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

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

Treatments tested in this trial

  • STUP-001

Treatment groups

9 Participants
are divided into 2 treatment groups

Sponsors and collaborators

Yonsei University

Lead sponsor

Stand Up Therapeutics INC.

Collaborator