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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
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