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

ConditionsSpinal Cord Injury Chronic Spinal Cord Injury

Trial statusRecruiting

Trial phasePhase 1, Phase 2

Trial typeInterventional

Biological sexAll

Age19-60

SponsorYonsei University

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Male and female adults aged 19 to 60 years at the time of obtaining informed consent.

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.

Disqualifiers

Patients diagnosed with traumatic spinal cord injury (SCI) classified as AIS-C or D 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.

Trial design

Design model

Sequential

Treatments tested in this trial

STUP-001
Drug
in vivo direct cell conversion gene therapy investigational product

Treatment groups

9 Participants

are divided into 2 treatment groups

Group A: STUP-001 (1/2X)Experimental treatment 1 intervention

Group B: STUP-001(1X)Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

Dos-limit toxicity evaluation post STUP-001 administration

National Cancer Institute-Comone Terminology Criteria for Adverse Events V5.0 (NCI-CTCAE V5.0)

Time frame

4weeks post STUP-001 administration

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Yonsei University

Lead sponsor

Stand Up Therapeutics INC.

Collaborator

Source ClinicalTrials.gov

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