NCT07169175

A Phase Ⅰ/Ⅱa Study of SNUG01 in Adult Subjects With ALS

A Multicenter, Open-label, Single-arm, Dose Escalation and Expansion, Phase I/IIa Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of SNUG01 in Adult Subjects With Amyotrophic Lateral Sclerosis (ALS)

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 21 (estimated)
• Sponsor: SineuGene Therapeutics Co., Ltd. · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy of SNUG01 in in adult subjects with Amyotrophic Lateral Sclerosis (ALS).

Detailed description

This is a 52-week, multicenter, open-label, single-arm, dose escalation and expansion Phase Ⅰ/Ⅱa study of SNUG01 in adults with ALS. Safety will be the primary focus, with secondary emphasis on immunogenicity, PK and preliminary clinical efficacy of SNUG01.

Conditions

• Amyotrophic Lateral Sclerosis (ALS)

Interventions

Type	Name	Description
DRUG	SNUG01	AAV (adeno-associated virus) Gene therapy

Timeline

• Start date: 2025-12-01
• Primary completion: 2028-03-30
• Completion: 2028-09-30
• First posted: 2025-09-11
• Last updated: 2025-09-11

Locations

4 sites across 2 countries: United States, China

Regulatory

• FDA-regulated drug study