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Clinical Trial Report

Gene Therapy Trial Report

Summary

Phase 1/2 Clinical Trial of LY3884963 in Patients With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)


NCTID NCT04408625 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Frontotemporal Dementia
Disease Ontology Term DOID:0060672
Compound Name LY3884963
Compound Alias PR006
Compound Description AAV9-CMVe-CBA-GRN
Sponsor Prevail Therapeutics
Funder Type Industry
Recruitment Status
Recruiting
Enrollment Count 30
Results Posted Not Available

Therapy Information


Target Gene/Variant GRN
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intracisterna magna
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 2.1E13 vg
Dose 2 4.2E13 vg
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2020-05-21
Completion Date 2030-04-30
Last Update 2025-03-18

Participation Criteria


Eligible Age 30 Years - 85 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 11
Locations Belgium,United States,United Kingdom,Australia,France,Spain

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation
Recent Updates

Resources/Links