Repeated application of gene therapy in patients with cystic fibrosis
- Conditions
- Cystic fibrosisMedDRA version: 20.0 Level: PT Classification code 10011763 Term: Cystic fibrosis lung System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
- Registration Number
- EUCTR2011-004761-33-GB
- Lead Sponsor
- Imperial College
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 116
1. Confirmed diagnosis of Cystic Fibrosis.
2. =12 years.
3. Mild to moderate cystic fibrosis lung disease based on forced expiratory volume in the first second (FEV1) of 50-90% predicted
4. Any CFTR mutation.
5. Clinical stability over the 4 weeks prior to the first dose.
6. Written informed consent from the patient (aged 16 years and above) or parent if a child aged 12-15 years.
7. Assent from a child aged 12-15 years.
8. Willing to adhere to contraceptive requirements.
Are the trial subjects under 18? yes
Number of subjects for this age range: 55
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 75
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Current participation in another interventional trial.
2. Infection with Burkholderia cepacia complex organisms, Mycobacterium abscessus or MRSA (unless local infection control guidelines can be adhered to).
3. Previous spontaneous pneumothorax unless pleurodesed (bronchoscopic group only).
4. Recurrent severe haemoptysis.
5. Current smoker.
6. Significant comorbidity eg severe CF liver disease or renal impairment.
7. Using second line immunosuppressants.
8. Pregnant or breast-feeding.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method