to the cells lining the nose of cystic fibrosis patients using cationic liposome (DMRIE/DOPE) mediated gene transfer.
Read the detailed description
PROTOCOL OUTLINE: Under direct visualization, patients receive lipid/DNA formulation (pGT-1 lipid complex) by syringe instillation over 30 minutes to the right inferior nasal turbinate.
Patients are followed daily for 7 days, twice a week for 3 weeks, every 2-3 weeks for 10 weeks, then every 3 months thereafter.
- Conditions
- Cystic Fibrosis
- Being tested
- pGT-1 gene lipid complex
- 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.
pGT-1 gene lipid complexGenetic
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 1999 (estimated)
- Study startAug 1995
- Primary completionMay 2001
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
- NCT00004471
- Sponsor's protocol ID
- 199/13941
- Other IDs
- UAB-6097, UAB-F930923001
- Status last verified
- Mar 2011
- First submitted
- 18 Oct 1999
- Last updated
- 29 Mar 2011
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Mar 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Pilot Study of Gene Therapy for Cystic Fibrosis Using Cationic Liposome Mediated Gene Transfer
- ClinicalTrials.gov ID
- NCT00004471
- Acronym / sponsor ID
- 199/13941
- 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
- Study start
- Aug 1995
- Primary completion
- May 2001 (actual)
- First posted
- 19 Oct 1999 (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:
Determine whether copies of the cystic fibrosis gene (pGT-1) can be delivered to the cells lining the nose of cystic fibrosis patients using cationic liposome (DMRIE/DOPE) mediated gene transfer.
Detailed description
PROTOCOL OUTLINE: Under direct visualization, patients receive lipid/DNA formulation (pGT-1 lipid complex) by syringe instillation over 30 minutes to the right inferior nasal turbinate.
Patients are followed daily for 7 days, twice a week for 3 weeks, every 2-3 weeks for 10 weeks, then every 3 months thereafter.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 16 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 |
|---|---|---|
| pGT-1 gene lipid complex | 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 29 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 pGT-1 gene lipid complex 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 29 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 29 Mar 2011.