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

This study is currently recruiting patients.

Sponsored by: Targeted Genetics Coporation
Cystic Fibrosis Foundation
Information provided by: Targeted Genetics Coporation

Purpose

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).

Condition Treatment or Intervention Phase
Cystic Fibrosis
  Gene Transfer: tgAAVCF
 Phase II

MedlinePlus related topics:  Cystic Fibrosis
Genetics Home Reference related topics:  cystic fibrosis

Study Type: Interventional
Study Design: Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study

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

Further Study Details: 

Expected Total Enrollment:  100

Study start: June 2003

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.

Eligibility

Ages Eligible for Study:  12 Years and above,  Genders Eligible for Study:  Both

Criteria

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.

Location and Contact Information


Alabama
      UAB-Childrens Health System, Birmingham,  Alabama,  35233,  United States; Recruiting
Valerie Eubanks  205-939-5743    vaeubanks@peds.uab.edu 
John P Clancy, MD,  Principal Investigator

California
      Stanford University Medical Center, Palo Alto,  California,  94304,  United States; Recruiting
Zoe Davies, PNP  650-498-5315    zdavies@leland.stanford.edu 
Colleen Dunn  650-736-0388    cedunn@leland.stanford.edu 
Richard B Moss, MD,  Principal Investigator

      UC San Diego, San Diego,  California,  92123,  United States; Not yet recruiting
Sarah Holland, RN  858-966-8153    sholland@chsd.org 
Mark Pian, MD,  Principal Investigator

Colorado
      University of Colorado-The Childrens Hospital, Denver,  Colorado,  80209,  United States; Recruiting
Sarah Brayshaw, RN  303-398-1263    brayshaws@njc.org 
Joni Koenig  303-266-9363    Koenig.Joni@tchden.org 
Frank Accurso, MD,  Principal Investigator

Florida
      University of Florida, Gainesville,  Florida,  32610,  United States; Recruiting
Marg Humphries, RN  352-846-2286    humphmr@mbi-00.mbi.ufl.edu 
Terry Spencer, MD,  Principal Investigator

Maryland
      The Johns Hopkins Hospital, Baltimore,  Maryland,  21287,  United States; Recruiting
Lois Brass, RN  410-955-9782    lbernst@jhmi.edu 
Barbara Coleman, RN  410-955-1167 
Pamela Zeitlin, MD,  Principal Investigator

Massachusetts
      Childrens Hospital, Boston,  Massachusetts,  02115,  United States; Recruiting
Dana Dorman, RN  617-355-3197    dana.dorma@TCH.Harvard.edu 
David Waltz, MD,  Principal Investigator

      Mass General Hospital, Boston,  Massachusetts,  02114,  United States; Recruiting
Monica Ulles, RN, PNP  617-726-2804    mulles@partners.org 
Henry Dorkin, MD,  Principal Investigator

Minnesota
      The Minnesota CF Center, Minneapolis,  Minnesota,  55455,  United States; Recruiting
Brooke Noren, RN  612-625-7995    boer0039@umn.edu 
Jackie Zirbes, RN  612-626-5896    Zirbe001@umn.edu 
Carlos Milla, MD,  Principal Investigator

Nebraska
      University of Nebraska, Omaha,  Nebraska,  68198,  United States; Recruiting
Dee Acquazzino  402-559-6275    dracquaz@unmc.edu 
John Colombo, MD,  Principal Investigator

Pennsylvania
      University of Pittsburgh Medical Center, Pittsburgh,  Pennsylvania,  15213,  United States; Recruiting
Elizbeth Hartigan, MPH, RN  412-692-7060    Elizabeth.hartigan@chp.edu 
Joseph Pilewski, MD,  Principal Investigator

More Information

Study ID Numbers:  25E01; RAC-0301-569
Record last reviewed:  October 2004
Record first received:  November 21, 2003
ClinicalTrials.gov Identifier:  NCT00073463
Health Authority: United States: Food and Drug Administration
ClinicalTrials.gov processed this record on 2004-11-16
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