Cystic Fibrosis Clinical Trial
Official title:
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.
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.
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