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Single Dose of pGM169/GL67A in CF Patients

This study has been completed.
Royal Brompton & Harefield NHS Foundation Trust
University of Oxford
University of Edinburgh
Cystic Fibrosis Trust
University of Pennsylvania
Information provided by (Responsible Party):
Imperial College London Identifier:
First received: November 11, 2008
Last updated: June 2, 2015
Last verified: August 2009
The study objectives are to assess safety, tolerability and gene expression after a single dose of non-viral CFTR gene therapy (pGM169/GL67A) administered to the nose and lungs of patients with cystic fibrosis.

Condition Intervention Phase
Cystic Fibrosis
Drug: pGM169/GL67A
Phase 1
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Evaluation of Safety and Gene Expression With a Single Dose of pGM169/GL67A Administered to the Nose and Lung of Individuals With Cystic Fibrosis

Resource links provided by NLM:

Further study details as provided by Imperial College London:

Primary Outcome Measures:
  • Safety: examination, oximetry, spirometry, clinical blood samples, chest CT, gas transfer Expression: transgene mRNA and CFTR protein expression; potential difference measurements in nose and bronchi, sputum inflammatory markers [ Time Frame: Days 2,6,15,28 post-treatment ]

Enrollment: 27
Study Start Date: November 2008
Study Completion Date: December 2010
Primary Completion Date: August 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: pGM169/GL67A
Single nebulised and nasal dose
Drug: pGM169/GL67A
Plasmid expressing the human CFTR gene complexed with cationic lipid 67 and helper lipids: 10 ml nebulised and 1 ml nasal administration (n=3) and 20 ml nebulised and 2 ml nasal (n=24)


Ages Eligible for Study:   16 Years to 70 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Cystic fibrosis confirmed by sweat testing or genetic analysis
  • Males and females aged 16 years and above
  • Forced expiratory volume in the 1st second (FEV1) > 60% predicted values
  • Clinical stability at entry
  • Prepared to take effective contraceptive precautions for the duration of their participation in the study and for 3 months thereafter
  • If taking regular rhDNase (pulmozyme) is willing, and considered able by independent medical carers, to withhold treatment for 24 hours before and 24 hours after the gene therapy dose
  • Written informed consent obtained
  • Permission to inform GP of participation in study

Exclusion Criteria:

  • Infection with Burkholderia cepacia complex organisms or MRSA
  • Significant nasal pathology including polyps, clinically-significant rhinosinusitis, or recurrent severe epistaxis (nose bleeds)
  • Acute upper respiratory tract infection within the last 2 weeks
  • Previous spontaneous pneumothorax without pleurodesis
  • Recurrent severe haemoptysis
  • Current smoker
  • Significant comorbidity including:

    1. Moderate/severe CF liver disease
    2. Significant renal impairment
    3. Significant coagulopathy
  • Receiving 2nd line immunosuppressant drugs such as methotrexate, cyclosporine, intravenous immunoglobulin preparations
  • Pregnant or breastfeeding
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Please refer to this study by its identifier: NCT00789867

United Kingdom
Royal Brompton Hospital
London, United Kingdom, SW3 6NP
Sponsors and Collaborators
Imperial College London
Royal Brompton & Harefield NHS Foundation Trust
University of Oxford
University of Edinburgh
Cystic Fibrosis Trust
University of Pennsylvania
Study Director: Eric Alton Imperial College London
Principal Investigator: Jane C Davies Imperial College London
Principal Investigator: Uta Griesenbach Imperial College London
Principal Investigator: Steve Hyde University of Oxford
Principal Investigator: Deborah Gill University of Oxford
Principal Investigator: David Porteous Edinburgh University
Principal Investigator: Chris Boyd Edinburgh University
Principal Investigator: Alastair Innes Edinburgh University
  More Information

Additional Information:
Responsible Party: Imperial College London Identifier: NCT00789867     History of Changes
Other Study ID Numbers: cro851
Version 3 08.09.08
Study First Received: November 11, 2008
Last Updated: June 2, 2015

Keywords provided by Imperial College London:
Single dose
Gene expression
CFTR gene
Cystic fibrosis

Additional relevant MeSH terms:
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases processed this record on May 25, 2017