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

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Chronic Spinal Cord Injury; Spinal Cord Injury
• Intervention / Treatment: Drug: STUP-001

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 Type: Interventional
• Enrollment (Estimated): 9
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Junsang Yoo, Ph D.; Phone Number: 82-2-512-0912; Email: info@stutps.com
• Study Contact Backup: Name: Chunggu Kim, Ph D.; Phone Number: +82-10-9784-2722; Email: chunggoo@stutps.com

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of
    • Severance Hospital
    • Contact: Yoon Ha, MD. Ph D.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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:

  1. AIS-A: Complete loss of sensory and motor function.
  2. 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:

(a) 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.

(b) 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:

  1. Complete abstinence.
  2. Tubal sterilization.
  3. Hormonal contraceptives with no known drug interactions (e.g., levonorgestrel intrauterine system (IUS) (Mirena), medroxyprogesterone).
  4. Copper intrauterine device (IUD).
  5. 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).

Exclusion Criteria:

• 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:

  1. Myocardial infarction or unstable angina within the past 6 months.
  2. QTc interval ≥ 450 msec or clinically significant electrocardiographic (ECG) abnormalities.
  3. Congestive heart failure (CHF) classified as New York Heart Association (NYHA) Class II or higher.
  4. Stroke or transient ischemic attack (TIA) within the past 6 months.
• 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:

  1. WBC < 2,000/mm³
  2. Platelet count < 100,000/mm³
  3. Hemoglobin < 10.0 g/dL
  4. Serum creatinine > 1.5×ULN
  5. Total bilirubin > 1.5×ULN
  6. AST, ALT > 3×ULN
  7. 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: STUP-001 (1/2X); The participant with chronic spinal cord injury was administered a total of 25 μL via the intraparenchymal route into the spinal cord.	Drug: STUP-001; in vivo direct cell conversion gene therapy investigational product
Experimental: STUP-001(1X); The participant with chronic spinal cord injury was administered a total of 50 μL via the intraparenchymal route into the spinal cord.	Drug: STUP-001; in vivo direct cell conversion gene therapy investigational product

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Dos-limit toxicity evaluation post STUP-001 administration	National Cancer Institute-Comone Terminology Criteria for Adverse Events V5.0 (NCI-CTCAE V5.0)	4weeks post STUP-001 administration

Collaborators and Investigators

• Sponsor: Yonsei University
• Collaborators: Stand Up Therapeutics INC.

Study record dates

Study Major Dates

• Study Start (Estimated): July 30, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2027

Study Registration Dates

• First Submitted: April 3, 2025
• First Submitted That Met QC Criteria: April 3, 2025
• First Posted (Actual): April 11, 2025

Study Record Updates

• Last Update Posted (Actual): April 13, 2025
• Last Update Submitted That Met QC Criteria: April 9, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Keywords: Direct Lineage Reprogramming; Cell Fate Conversion; First-in-Human Multi-Cistronic Vector Application
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Trauma, Nervous System; Spinal Cord Diseases; Wounds and Injuries; Spinal Cord Injuries
• Other Study ID Numbers: STUP-001_PRT; Stand Up Tx1 (Other Identifier: Stand Up Therapeutics, Inc.)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No