Safety and Efficacy of Recombinant Adeno-Associated Virus Containing the CFTR Gene in the Treatment of Cystic Fibrosis

January 24, 2008 updated by: Targeted Genetics Corporation

A Multicenter, Double-Blind, Placebo Controlled, Phase II Study of Aerosolized tgAAVCF for the Treatment of Cystic Fibrosis

The purpose of this study is to confirm the improvement in pulmonary function and cytokine levels observed in the recently completed multidose aerosol study for the treatment of Cystic Fibrosis (CF).

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

Cystic Fibrosis is an autosomal recessive disorder with an incidence of approximately 1 in 33000 live births. It is due to defects in the CFTR gene, which is located on chromosome 7. Gene Therapy holds the promise of addressing the primary defect in CF by reconstituting the CFTR function in the lung. tgAAVCF, which has been genetically engineered to contain the CFTR gene, has been extremely well tolerated following single and multiple dose administrations to the nose, sinus, and lung. Dose-dependent gene transfer has been demonstrated. Although vector gene expression has not been detected, evidence consistent with biological activity was observed in maxillary sinus study, and statistically significant changes in the FEV1 and IL-8 levels were observed in the recently completed multidose aerosol study. These findings are worthy of further investigation.

Study Type

Interventional

Enrollment

100

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • UAB-Childrens Health System
    • California
      • Palo Alto, California, United States, 94304
        • Stanford University Medical Center
      • San Diego, California, United States, 92123
        • UC San Diego
    • Colorado
      • Denver, Colorado, United States, 80209
        • University of Colorado-The Childrens Hospital
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • The Johns Hopkins Hospital
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Childrens Hospital
      • Boston, Massachusetts, United States, 02114
        • Mass General Hospital
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • The Minnesota CF Center
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • University of Nebraska
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • University of Pittsburgh Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Eligible subjects will be randomized to two aerosolized doses of either tgAAVCF or placebo 30 days apart. Subjects will undergo pulmonary function testing every two weeks during the active portion of the study (three months) and will be followed for safety for a total of seven months.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Targeted Genetics Corporation

Collaborators

Cystic Fibrosis Foundation

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2003

Study Completion

October 1, 2005

Study Registration Dates

First Submitted

November 21, 2003

First Submitted That Met QC Criteria

November 24, 2003

First Posted (Estimate)

November 25, 2003

Study Record Updates

Last Update Posted (Estimate)

January 28, 2008

Last Update Submitted That Met QC Criteria

January 24, 2008

Last Verified

January 1, 2008

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 25E01
  • RAC-0301-569

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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