Phase 2
N=35
Single Dose of pGM169/GL67A in CF Patients
Cystic Fibrosis
Bottom Line
View on ClinicalTrials.gov: NCT00789867 ↗Enrolled (actual)
35
Serious AEs
5.7%
Results posted
Jan 2020
Primary outcome: Primary: Body Maximum Temperature — 38.6; 38.0; 37.4 celsius — p=0.0002
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- pGM169/GL67A (Drug)
- Age
- Pediatric, Adult, Older Adult · 16+ yrs
- Sex
- All
- Sponsor
- Imperial College London
- Primary completion
- Aug 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Body Maximum Temperature |
38.6; 38.0; 37.4 | 0.0002 sig |
| PRIMARY Blood Leukocytes |
15.8; 14.1; 12.8 | 0.22 |
| PRIMARY Blood Neutrophils |
13.9; 11.3; 9.8 | 0.06 |
| PRIMARY FEV1 Relative % Drop |
24.6; 17.5; 16.8 | 0.08 |
| PRIMARY FVC Relative % Drop |
20.7; 13.7; 14.7 | 0.22 |
| PRIMARY Lung Clearance Index - LCI |
0.75; 0.32; 0.32 | 0.003 sig |
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.
Eligibility 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:
- 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
Data sourced from ClinicalTrials.gov (NCT00789867). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.