- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00004533
Phase I Randomized Study of Adeno-Associated Virus-CFTR Vector in Patients With Cystic Fibrosis
OBJECTIVES:
I. Determine the maximum tolerated dose of recombinant adeno-associated virus-CFTR vector in patients with cystic fibrosis.
II. Assess the safety of this gene therapy in these patients.
III. Assess the in vivo gene transfer of this vector in these patients.
IV. Assess the CFTR gene expression and physiologic activity following gene transfer in these patients.
V. Assess the clinical impact of CFTR gene expression following gene transfer in these patients.
VI. Monitor patient immune response directed against CFTR or vector components following vector administration.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
PROTOCOL OUTLINE:
This is a randomized, dose escalation, double blind, placebo controlled, multicenter study. Patients are randomized to receive either adeno-associated virus-CFTR (AAV-CFTR) vector or placebo.
Patients undergo tests on days -10 to -4 to rule out adenovirus and adeno-associated virus infections. Patients then receive AAV-CFTR vector intranasally to the right or left inferior turbinates and placebo to the other side of the nose. The next day, patients receive an endobronchial dose of AAV-CFTR vector to the superior segment of the right lower lobe.
Cohorts of 2-4 patients each receive escalating doses of AAV-CFTR vector until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose level immediately preceding the dose at which at least 2 subjects develop dose limiting toxicity.
Patients are followed at day 10, then at 1, 2, 3, 6, 9, and 12 months.
Study Type
Enrollment
Phase
- Phase 1
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Diagnosis of cystic fibrosis (CF) with mild to moderate lung disease; FVC at least 60% predicted; Schwachman-Kulczycki score at least 65; Sweat chloride greater than 60 mEq/L
All CF genotypes allowed
No sputum colonization with Burkholderia cepacia or another multiply resistant organism
No recurrent or intermittent hemoptysis; At least 1 year since significant hemoptysis requiring transfusion or embolization OR Coughing of greater than 30 mL of blood more often than once a week
--Prior/Concurrent Therapy--
At least 30 days since hospitalization or home intravenous antibiotic therapy for pulmonary exacerbation
No concurrent investigational drugs or therapy
--Patient Characteristics--
Other: No concurrent cigarette smoking; Not pregnant or nursing; Negative pregnancy test; Fertile patients must use effective contraception
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Masking: Double
Collaborators and Investigators
Collaborators
Investigators
- Study Chair: Terence Flotte, University of Florida
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 199/14137
- UF-G-037-1996 (Other Identifier: University of Florida)
- TGC-AAV-9502
- P01DK058327 (U.S. NIH Grant/Contract)
- R01DK051809 (U.S. NIH Grant/Contract)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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