NCT06922890

First-in-Human Clinical Trial of STUP-001, an In Vivo Direct Cell Conversion Gene Therapy for AIS-A/B Chronic Spinal Cord Injury

A Single-center, Prospective Investigator-initiated Trial to Evaluate the Safety and Exploratory Efficacy of STUP-001 in Patients With Chronic Spinal Cord Injury of AIS-A or B Level

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: Yonsei University · Academic / Other
• Sex: All
• Age: 19 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

This study aim to investigate the safety and exploratory efficacy of STUP-001 which is AAV-based spinal cord injury investigational product.

Detailed description

Traumatic spinal cord injury (tSCI) often leads to irreversible paralysis and severely diminishes quality of life. Despite its profound impact, no fundamental treatment is currently available. STUP-001, developed by Stand Up Therapeutics, Inc. under the leadership of Dr. Junsang Yoo, is an investigational in vivo gene therapy designed to directly convert astrocytes into functional neurons within the spinal cord. In preclinical studies, STUP-001 demonstrated both successful astrocyte-to-neuron conversion and meaningful improvements in motor function. Building on these findings, this single-center, prospective, investigator-initiated clinical trial - "A Single-center, Prospective, Investigator-Initiated Trial to Evaluate the Safety and Exploratory Efficacy of STUP-001 in Patients with Chronic Spinal Cord Injury of AIS-A or B Level" - aims to assess the safety and exploratory efficacy of STUP-001 in patients aged 19 to 60 years with chronic SCI classified as AIS-A or B. Including the screening phase, each participant will undergo a 7-month evaluation period, during which safety, tolerability, and preliminary efficacy endpoints will be closely monitored.

Conditions

• Spinal Cord Injury
• Chronic Spinal Cord Injury

Interventions

Type	Name	Description
DRUG	STUP-001	in vivo direct cell conversion gene therapy investigational product

Timeline

• Start date: 2025-07-30
• Primary completion: 2026-12-31
• Completion: 2027-12-31
• First posted: 2025-04-11
• Last updated: 2025-07-16

Locations

1 site across 1 country: South Korea