Phase I Study of the Third Generation Adenovirus H5.001CBCFTR in Patients With Cystic Fibrosis
Phase 1
Completed
- Conditions
- Cystic Fibrosis
- Registration Number
- NCT00004287
- Lead Sponsor
- National Center for Research Resources (NCRR)
- Brief Summary
OBJECTIVES:
I. Assess the safety and feasibility of gene transfer with the third generation adenovirus H5.001CBCFTR in patients with cystic fibrosis.
- Detailed Description
PROTOCOL OUTLINE: H5.001CBCFTR, an adenovirus vector containing the cystic fibrosis transmembrane conductance regulator gene, is administered endobronchially.
Cohort of 2 patients receive 1 of 6 H5.001CBCFTR concentrations. There is no intrapatient dose escalation.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 14
Inclusion Criteria
Not provided
Exclusion Criteria
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Related Research Topics
Explore scientific publications, clinical data analysis, treatment approaches, and expert-compiled information related to the mechanisms and outcomes of this trial. Click any topic for comprehensive research insights.
What molecular mechanisms does H5.001CBCFTR use to deliver the CFTR gene in cystic fibrosis patients?
How does the safety profile of third-generation adenovirus vectors compare to standard gene therapy approaches in cystic fibrosis?
What biomarkers are associated with successful gene transfer outcomes in NCT00004287 cystic fibrosis trials?
Are there combination therapies involving H5.001CBCFTR and CFTR modulators that improve treatment efficacy in cystic fibrosis?
What adverse events have been reported in adenovirus-based gene therapy trials for cystic fibrosis and how are they managed?