Phase I/II study to evaluate the safety and effects on Progranulin levels of PR006 in patients with Fronto-Temporal Dementia with Progranulin mutations (FTD-GRN).

Study title: 
Phase I/II study to evaluate the safety and effects on Progranulin levels of PR006 in patients with Fronto-Temporal Dementia with Progranulin mutations (FTD-GRN).
Long title: 
A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on Progranulin Levels of PR006A in Patients with Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN) (PROCLAIM)
Date receipt dossier: 
23 juil 2021
EU record number: 
B/BE/21/BVW3
EudraCT number: 
2019-003159-12
Company / Sponsor: 
Prevail Therapeutics
Phase: 
I/II
Treated organism: 
Humans
Indication category: 
Genetic disorders
Disease: 
Fronto-temporal dementia with progranulin mutations
Therapeutic approach: 
Gene therapy
Genetic modification: 
The study involves a replication-incompetent adeno-associated virus, AAV9, lacking all AAV viral genes and encoding for the human Progranulin (GRN) gene
Method of transfer of nucleic acid of interest: 
Non-replicating recombinant adeno-associated virus serotype 9 (AAV9)
Administered biological material: 
Genetically modified virus
Route of administration: 
Intracisternal magna (ICM) injection
Locations in Belgium: 
UZ Leuven
Nr of subjects: 
2
Foreseen duration: 
Q4 2021 to end of treatment period Q1 2028
Type of procedure: 
Contained use and Deliberate release
Current status: 
Authorized

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