Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

NCTID	NCT06100276 (View at clinicaltrials.gov)
Description	This is the study of AMT-162 in Participants with SOD1-ALS and is designed to evaluate the safety, tolerability, and exploratory efficacy of intrathecally administered gene therapy AMT-162. AMT-162-001 is a Phase 1/2, multi-center, single ascending dose study. (Show More)
Indication	Amyotrophic Lateral Sclerosis
Compound Name	AMT-162
Sponsor	UniQure Biopharma B.V.
Funder Type	Industry
Status	Recruiting
Enrollment Count	20

Target Gene/Variant	MiSOD1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	MiRNA knockdown of mutant/aberrant gene
Route of Administration	Intrathecal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVrh10
Editor Type	none
Dose 1	Undisclosed low dose
Dose 2	Undisclosed intermediate dose
Dose 3	Undisclosed high dose
Dose 4
Dose 5

No.of Trial Sites	10
Locations	United States

Has US IND	True
Recent Updates	First patient dosed October 2024; IDMC recommended proceeding with 2nd cohort