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Trial record 1 of 1 for:    NCT00789867
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Single Dose of pGM169/GL67A in CF Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00789867
Recruitment Status : Completed
First Posted : November 13, 2008
Last Update Posted : June 3, 2015
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

Brief Summary:
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 or disease Intervention/treatment Phase
Cystic Fibrosis Drug: pGM169/GL67A Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 27 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : November 2008
Actual Primary Completion Date : August 2009
Actual Study Completion Date : December 2010

Arm Intervention/treatment
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)

Primary Outcome Measures :
  1. 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 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 70 Years   (Child, Adult, Older Adult)
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00789867

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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
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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

Additional Information:
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Responsible Party: Imperial College London Identifier: NCT00789867     History of Changes
Other Study ID Numbers: cro851
Version 3 08.09.08
First Posted: November 13, 2008    Key Record Dates
Last Update Posted: June 3, 2015
Last Verified: August 2009
Keywords provided by Imperial College London:
Single dose
Gene expression
CFTR gene
Cystic fibrosis
Additional relevant MeSH terms:
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Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases