Status:
COMPLETED
Single Dose of pGM169/GL67A in CF Patients
Lead Sponsor:
Imperial College London
Collaborating Sponsors:
Royal Brompton & Harefield NHS Foundation Trust
University of Oxford
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
16-70 years
Phase:
PHASE1
PHASE2
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...
Detailed Description
The trial is designed as single administration to nose and lung. Initially, in Part A, 3 patients will be dosed individually one week apart with 10 ml (26.5mg pDNA) via nebuliser and a nasal dose equi...
Eligibility Criteria
Inclusion
- 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
- 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:
- Moderate/severe CF liver disease
- Significant renal impairment
- Significant coagulopathy
- Receiving 2nd line immunosuppressant drugs such as methotrexate, cyclosporine, intravenous immunoglobulin preparations
- Pregnant or breastfeeding
Key Trial Info
Start Date :
November 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2010
Estimated Enrollment :
35 Patients enrolled
Trial Details
Trial ID
NCT00789867
Start Date
November 1 2008
End Date
December 1 2010
Last Update
January 18 2020
Active Locations (1)
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1
Royal Brompton Hospital
London, United Kingdom, SW3 6NP