Actively Recruiting
First-in-Human Clinical Trial of STUP-001, an In Vivo Direct Cell Conversion Gene Therapy for AIS-A/B Chronic Spinal Cord Injury
Led by Yonsei University · Updated on 2025-07-16
9
Participants Needed
1
Research Sites
126 weeks
Total Duration
On this page
Sponsors
Y
Yonsei University
Lead Sponsor
S
Stand Up Therapeutics INC.
Collaborating Sponsor
AI-Summary
What this Trial Is About
This study aim to investigate the safety and exploratory efficacy of STUP-001 which is AAV-based spinal cord injury investigational product.
CONDITIONS
Official Title
First-in-Human Clinical Trial of STUP-001, an In Vivo Direct Cell Conversion Gene Therapy for AIS-A/B Chronic Spinal Cord Injury
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 19 to 60 years at the time of informed consent
- Diagnosed with traumatic spinal cord injury classified as AIS-A (complete loss of sensory and motor function) or AIS-B (partial sensory preservation with complete motor paralysis)
- Traumatic spinal cord injury diagnosed for at least 6 months at screening
- Women must provide evidence of non-fertile status by being postmenopausal, having irreversible surgical infertility (excluding tubal ligation), or meeting hormone level criteria
- Acceptable contraception methods include complete abstinence, tubal sterilization, certain hormonal contraceptives without known drug interactions, copper IUD, or partner vasectomy
- Able to understand study information and provide informed consent or have a legally authorized representative sign on their behalf
You will not qualify if you...
- Diagnosed with traumatic spinal cord injury classified as AIS-C or D at screening
- History of myocardial infarction or unstable angina within 6 months
- QTc interval ≥ 450 msec or significant ECG abnormalities
- Congestive heart failure NYHA Class II or higher
- Stroke or transient ischemic attack within 6 months
- Uncontrolled diabetes mellitus (e.g., HbA1c > 8%)
- Uncontrolled hypertension (systolic BP > 180 mmHg or diastolic BP > 100 mmHg)
- History or treatment of malignancy within 5 years
- Positive tests for HBsAg, anti-HCV Ab (unless RNA negative after treatment), or anti-HIV Ab
- Severe infections requiring continuous medication for 3 weeks or more during the trial
- Hypersensitivity to any component of the investigational drug
- History of gene or cell therapy involving AAV2
- Abnormal lab results: WBC < 2,000/mm³, platelets < 100,000/mm³, hemoglobin < 10.0 g/dL, serum creatinine > 1.5× upper limit, total bilirubin > 1.5× upper limit, AST or ALT > 3× upper limit, PT-INR/aPTT > 1.5× upper limit
- Substance abuse, alcohol dependence, or psychiatric disorders
- Unable to undergo general anesthesia
- Pregnant or breastfeeding or positive pregnancy test
- Received another investigational drug within 4 weeks prior to screening
- Any other condition deemed exclusionary by the investigator
AI-Screening
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Trial Site Locations
Total: 1 location
1
Severance Hospital
Seoul, South Korea
Actively Recruiting
Research Team
J
Junsang Yoo, Ph D.
CONTACT
C
Chunggu Kim, Ph D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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