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

Drug
General health
Online since 4 September 2025, updated 62 days ago

About this trial

This trial is enrolling 9 participants with chronic spinal cord injury to test the safety of STUP-001, a gene therapy that converts support cells in the spinal cord into nerve cells. If included, participants will be randomly assigned to receive a single administration of either 25 μL or 50 μL of STUP-001. Final testing of safety will be performed after 7 months.

Included participants

Gender
All
Age
19 - 60 years
Injury level
C1 - S5
  • Severity (AIS)?
  • AIS-A
    AIS-B
    Time since injury
    ≥ 6 months
  • Injury type
  • Traumatic

    Healthy volunteers
    No
    C1-S5
    Additionally, participants must
    Be able to undergo general anesthesia
    Be able to provide evidence of non-fertile status, if female
    NOT have significant contraindications for elective surgery

    What’s involved

    Type

    Drug

    Details

    - Participants will be randomly assigned to receive a single intraoperative injection into the spinal cord of either 25 μL or 50 μL of STUP-001. - STUP-001 is a gene therapy that works to convert support cells in the spinal cord into nerve cells. - Final testing of safety will be performed after 7 months.

    Potential benefits

    Main benefits

    General health

    Additional benefits

    Good to know: Potential benefits are defined as outcomes that are being measured during and/or after the trial.

    Wings for Life supports SCITrialsFinder

    Wings for Life has proudly initiated, led and funded the new version of the SCI Trials Finder website. Wings for Life aims to find a cure for spinal cord injuries. The not-for-profit foundation funds world-class scientific research and clinical trials around the globe.

    Learn more


    • Trial recruitment status
    • Recruiting
    • Trial start date
    • 30 Jul 2025
    • Organisation
    • Yonsei University
    • Trial recruitment status
    • Recruiting
    • Trial start date
    • 30 Jul 2025
    • Organisation
    • Yonsei University

    Wings for Life supports SCITrialsFinder

    Wings for Life has proudly initiated, led and funded the new version of the SCI Trials Finder website. Wings for Life aims to find a cure for spinal cord injuries. The not-for-profit foundation funds world-class scientific research and clinical trials around the globe.

    Learn more