Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial.

Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial. Research Abstract Details 

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Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial. Abstract Text:

Richard B Moss,Carlos Milla,John Colombo,Frank Accurso,Pamela L Zeitlin,John P Clancy,L Terry Spencer,Joseph Pilewski,David A Waltz,Henry L Dorkin,Thomas Ferkol,Mark Pian,Bonnie Ramsey,Barrie J Carter,Dana B Martin,Alison E Heald,

Previous studies have demonstrated that delivery of a recombinant adeno-associated virus (AAV) vector encoding the complete human cystic fibrosis transmembrane regulator (CFTR) cDNA (tgAAVCF) to the nose, sinus, and lungs of subjects with cystic fibrosis (CF) was safe and well tolerated. In a small randomized, double-blind study of three doses of aerosolized tgAAVCF or placebo at 30-day intervals, encouraging but non-significant trends in pulmonary function and induced sputum interleukin 8 (IL-8) levels were seen at early time points. This larger study was conducted to verify these trends. One hundred and two subjects aged 12 years and older with mild-to-moderate cystic fibrosis (forced expiratory flow in 1 sec [FEV1]:60% predicted) were randomized to two aerosolized doses of 1x10(13)DNase-resistant particles of tgAAVCF (n=51) or matching placebo (n=51) administered 30 days apart. Although tgAAVCF was well tolerated, the study did not meet its primary efficacy end point of statistically significant improvement in FEV1 30 days after initial administration of tgAAVCF compared with placebo. There were no significant differences in spirometric lung function over time, induced sputum biologic markers, or days of antibiotic use in either treatment group. Thus repeated doses of aerosolized tgAAVCF were safe and well tolerated, but did not result in significant improvement in lung function over time. Because gene transfer is the simplest, most basic way to correct the underlying genetic defect that leads to disease in CF, further research is warranted to develop an effective gene transfer agent for the treatment of CF.

Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial. Publishing Authors By Initials

RB Moss,C Milla,J Colombo,F Accurso,PL Zeitlin,JP Clancy,LT Spencer,J Pilewski,DA Waltz,HL Dorkin,T Ferkol,M Pian,B Ramsey,BJ Carter,DB Martin,AE Heald,

For similar pharmaceutical preparations: placebos research abstracts see: pharmaceutical preparations: placebos research

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Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial. Journal Published:

PUBLICATION TYPE: Research Support, Non-U.S. Gov

Journal: Human gene therapy

VOLUME: 18

Page Numbers: 726-32

Journal Abbreviation: Hum. Gene Ther.

ISSN: 1043-0342

DAY: 3

MONTH: Aug

YEAR: 2007

Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial. Information

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LANGUAGE: eng

NlmUniqueID: 9008950

Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial. Keywords Mesh Terms:

KEYWORDS: Placebos

MESH TERMS: genetics

Chemical & Substance for Abstract: Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial. Information

Substance Name: Cystic Fibrosis Transmembrane Conductanc

Registry Number: 126880-72-6

Grant and Affiliation Information for Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial.

AFFILIATION: Department of Pediatrics, Stanford University, Stanford, CA 94305, and Division of Respiratory Diseases, Children's Hospital Boston, MA 02115, USA. rmoss@stanford.edu

Country: United States

AGENCY: United States NCRR

GRANT: RR02172

ACRONYM: RR

MEDLINETA: Hum Gene Ther

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