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

NCTID	NCT05243017 (View at clinicaltrials.gov)
Description	This is the second study of AMT-130 in patients with early manifest HD and is designed as part of an integrated two-study phase I/II program under a single data safety monitoring board (DSMB) with staggered enrollment based upon continued demonstration of safety of AMT-130 administration. Cohort 3 participants will receive either high or low dose (1:1 randomization). Participants enrolled in Cohort 3 will also receive an immunosuppression regimen consisting of dexamethasone, sirolimus, and rituximab. (Show More)
Development Status	Active
Indication	Huntington's Disease
Disease Ontology Term	DOID:12858
Compound Name	AMT-130
Compound Description	rAAV5-miHTT
Sponsor	UniQure Biopharma B.V.
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	14
Results Posted	Not Available

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	none
Dose 1	6E12 gc/subject
Dose 2	6E13 gc/subject
Dose 3
Dose 4
Dose 5

No.of Trial Sites	4
Locations	Poland,United Kingdom

Has US IND	False
FDA Designations	Regenerative Medicine Advanced Therapy
Recent Updates	FDA granted RMAT designation Q2 2024, FDA allowing Sponsor to seek Accelerated Approval