An IIT Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Injection of SNUG01 in Patients with Amyotrophic Lateral Sclerosis

NCTID	NCT06645197 (View at clinicaltrials.gov)
Description	This is a multicenter, open-label, single-arm investigator-initiated clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of SNUG01 in patients with Amyotrophic Lateral Sclerosis (ALS). (Show More)
Development Status	Active
Indication	Amyotrophic Lateral Sclerosis (ALS)
Disease Ontology Term	DOID:332
Compound Name	SNUG01
Sponsor	Peking University Third Hospital
Funder Type	Other
Recruitment Status	Not yet recruiting
Enrollment Count	7
Results Posted	Not Available

Target Gene/Variant	TRIM72
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Genetic delivery of therapeutic protein
Route of Administration	Intrathecal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type	none
Dose 1	3 undisclosed dose levels
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	5
Locations	China