Safety and Exploratory Efficacy of Transplantation Therapy Using PSA-NCAM(+) NPC in AIS-A Level of Sub-acute SCI

NCT04812431 | Recruiting Phase 1

• 1 other identifier: HI20C0168000020
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 2

Brief Summary

This study intends to evaluate the safety and exploratory efficacy of transplantation therapy using neural precursor cells (PSA-NCAM(+) NPC) derived from the human embryonic stem cell line for the treatment of paralysis and other related symptoms from sub-acute spinal cord injury.

Conditions

Spinal Cord Injury, Acute; Spinal Cord Injury at C4 Level With Complete Lesion; Spinal Cord Injury at C5-C7 Level With Complete Lesion

Keywords

Spinal cord injury; AIS-A

Outcome Measures

Primary Outcomes (10)

• Changes of occurred adverse events during the clinical study

  Assessment of the presentation rate of adverse events occurred from IP administration until the end of the clinical study (Visit 11).

  Visit 3(Day 0), Visit 4(1 week), Visit 5(2 weeks), Visit 6(4 weeks), Visit 7(8 weeks), Visit 8(12 weeks), Visit 9(24 weeks), Visit 10(48 weeks), Visit 11(72 weeks), Telephone visit 1(13 weeks), Telephone visit 2(14 weeks), Telephone visit 3(16 weeks)

• Rate of abnormal signs as assessed through physical examination

  Assessment of the normal/abnormal changes at the baseline (Visit 1) and changes at the end of the clinical study (Visit 11) as assessed through physical examination (appearance, skin, head/neck, chest/lung, heart, abdomen, genitourinary system, extremities, musculoskeletal system, nervous system, and lymph nodes).

  Visit 1(Screening), Visit 11(72 weeks)

• Measured changes of Systolic blood pressure (mm Hg)

  Comparative assessment of measured value using systolic blood pressure (mm Hg) from the baseline (Visit 1) to the end of the clinical study (Visit 11).

  Visit 1(Screening), Visit 2(-2 Day), Visit 3(Day 0), Visit 4(1 week), Visit 5(2 weeks), Visit 6(4 weeks), Visit 7(8 weeks), Visit 8(12 weeks), Visit 9(24 weeks), Visit 10(48 weeks), Visit 11(72 weeks)

• Measured changes of Body temperature (degrees Celsius)

  Comparative assessment of measured value using body temperature (degrees Celsius) from the baseline (Visit 1) to the end of the clinical study (Visit 11).

  Visit 1(Screening), Visit 2(-2 Day), Visit 3(Day 0), Visit 4(1 week), Visit 5(2 weeks), Visit 6(4 weeks), Visit 7(8 weeks), Visit 8(12 weeks), Visit 9(24 weeks), Visit 10(48 weeks), Visit 11(72 weeks)

• Measured changes of Heart rate (beats per minute)

  Comparative assessment of measured value using heart rate (beats per minute) from the baseline (Visit 1) to the end of the clinical study (Visit 11).

  Visit 1(Screening), Visit 2(-2 Day), Visit 3(Day 0), Visit 4(1 week), Visit 5(2 weeks), Visit 6(4 weeks), Visit 7(8 weeks), Visit 8(12 weeks), Visit 9(24 weeks), Visit 10(48 weeks), Visit 11(72 weeks)

• Measured changes of Respiratory rate (breaths per minute)

  Comparative assessment of measured value using respiratory rate (breaths per minute) from the baseline (Visit 1) to the end of the clinical study (Visit 11).

  Visit 1(Screening), Visit 2(-2 Day), Visit 3(Day 0), Visit 4(1 week), Visit 5(2 weeks), Visit 6(4 weeks), Visit 7(8 weeks), Visit 8(12 weeks), Visit 9(24 weeks), Visit 10(48 weeks), Visit 11(72 weeks)

• Rate of abnormal hematology values

  Assessment of the normal/abnormal changes at the baseline (Visit 1) and changes at the end of the clinical study (Visit 11) as measured value using hematology values(WBC, WBC differential, RBC, Hemoglobin, Hematocrit, and Platelet).

  Visit 1(Screening), Visit 2(-2 Day), Visit 3(Day 0), Visit 4(1 week), Visit 5(2 weeks), Visit 6(4 weeks), Visit 7(8 weeks), Visit 8(12 weeks), Visit 9(24 weeks), Visit 10(48 weeks), Visit 11(72 weeks)

• Rate of abnormal chemistry values

  Assessment of the normal/abnormal changes at the baseline (Visit 1) and changes at the end of the clinical study (Visit 11) as measured value using chemistry values(Sodium, Potassium, Calcium, Chloride, Inorganic Phosphate, SGOT(AST), SGPT(ALT), Alkaline Phosphatase, GGT, Creatinine, BUN, Total Bilirubin, Total Protein, Albumin, and Glucose).

  Visit 1(Screening), Visit 2(-2 Day), Visit 3(Day 0), Visit 4(1 week), Visit 5(2 weeks), Visit 6(4 weeks), Visit 7(8 weeks), Visit 8(12 weeks), Visit 9(24 weeks), Visit 10(48 weeks), Visit 11(72 weeks)

• Rate of abnormal urinalysis values

  Assessment of the normal/abnormal changes at the baseline (Visit 1) and changes at the end of the clinical study (Visit 11) as measured value using urinalysis values(Protein, Glucose, Ketones, and Occult Blood).

  Visit 1(Screening), Visit 2(-2 Day), Visit 3(Day 0), Visit 4(1 week), Visit 5(2 weeks), Visit 6(4 weeks), Visit 7(8 weeks), Visit 8(12 weeks), Visit 9(24 weeks), Visit 10(48 weeks), Visit 11(72 weeks)

• Measured changes of diastolic blood pressure (mm Hg)

  Comparative assessment of measured value using diastolic blood pressure (mm Hg) from the baseline (Visit 1) to the end of the clinical study (Visit 11).

  Visit 1(Screening), Visit 2(-2 Day), Visit 3(Day 0), Visit 4(1 week), Visit 5(2 weeks), Visit 6(4 weeks), Visit 7(8 weeks), Visit 8(12 weeks), Visit 9(24 weeks), Visit 10(48 weeks), Visit 11(72 weeks)

Secondary Outcomes (8)

• Assessment of Graft survival at the transplant site as observed in MRI examination

  Visit 1(Screening), Visit 11(72 weeks)

• Number of participants with Transplant rejection against transplanted cells

  Visit 1(Screening), Visit 6(4 weeks)

• Changes in the ASIA Damage Scale

  Visit 1(Screening), Visit 8(12 weeks), Visit 11(72 weeks)

• International standards for neurological classification of spinal cord injury (ISNCSCI) motor index score

  Visit 1(Screening), Visit 6(4 weeks), Visit 8(12 weeks), Visit 9(24 weeks), Visit 10(48 weeks), Visit 11(72 weeks)

• Measured changes of International standards for neurological classification of spinal cord injury (ISNCSCI) sensory index score

  Visit 1(Screening), Visit 8(12 weeks), Visit 11(72 weeks)

Study Arms (1)

PSA-NCAM(+) NPC

EXPERIMENTAL

Cells are administered through intrathecal injection. Injection is administered to a total of five areas.

Biological: Neural precursor cells derived from human embryonic stem cell line

Interventions

Neural precursor cells derived from human embryonic stem cell line BIOLOGICAL

When the Dose Limiting Toxicity (DLT) is not presented in the first three subjects administered with PSA-NCAM(+) NPC, two additional patients are added to the clinical study.

PSA-NCAM(+) NPC

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Upon written consent of the patient or the legally acceptable representative of the patient
• Male and female patients 18 to 65 years of age
• Female patients who showed negative results on the pregnancy test, use an acceptable contraceptive or have no possibility of pregnancy (at least 2 years have elapsed after menopause or have undergone hysterectomy, ovariectomy, or sterilization operation), or male patients who have received vasectomy or are willing to use contraceptives for up to 90 days after administration of the investigational product using a dual contraceptive method\*
• \*Dual contraceptive method: Method of using multiple contraceptives, such as using a cervical cap or contraceptive diaphragm in addition to a condom for males
• Patients who are capable of being administered with the investigational product on Day 7 to 60 after spinal cord injury
• Patients who have been confirmed to have spinal cord injury classified as AIS-A on ISNCSCI, SCIM and/or MRI by the investigator, physiatrist, or other spinal cord injury specialist
• Patients whose ASIA Impairment Scale (AIS) satisfies AIS-A criteria (spinal segment within C4-C7, complete injury)

You may not qualify if:

• Patients with spinal cord injury caused by penetrating trauma such as gunshot or stab wounds
• Patients with complete transection on the spinal cord
• Patients with spinal cord injury that require more than the mono-segment treatment
• Patients with other complications, including neurological defects related to peripheral nerve injury (neuromuscular injury or central spinal cord syndrome), or etiological causes of paraplegia or sensorimotor defects related to an additional underlying condition
• Patients with multiple lesions or lesions longer than 2 cm in length found on MRI examination
• Patients administered with cells excluding blood transfusion before participating in the clinical study
• Patients with the following intercurrent diseases or conditions:
• Coagulopathy with INR\> 1.4 at the time of administration of the investigational product (Day 0)
• Active infection
• Active hypotension requiring vasoconstrictor treatment (less than 60 mmHg of diastolic blood pressure)
• Rupture of the skin on the area of surgery
• Medical history of malignant tumor
• Primary or secondary immunodeficiency
• Clinically significant abnormal values discovered as a result of laboratory tests
• Creatinine \> 1.5 mg/dL

Sponsors & Collaborators

• S.Biomedics Co., Ltd.: lead
• Linical Co., Ltd.: collaborator
• Yonsei University: collaborator

Study Sites (2)

Ajou University Hospital

Suwon, Gyeonggido, 16499, South Korea

RECRUITING

Yonsei University Health System, Severance Hospital

Seoul, 03722, 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

Study Officials

• Dong Ah Shin, MD

  Yonsei University Health System, Severance Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sarang Kim

CONTACT

+82 70-2205-0023; info@sbiomedics.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: All subjects with damage to C4-C7 cords diagnosed as AIS-A are administered with PSA-NCAM(+) NPC.

Study Record Dates

• First Submitted: February 24, 2021
• First Posted: March 23, 2021
• Study Start: September 23, 2021
• Primary Completion (Estimated): October 1, 2028
• Study Completion (Estimated): September 1, 2030
• Last Updated: May 18, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

KR (2)