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Clinical Trial Report

Gene Therapy Trial Report

Summary

Safety and Efficacy of AMT-130 in European Adults with Early Manifest Huntington's Disease


NCTID NCT05243017 (View at clinicaltrials.gov)
Description
Indication Huntington's Disease, Early Manifest
Compound Name AMT-130 (rAAV5-miHTT)
Sponsor UniQure Biopharma B.V.
Funder Type Industry
Status
Recruiting
Enrollment Count 14

Therapy Information


Target Gene/Variant MiHTT
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action MiRNA knockdown of mutant/aberrant gene
Route of Administration Intraparenchymal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV5
Editor Type
Dose 1 6 x 10^12 gc/subject
Dose 2 6 x 10^13 gc/subject
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2021-11-01
Completion Date 2029-10-07
Last Update 2024-10-24

Participation Criteria


Eligible Age 25 Years - 65 Years
Standard Ages Adult, Older adult
Eligible Sex ALL

Locations


No.of Trial Sites 6
Locations Poland,United Kingdom,Germany

Regulatory Information


Has US IND False
Recent Updates FDA granted RMAT designation Q2 2024

Resources/Links