Repeated Application of Gene Therapy in CF Patients

Summary

Cystic fibrosis is a genetic condition where epithelial cells, including from the respiratory tract, have an abnormal function of a surface protein, the cystic fibrosis transmembrane conductance regulator (CFTR) protein resulting from abnormal gene expression. The trial will assess the clinical efficacy, safety \& tolerability and gene expression following repeated nebulised doses of a gene product coding for a normal CFTR protein, with the primary outcome of the trial assessing lung function.

Conditions

• Cystic Fibrosis

Interventions

DRUG

pGM169/GL67A

5ml Gene Product/Lipid Vector pGM169/GL67A nebulised; 2ml nasal application of pGM169/GL67A in addition to 5ml nebulised gene product (Nasal Subgroup)

DRUG

Placebo

5ml Nebulised non-active placebo; 2ml nasal administration of non-active placebo (nasal subgroup)

Sponsors & Collaborators

• University of Edinburgh

  collaborator OTHER

• University of Oxford

  collaborator OTHER

• Royal Brompton & Harefield NHS Foundation Trust

  collaborator OTHER

• Imperial College London

  lead OTHER

Principal Investigators

• Eric Alton, MD, FMedSci · Imperial College London

• Jane Davies, MD · Imperial College London

• Uta Griesenbach, PhD · Imperial College London

• Steve Hyde, MA, DPhil · University of Oxford

• Deborah Gill, PhD · University of Oxford

• Chris Boyd, PhD · Edinburgh University

• David Porteous, FMedSci · Edinburgh University

• Alastair Innes, PhD · Edinburgh University

• Steve Cunningham, PhD · Edinburgh University

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Model

SINGLE_GROUP

Eligibility

Min Age

12 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2012-05-31

Primary Completion

2014-05-31

Completion

2014-05-31

Countries

• United Kingdom

Study Locations