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

NCT06922890 | Recruiting Phase 1 DMC

• 2 other identifiers: STUP-001_PRTStand Up Tx1
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Spinal Cord Injury; Chronic Spinal Cord Injury

Keywords

Direct Lineage Reprogramming; Cell Fate Conversion; First-in-Human Multi-Cistronic Vector Application

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (2)

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.

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.

Drug: STUP-001

Interventions

STUP-001 DRUG

in vivo direct cell conversion gene therapy investigational product

STUP-001 (1/2X); STUP-001(1X)

Eligibility Criteria

• Age: 19 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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:
• (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:
• 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).

You may not qualify if:

• 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.
• 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

Sponsors & Collaborators

• Yonsei University: lead
• Stand Up Therapeutics INC.: collaborator

Study Sites (1)

Severance Hospital

Seoul, South Korea

RECRUITING

MeSH Terms

Conditions

Spinal Cord Injuries

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Trauma, Nervous System; Wounds and Injuries

Central Study Contacts

Junsang Yoo, Ph D.

CONTACT

82-2-512-0912; info@stutps.com

Chunggu Kim, Ph D.

CONTACT

+82-10-9784-2722; chunggoo@stutps.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor, Department of Neurosurgery, M.D., Ph.D.

Study Record Dates

• First Submitted: April 3, 2025
• First Posted: April 11, 2025
• Study Start: July 30, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2027
• Last Updated: July 16, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)