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Clinical Trial Report

Gene Therapy Trial Report

Summary

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
Indication Amyotrophic Lateral Sclerosis
Compound Name AMT-162
Sponsor UniQure Biopharma B.V.
Funder Type Industry
Status
Recruiting
Enrollment Count 20

Therapy Information


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
Dose 1 Undisclosed low dose
Dose 2 Undisclosed intermediate dose
Dose 3 Undisclosed high dose
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2023-10-20
Completion Date 2031-03-30
Last Update 2024-10-15

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Eligible Sex ALL

Locations


No.of Trial Sites 10
Locations United States

Regulatory Information


Has US IND True
Recent Updates First patient dosed October 2024

Resources/Links