Phase I Randomized Study of Adeno-Associated Virus-CFTR Vector in Patients With Cystic Fibrosis
NCT ID: NCT00004533
Last Updated: 2017-06-01
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1
19 participants
INTERVENTIONAL
1999-08-31
2002-08-31
Brief Summary
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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.
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Detailed Description
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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.
Conditions
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Study Design
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RANDOMIZED
TREATMENT
DOUBLE
Interventions
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Adeno-associated virus-CFTR vector
Eligibility Criteria
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Inclusion 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
15 Years
ALL
No
Sponsors
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University of Florida
OTHER
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Responsible Party
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Principal Investigators
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Terence Flotte
Role: STUDY_CHAIR
University of Florida
References
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Flotte TR, Zeitlin PL, Reynolds TC, Heald AE, Pedersen P, Beck S, Conrad CK, Brass-Ernst L, Humphries M, Sullivan K, Wetzel R, Taylor G, Carter BJ, Guggino WB. Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study. Hum Gene Ther. 2003 Jul 20;14(11):1079-88. doi: 10.1089/104303403322124792.
Other Identifiers
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UF-G-037-1996
Identifier Type: OTHER
Identifier Source: secondary_id
TGC-AAV-9502
Identifier Type: -
Identifier Source: secondary_id
199/14137
Identifier Type: -
Identifier Source: org_study_id
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