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Single Dose of pGM169/GL67A in CF Patients

The recruitment status of this study is unknown because the information has not been verified recently.
Verified August 2009 by Imperial College London.
Recruitment status was  Recruiting
Sponsor:
Collaborators:
Royal Brompton & Harefield NHS Foundation Trust
University of Oxford
University of Edinburgh
Cystic Fibrosis Trust
University of Pennsylvania
Information provided by:
Imperial College London
ClinicalTrials.gov Identifier:
NCT00789867
First received: November 11, 2008
Last updated: August 13, 2009
Last verified: August 2009
History of Changes
  Purpose

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: Endpoint Classification: Safety/Efficacy Study
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:

MedlinePlus related topics: Cystic Fibrosis
U.S. FDA Resources

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 ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 27
Study Start Date: November 2008
Estimated Study Completion Date: December 2009
Estimated 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)

  Eligibility

Ages Eligible for Study:   16 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00789867

Contacts
Contact: Jane Davies jcdavies@imperial.ac.uk
Contact: Nia Voase n.voase@imperial.ac.uk

Locations
United Kingdom
Royal Brompton Hospital Recruiting
London, United Kingdom, SW3 6NP
Contact: Jane C Davies     00 44 207 351 8398     j.c.davies@imperial.ac.uk    
Principal Investigator: Eric Alton            
Sub-Investigator: Jane Davies            
Sub-Investigator: Uta Griesenbach            
Sponsors and Collaborators
Imperial College London
Royal Brompton & Harefield NHS Foundation Trust
University of Oxford
University of Edinburgh
Cystic Fibrosis Trust
University of Pennsylvania
Investigators
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 Oxford University
Principal Investigator: Deborah Gill Oxford University
Principal Investigator: David Porteous Edinburgh University
Principal Investigator: Chris Boyd Edinburgh University
Principal Investigator: Alastair Innes Edinburgh University
  More Information

Additional Information:
UK CF Gene Therapy Consortium website  This link exits the ClinicalTrials.gov site

Publications:

Responsible Party: Gary Roper, Imperial College
ClinicalTrials.gov Identifier: NCT00789867     History of Changes
Other Study ID Numbers: cro851, Version 3 08.09.08
Study First Received: November 11, 2008
Last Updated: August 13, 2009
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Imperial College London:
Single dose
Pilot
Safety
Gene expression
 Tolerability
CFTR gene
Cystic fibrosis
Non-viral

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
 Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on June 18, 2013