Spinal Cord InjuryPhase 1/2STUP-001NCT06922890
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 Phase 1/2 study testing STUP-001 in people with Spinal Cord Injury and Chronic Spinal Cord Injury, run by Yonsei University at 1 site in South Korea.
Trial summary
This study aim to investigate the safety and exploratory efficacy of STUP-001 which is AAV-based spinal cord injury investigational product. Read more about this study
- Who is it for?
- Ages 19 to 60 years
- Conditions the study is for
- Spinal Cord Injury, Chronic Spinal Cord Injury
- What is being tested?
- STUP-001
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteSouth Korea
- Study dates
- 30 Jul 2025to 31 Dec 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Male and female adults aged 19 to 60 years at the time of obtaining informed consent.
- Patients diagnosed with traumatic spinal cord injury classified as ASIA Impairment Scale (AIS) grade A or B:
- AIS-A: Complete loss of sensory and motor function.
- AIS-B: Partial preservation of sensory function with complete motor paralysis
- Patients who have been diagnosed with traumatic spinal cord injury for at least 6 months at the time of screening.
- At the time of screening, women must provide evidence of non-fertile status by meeting at least one of the following criteria:
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- Postmenopausal women: Defined as women over the age of 50 who have been amenorrheic for at least 12 consecutive months after discontinuing all exogenous hormonal treatments.
- Women with irreversible surgical infertility, including hysterectomy, bilateral oophorectomy, or bilateral salpingectomy. (Tubal ligation is not accepted.) (c) Women 50 years or younger who have been amenorrheic for at least 12 consecutive months after discontinuing all exogenous hormonal treatments and have luteinizing hormone (LH) and follicle-stimulating hormone (FSH) levels within the postmenopausal range defined by the study site.
- Acceptable methods of contraception include:
- Complete abstinence.
- Tubal sterilization.
- Hormonal contraceptives with no known drug interactions (e.g., levonorgestrel intrauterine system (IUS) (Mirena), medroxyprogesterone).
- Copper intrauterine device (IUD).
- Partner vasectomy. (Note: Periodic abstinence methods (e.g., ovulation timing, symptothermal method, or post-ovulatory methods) and withdrawal are NOT considered acceptable contraception methods.)
- Individuals who are able to understand the provided information and can voluntarily sign the written informed consent (or have a legally authorized representative sign on their behalf).
Who cannot join
- Patients diagnosed with traumatic spinal cord injury (SCI) classified as AIS-C or D at the time of screening.
- Patients with the following cardiovascular conditions at the time of screening:
- Myocardial infarction or unstable angina within the past 6 months.
- QTc interval ≥ 450 msec or clinically significant electrocardiographic (ECG) abnormalities.
- Congestive heart failure (CHF) classified as New York Heart Association (NYHA) Class II or higher.
- Stroke or transient ischemic attack (TIA) within the past 6 months.
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- Patients with uncontrolled diabetes mellitus (e.g., HbA1c > 8% at screening).
- Patients with uncontrolled hypertension (e.g., systolic BP > 180 mmHg or diastolic BP > 100 mmHg).
- Patients diagnosed with, undergoing treatment for, or with a history of malignancy within the past 5 years at the time of screening.
- Patients with positive serologic test results for HBsAg, anti-HCV Ab, or anti-HIV Ab at the time of screening. However, patients with positive anti-HCV Ab may be eligible if HCV RNA test results are negative after treatment.
- Patients with clinically significant severe infections, as determined by the investigator (e.g., infections requiring continuous concomitant medication for ≥3 weeks during the clinical trial period).
- Patients with a history of hypersensitivity to any component of the investigational drug.
- Patients with a history of gene or cell therapy involving AAV2.
- Patients with the following abnormal laboratory test results at screening:
- WBC < 2,000/mm³
- Platelet count < 100,000/mm³
- Hemoglobin < 10.0 g/dL
- Serum creatinine > 1.5×ULN
- Total bilirubin > 1.5×ULN
- AST, ALT > 3×ULN
- PT-INR/aPTT > 1.5×ULN
- Patients with substance abuse, alcohol dependence, or psychiatric disorders.
- Patients who are unable to undergo general anesthesia.
- Pregnant or breastfeeding women, or those with a positive pregnancy test at screening.
- Patients who have received another investigational drug within 4 weeks prior to screening. However, patients who have not received an investigational drug or participated in non-interventional observational studies may be eligible.
- Any other individuals deemed ineligible for the clinical trial at the investigator's discretion.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
South Korea1 site, 1 recruiting
- Recruiting
Severance Hospital
Seoul
Yoon Ha, MD. Ph. D., +82-2-2228-5559, info@stutps.com
chunggoo Kim, Ph. D., +82-2-512-0912, chunggoo@stutps.com
About this study
Official title: 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
This study aim to investigate the safety and exploratory efficacy of STUP-001 which is AAV-based spinal cord injury investigational product.
Read the 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
- Being tested
- STUP-001
- Keywords
- Direct Lineage Reprogramming, Cell Fate Conversion, First-in-Human Multi-Cistronic Vector Application
- Medical subject headings
- Spinal Cord Injuries
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
STUP-001Drug
in vivo direct cell conversion gene therapy investigational product
| Study group | Role | What they receive |
|---|---|---|
| STUP-001 (1/2X) | Experimental | The participant with chronic spinal cord injury was administered a total of 25 μL via the intraparenchymal route into the spinal cord. Receives: Drug: STUP-001 |
| STUP-001(1X) | Experimental | The participant with chronic spinal cord injury was administered a total of 50 μL via the intraparenchymal route into the spinal cord. Receives: Drug: STUP-001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dos-limit toxicity evaluation post STUP-001 administration
National Cancer Institute-Comone Terminology Criteria for Adverse Events V5.0 (NCI-CTCAE V5.0)
Measured: 4weeks post STUP-001 administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Apr 2025
- Study start30 Jul 2025 (estimated)
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Cord InjuryStudies of the same condition, open and under way.
- More trials run by Yonsei UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Yonsei University
- Sponsor type
- University, hospital or other
- Collaborators
- Stand Up Therapeutics INC.
- Responsible party
- Principal investigator, Yoon Ha, Yonsei University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06922890
- Sponsor's protocol ID
- STUP-001_PRT
- Other IDs
- Stand Up Tx1
- Acronym
- STUP-001
- Status last verified
- Jul 2025
- First submitted
- 3 Apr 2025
- Last updated
- 16 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06922890
- Acronym / sponsor ID
- STUP-001 / STUP-001_PRT
- Conditions
- Spinal Cord Injury, Chronic Spinal Cord Injury
- Keywords
- Direct Lineage Reprogramming, Cell Fate Conversion, First-in-Human Multi-Cistronic Vector Application
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Yonsei UniversityUniversity, hospital or other
- Collaborators
- Stand Up Therapeutics INC.
- Study start
- 30 Jul 2025 (estimated)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 11 Apr 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| STUP-001 (1/2X) | Experimental | Drug: STUP-001The participant with chronic spinal cord injury was administered a total of 25 μL via the intraparenchymal route into the spinal cord. |
| STUP-001(1X) | Experimental | Drug: STUP-001The participant with chronic spinal cord injury was administered a total of 50 μL via the intraparenchymal route into the spinal cord. |
Who may qualify
- Ages
- 19 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (15 listed)
- Male and female adults aged 19 to 60 years at the time of obtaining informed consent.
- Patients diagnosed with traumatic spinal cord injury classified as ASIA Impairment Scale (AIS) grade A or B:
- Patients who have been diagnosed with traumatic spinal cord injury for at least 6 months at the time of screening.
- At the time of screening, women must provide evidence of non-fertile status by meeting at least one of the following criteria:
- Postmenopausal women: Defined as women over the age of 50 who have been amenorrheic for at least 12 consecutive months after discontinuing all exogenous hormonal treatments.
Key exclusion criteria (26 listed)
- Patients diagnosed with traumatic spinal cord injury (SCI) classified as AIS-C or D at the time of screening.
- Patients with the following cardiovascular conditions at the time of screening:
- Patients with uncontrolled diabetes mellitus (e.g., HbA1c > 8% at screening).
- Patients with uncontrolled hypertension (e.g., systolic BP > 180 mmHg or diastolic BP > 100 mmHg).
- Patients diagnosed with, undergoing treatment for, or with a history of malignancy within the past 5 years at the time of screening.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| STUP-001in vivo direct cell conversion gene therapy investigational product | Drug | STUP-001 (1/2X), STUP-001(1X) |
Primary outcome
Dos-limit toxicity evaluation post STUP-001 administration
Time frame: 4weeks post STUP-001 administration
National Cancer Institute-Comone Terminology Criteria for Adverse Events V5.0 (NCI-CTCAE V5.0)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing STUP-001 in people with Spinal Cord Injury and Chronic Spinal Cord Injury, run by Yonsei University at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Junsang Yoo, Ph D. (82-2-512-0912, info@stutps.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dos-limit toxicity evaluation post STUP-001 administration (time frame: 4weeks post STUP-001 administration). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2025.