B/BE/21/BVW3 - Summary Notification Information Format (SNIF)Public information presented according to the provisions of Directive 2001/18/EC
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
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