Cystic fibrosis MedDRA version: 9.1 Level: LLT Classification code 10011762 Term: Cystic fibrosis
Conditions
Interventions
Product Name: pGM169/GL67A
Product Code: pGM169/GL67A
Pharmaceutical Form: Inhalation vapour, solution
Sponsors
Imperial College
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Cystic fibrosis confirmed by sweat testing or genetic analysis 2. Males and females aged 16 years and above 3. Forced expiratory volume in the 1st second (FEV1) > 60% predicted values [ we consider that this population of patients will have relatively clear airways and thus the best chance of successful gene transfer] 4. Clinical stability at entry defined by: a. Not on any extra antibiotics (excluding routine, long-term treatments) for the previous 2 weeks b. No increase in symptoms such as change in sputum production/colour, increased wheeze or breathlessness over the previous 2 weeks c. No change in regular respiratory treatments over the previous 4 weeks d. If any of these apply, entry into the study can be deferred 5. Prepared to take effective contraceptive precautions for the duration of their participation in the study and for 3 months thereafter [as stated in GTAC guidelines] 6. If taking regular rhDNase (pulmozyme) is willing, and considered able by independent medical carers, to withhold treatment for 48 hours around the time of the gene therapy dose 7. Written informed consent obtained 8. Permission to inform GP of participation in study Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Infection with Burkholderia cepacia complex organisms or MRSA [for infection control reasons]) 2. Significant nasal pathology including polyps, clinically-significant rhinosinusisitis, or recurrent severe epistaxis (nose bleeds) [either dose delivery or assays may be adversely affected] 3. Acute upper respiratory tract infection within the last 2 weeks (entry can be deferred) 4. Previous spontaneous pneumothorax, unless patient has had a subsequent pleurodesis [this subgroup are at increased risk of future pneumothoraces] 5. Recurrent severe haemoptysis [this group may be at increased risk of bleeding due to bronchoscopy and biopsy] 6. Current smoker (will be assessed by measuring cotinine level prior to study entry) [we cannot determine the effect of smoking on gene transfer or assays] 7. Significant comorbidity including: a. Moderate/severe CF liver disease (varices or significant, sustained elevation of transaminases: ALT/ AST>100 IU/l) b. Significant renal impairment (serum creatinine > 150 micro mol/l) c. Significant coagulopathy 8. Receiving 2nd line immunosuppressant drugs such as methotrexate, cyclosporine, intravenous immunoglobulin preparations 9. Pregnant or breastfeeding
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the safety and gene transfer resulting from a single dose of pGM169/GL67A to the lungs and nose of patients with cystic fibrosis;Secondary Objective: ;Primary end point(s): Safety: clinical examination, oxygen saturation, spirometry, routine clinical blood samples, chest CT, gas transfer (TLCO) Expression: transgene mRNA and CFTR protein expression in airway samples; potential difference measurements in nose and bronchi | — |
Countries
United Kingdom
Outcome results
None listed