Single Dose of pGM169/GL67A in CF Patients

This study is not yet open for participant recruitment.

Verified by Imperial College London, November 2008

Sponsors and Collaborators:	Imperial College London Royal Brompton Hospital NHS Trust University of Oxford University of Edinburgh Cystic Fibrosis Trust University of Colorado at Denver and Health Sciences Center University of Pennsylvania
Information provided by:	Imperial College London
ClinicalTrials.gov Identifier:	NCT00789867

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 I Phase II

Genetics Home Reference related topics: cystic fibrosis

MedlinePlus related topics: Cystic Fibrosis

Drug Information available for: Lipids Cystic fibrosis transmembrane conductance regulator

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
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

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
pGM169/GL67A: Experimental 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

Locations

United Kingdom
Royal Brompton Hospital
London, United Kingdom, SW3 6NP

Sponsors and Collaborators

Imperial College London

Royal Brompton Hospital NHS Trust

University of Oxford

University of Edinburgh

Cystic Fibrosis Trust

University of Colorado at Denver and Health Sciences Center

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

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

Publications:

Alton EW, Stern M, Farley R, Jaffe A, Chadwick SL, Phillips J, Davies J, Smith SN, Browning J, Davies MG, Hodson ME, Durham SR, Li D, Jeffery PK, Scallan M, Balfour R, Eastman SJ, Cheng SH, Smith AE, Meeker D, Geddes DM. Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial. Lancet. 1999 Mar 20;353(9157):947-54.

Responsible Party:	Imperial College ( Gary Roper )
Study ID Numbers:	cro851, Version 3 08.09.08
Study First Received:	November 11, 2008
Last Updated:	November 11, 2008
ClinicalTrials.gov Identifier:	NCT00789867
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

Study placed in the following topic categories:

Digestive System Diseases
Genetic Diseases, Inborn
Respiratory Tract Diseases
Cystic Fibrosis
Fibrosis

Lung Diseases
Infant, Newborn, Diseases
Pancreatic Diseases
Cystic fibrosis

Additional relevant MeSH terms:

Pathologic Processes

ClinicalTrials.gov processed this record on January 16, 2009