Inclusion and exclusion criteria
| Inclusion criteria| | Exclusion criteria |
|---|---|
| Confirmed diagnosis of cystic fibrosis (sweat testing or genetic analysis) | Infection with Burkholderia cepacia complex organisms, MRSA or Mycobacterium abscessus |
| Men and women aged 12 years and above | Recent acute upper respiratory tract infection; |
| FEV1 of between 50 and 90% predicted | Significant nasal pathology* |
| Clinical stability at enrolment | Chloride secretory response on nasal PD of >5 mV (nasal cohort only)*; |
| Prepared to take effective contraceptive precautions | Previous spontaneous pneumothorax without pleurodesis† |
| Able and willing to withhold rhDNase treatment for 24 h before and after the gene therapy dose. | Recurrent severe haemoptysis† |
| Current smoker | |
| Significant comorbidity (moderate or severe CF liver disease, significant renal impairment, significant coagulopathy†) | |
| Receiving second-line immunosuppressant drugs‡ | |
| Pregnant or breastfeeding. |
*Applies to nasal subgroup only.
†Applies to bronchoscopic subgroup only.
‡Examples: methotrexate, intravenous immunoglobulin.
CF, cystic fibrosis;FEV1, forced expiratory volume in 1 second; MRSA, methicillin-resistant Staphylococcus aureus; PD, potential difference; rhDNase, recombinant human deoxyribonuclease.