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Clinical Trial Report
Gene Therapy Trial Report
Summary
Repeated Application of Gene Therapy in CF Patients
NCTID
NCT01621867
(View at clinicaltrials.gov)
Description
Cystic fibrosis is a genetic condition where epithelial cells, including from the respiratory tract, have an abnormal function of a surface protein, the cystic fibrosis transmembrane conductance regulator (CFTR) protein resulting from abnormal gene expression. The trial will assess the clinical efficacy, safety \& tolerability and gene expression following repeated nebulised doses of a gene product coding for a normal CFTR protein, with the primary outcome of the trial assessing lung function.
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Development Status
Inactive
Indication
Cystic Fibrosis
Disease Ontology Term
DOID:1485
Compound Name
PGM169/GL67A
Sponsor
Imperial College London
Funder Type
Other
Recruitment Status
Completed
Enrollment Count
130
Results Posted
Not Available
Therapy Information
Target Gene/Variant
CFTR
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Inhalational
Drug Product Type
Encapsulated plasmid
Target Tissue/Cell
Delivery System
Liposome
Vector Type
GL67A
Editor Type
Dose 1
Concentration: 26.5mg pDNA/10ml
Dose 2
5ml (n=8) selected as optimal dose, n=78 for phase 2b
Dose 3
10ml (n=10)
Dose 4
20ml (n=17) was discontinued due to adverse effects
Dose 5
Study Record Dates
Current Stage
Phase2
Submit Date
2012-06-14
Completion Date
2014-05
Last Update
2015-10-22
Participation Criteria
Eligible Age
>=12 Years
Standard Ages
Child, Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
3
Locations
United Kingdom
Regulatory Information
Has US IND
FDA Designations
Recent Updates
Benefits were too small to enable Phase 3 development, partnered with Boehringer Ingelheim to develop a new strategy using viral delivery. Clinical trial (NCT06515002) is ongoing
Resources/Links
Clinical Publications
Safety of a single aerosol administration of escalating doses of the cationic lipid GL-67/DOPE/DMPE-PEG5000 formulation to the lungs of normal volunteers
A Phase I/IIa Safety and Efficacy Study of Nebulized Liposome-mediated Gene Therapy for Cystic Fibrosis Supports a Multidose Trial
Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial
(Abstract P204) Immune Responses To Single And Repeated Administration Of Pgm169/gl67a: The Uk Cf Gene Therapy Consortium Clinical Trials
A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis
News and Press Releases
UK Respiratory Gene Therapy Consortium: Our First Clinical Trial Product
The UK Respiratory Gene Therapy Consortium are delighted to announce the start of recruitment for a second major gene therapy trial for people with cystic fibrosis.
Preclinical Publications
Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung
Limitations of the murine nose in the development of nonviral airway gene transfer
The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways
Pre-clinical evaluation of three non-viral gene transfer agents for cystic fibrosis after aerosol delivery to the ovine lung
Assessment of the nuclear pore dilating agent trans-cyclohexane-1,2-diol in differentiated airway epithelium
The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep
(Abstract 915) Towards Gene Therapy for Cystic Fibrosis: Bio-Distribution of GL67A/pGM169 DNA and mRNA Following Aerosol Delivery to the Mouse Lung - ASGCT 2008
CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression
Related NCTID
Phase 1/2: NCT00789867