NCT06645197

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

A Multicenter, Single-Arm, Open-Label, IIT Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Injection of SNUG01 in Patients with Amyotrophic Lateral Sclerosis (ALS)

• Status: Not Yet Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 7 (estimated)
• Sponsor: Peking University Third Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

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).

Detailed description

SNUG01 is a gene therapy designed to deliver a protective protein gene to the central nervous system. Safety will be the primary focus during the initial 24 weeks post-treatment (primary study period). After the primary study period, participants will be assessed for both safety and efficacy for up to a total of 5 years following SNUG01 treatment.

Conditions

• Amyotrophic Lateral Sclerosis (ALS)

Interventions

Type	Name	Description
DRUG	SNUG01	Recombinant adeno-associated virus serotype 9 capsid containing human SG001 expression cassette

Timeline

• Start date: 2024-10-16
• Primary completion: 2025-04-15
• Completion: 2029-10-15
• First posted: 2024-10-16
• Last updated: 2024-10-16

Locations

5 sites across 1 country: China