Phase I Randomized Study of Adeno-Associated Virus-CFTR Vector in Patients With Cystic Fibrosis
Status:
Completed
Trial end date:
2002-08-01
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 1
Details
Lead Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)