Single Dose of pGM169/GL67A in CF Patients

• Status: Completed
• Trial end date: 2010-12-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Imperial College London
• Collaborators: Cystic Fibrosis Trust; Royal Brompton & Harefield NHS Foundation Trust; University of Edinburgh; University of Oxford; University of Pennsylvania

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