Phase I Randomized Study of Adeno-Associated Virus-CFTR Vector in Patients With Cystic Fibrosis
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|ClinicalTrials.gov Identifier: NCT00004533|
Recruitment Status : Completed
First Posted : January 31, 2000
Last Update Posted : June 1, 2017
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.
|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis||Drug: Adeno-associated virus-CFTR vector||Phase 1|
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 :||Interventional (Clinical Trial)|
|Estimated Enrollment :||19 participants|
|Official Title:||Phase I Randomized Study of Adeno-Associated Virus-CFTR Vector in Patients With Cystic Fibrosis|
|Study Start Date :||August 1999|
|Primary Completion Date :||August 2002|
|Study Completion Date :||August 2002|
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004533
|Study Chair:||Terence Flotte||University of Florida|