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Phase I Randomized Study of Adeno-Associated Virus-CFTR Vector in Patients With Cystic Fibrosis

NCT00004533 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 19

Last updated 2017-06-01

No results posted yet for this study

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.

Conditions

Interventions

DRUG

Adeno-associated virus-CFTR vector

Sponsors & Collaborators

  • University of Florida

    collaborator OTHER

  • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

    lead NIH

Principal Investigators

  • Terence Flotte · University of Florida

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE

Eligibility

Min Age
15 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
1999-08-31
Primary Completion
2002-08-31
Completion
2002-08-31

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00004533 on ClinicalTrials.gov