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Safety and Exploratory Efficacy of Transplantation Therapy Using PSA-NCAM(+) NPC in AIS-A Level of Sub-acute SCI (SB-SCI-001)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04812431
Recruitment Status : Recruiting
First Posted : March 23, 2021
Last Update Posted : May 5, 2022
Sponsor:
Collaborators:
Linical Co., Ltd.
Yonsei University
Information provided by (Responsible Party):
S.Biomedics Co., Ltd.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Spinal Cord Injury, Acute Spinal Cord Injury at C4 Level With Complete Lesion Spinal Cord Injury at C5-C7 Level With Complete Lesion Biological: Neural precursor cells derived from human embryonic stem cell line Phase 1 Phase 2

Detailed Description:

Subjects with damage to C4-C7 cords diagnosed as AIS-A are administered with PSA-NCAM(+) NPC.

For evaluation of safety and exploratory efficacy, 2 to 6 subjects will be enrolled depending on the presentation of dose-limiting toxicity.

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. When the DLT is presented in two or more of the first three patients, the clinical study is discontinued; when the DLT is presented in one of the three patients, three additional new patients are added. In case of presentation of the DLT in at least one of the three additional patients, the study is discontinued; the clinical study is continued only when the DLT is not presented in all three patients.

Screening visit (Visit 1), surgery and recovery visit (Visit 2 to Visit 6), follow-up visit (Visit 7 to Visit 8 + phone screening I, II, III), additional visit (Visit 9 to Visit 10), and close-out visit (Visit 11) are conducted. A clinical study period of at least 68 weeks is secured after Visit 6 (at least 5 visits and 3 phone screenings).

All subjects are to be conducted of follow-up study of a period of 1 year and 5 months at Weeks 1, 2, 4, 8, 12, 24, 48, and 72 after surgery.

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 5 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: All subjects with damage to C4-C7 cords diagnosed as AIS-A are administered with PSA-NCAM(+) NPC.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Single Center, Open Label, Single Group, Phase 1/2a Clinical Study to Evaluate the Safety and Exploratory Efficacy of Transplantation Therapy Using PSA-NCAM(+) NPC Derived From hESC Line in AIS-A Level of Sub-acute SCI(From 7 to 60 Days)
Actual Study Start Date : September 23, 2021
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : September 2028

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: PSA-NCAM(+) NPC
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
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.


Outcome Measures
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Primary Outcome Measures :
  1. Changes of occurred adverse events during the clinical study [ Time Frame: 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) ]
    Assessment of the presentation rate of adverse events occurred from IP administration until the end of the clinical study (Visit 11).

  2. Rate of abnormal signs as assessed through physical examination [ Time Frame: Visit 1(Screening), Visit 11(72 weeks) ]
    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).

  3. Measured changes of Systolic blood pressure (mm Hg) [ Time Frame: 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) ]
    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).

  4. Measured changes of Body temperature (degrees Celsius) [ Time Frame: 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) ]
    Comparative assessment of measured value using body temperature (degrees Celsius) from the baseline (Visit 1) to the end of the clinical study (Visit 11).

  5. Measured changes of Heart rate (beats per minute) [ Time Frame: 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) ]
    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).

  6. Measured changes of Respiratory rate (breaths per minute) [ Time Frame: 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) ]
    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).

  7. Rate of abnormal hematology values [ Time Frame: 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) ]
    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).

  8. Rate of abnormal chemistry values [ Time Frame: 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) ]
    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).

  9. Rate of abnormal urinalysis values [ Time Frame: 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) ]
    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).

  10. Measured changes of diastolic blood pressure (mm Hg) [ Time Frame: 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) ]
    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).


Secondary Outcome Measures :
  1. Assessment of Graft survival at the transplant site as observed in MRI examination [ Time Frame: Visit 1(Screening), Visit 11(72 weeks) ]
    Number of participants with measured value(spinal cord changes, spinal cord atrophy far from the lesion, edema, cystic degeneration, spinal sinus, and diameter change) using MRI examination.

  2. Number of participants with Transplant rejection against transplanted cells [ Time Frame: Visit 1(Screening), Visit 6(4 weeks) ]
    Comparative Assessment of PSA-NCAM(+) NPC-specific antibody that may result in transplant rejection in the HLA antigen/antibody reaction test.

  3. Changes in the ASIA Damage Scale [ Time Frame: Visit 1(Screening), Visit 8(12 weeks), Visit 11(72 weeks) ]
    Number of participants with changes in ISNCSCI ASIA at Week 12 (Visit 8) and Week 72 (Visit 11) of administration compared to the baseline (Visit 1).

  4. International standards for neurological classification of spinal cord injury (ISNCSCI) motor index score [ Time Frame: Visit 1(Screening), Visit 6(4 weeks), Visit 8(12 weeks), Visit 9(24 weeks), Visit 10(48 weeks), Visit 11(72 weeks) ]

    Number of participants with motor level recovered* at Week 4 (Visit 6), Week 12 (Visit 8), Week 24 (Visit 9), Week 48 (Visit 10), and Week 72 (Visit 11) of drug administration compared to the baseline (Visit 1).

    * Recovery: Subjects with recovery of at least 2 stages in the upper limb motor score


  5. Measured changes of International standards for neurological classification of spinal cord injury (ISNCSCI) sensory index score [ Time Frame: Visit 1(Screening), Visit 8(12 weeks), Visit 11(72 weeks) ]
    Rate of changes in the total ISNCSCI ASIA sensory index score at Week 12 (Visit 8) and Week 72 (Visit 11) of administration compared to the baseline (Visit 1).

  6. Pain assessment [ Time Frame: Visit 1(Screening), Visit 8(12 weeks), Visit 11(72 weeks) ]
    Rate of measured changes in the pain scores at Week 12 (Visit 8) and Week 72 (Visit 11) compared to the baseline (Visit 1).

  7. Spinal Cord Independence Measure (SCIM) score [ Time Frame: Visit 1(Screening), Visit 8(12 weeks), Visit 11(72 weeks) ]
    Rate of change in the SCIM score at Week 12 (Visit 8) and Week 72 (Visit 11) compared to the baseline (Visit 1).

  8. Measured changes of International standards for neurological classification of spinal cord injury (ISNCSCI) motor index score [ Time Frame: Visit 1(Screening), Visit 8(12 weeks), Visit 11(72 weeks) ]
    Rate of changes in the total ISNCSCI ASIA motor score at Week 12 (Visit 8) and Week 72 (Visit 11) of administration compared to the baseline (Visit 1).


Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Upon written consent of the patient or the legally acceptable representative of the patient
  2. Male and female patients 18 to 65 years of age

  3. 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

  4. Patients who are capable of being administered with the investigational product on Day 7 to 60 after spinal cord injury
  5. 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
  6. Patients whose ASIA Impairment Scale (AIS) satisfies AIS-A criteria (spinal segment within C4-C7, complete injury)

Exclusion Criteria:

  1. Patients with spinal cord injury caused by penetrating trauma such as gunshot or stab wounds
  2. Patients with complete transection on the spinal cord
  3. Patients with spinal cord injury that require more than the mono-segment treatment
  4. 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
  5. Patients with multiple lesions or lesions longer than 2 cm in length found on MRI examination
  6. Patients administered with cells excluding blood transfusion before participating in the clinical study

  7. Patients with the following intercurrent diseases or conditions:

    1. Coagulopathy with INR> 1.4 at the time of administration of the investigational product (Day 0)
    2. Active infection
    3. Active hypotension requiring vasoconstrictor treatment (less than 60 mmHg of diastolic blood pressure)
    4. Rupture of the skin on the area of surgery
    5. Medical history of malignant tumor
    6. Primary or secondary immunodeficiency

    7. Clinically significant abnormal values discovered as a result of laboratory tests

      • Creatinine > 1.5 mg/dL
      • When the level found in the liver-function examination is more than twice the upper limit of the normal level
      • Hematocrit/hemoglobin <30%/10 g/dL
      • Total WBC < 1000/μL
      • Uncontrolled hypertension (systole> 180 mmHg or diastole> 100 mmHg)
      • Uncontrolled diabetes (HbA1c> 8%)
      • Evidence of GI bleeding on the stool guaiac test
      • Positive on tuberculosis test (However, a chest X-ray may be performed if found positive on the TB test, and the patient may be enrolled upon no findings that suggest active tuberculosis on the chest X-ray.)
      • Hepatitis B or C
      • Human Immunodeficiency Virus (HIV)
    8. Substance abuse or alcoholism
    9. Unstable or untreated psychiatric disorder
  8. Patients with known hypersensitivity to basiliximab, tacrolimus, mycophenolate mofetil, methylprednisolone, or prednisone
  9. Patients incapable of receiving physical therapy or combination therapy
  10. Patients incapable of going under general anesthesia due to other reasons
  11. Patients judged unsuitable for participation in this clinical study by the investigator
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04812431


Contacts
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Contact: Sarang Kim +82 70-2205-0023 info@sbiomedics.com

Locations
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Korea, Republic of
Ajou University Hospital Recruiting
Suwon, Gyeonggido, Korea, Republic of, 16499
Contact: Nam Gyu Yu, MD       nkyou@ajou.ac.kr   
Yonsei University Health System, Severance Hospital Not yet recruiting
Seoul, Korea, Republic of, 03722
Contact: Dong Ah Shin, MD    +82 2-2228-2150    CISTERN@yuhs.ac   
Sponsors and Collaborators
S.Biomedics Co., Ltd.
Linical Co., Ltd.
Yonsei University
Investigators
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Principal Investigator: Dong Ah Shin, MD Yonsei University Health System, Severance Hospital
More Information
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Responsible Party: S.Biomedics Co., Ltd.
ClinicalTrials.gov Identifier: NCT04812431    
Other Study ID Numbers: HI20C0168000020
First Posted: March 23, 2021    Key Record Dates
Last Update Posted: May 5, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by S.Biomedics Co., Ltd.:
Spinal cord injury
AIS-A
Additional relevant MeSH terms:
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Spinal Cord Injuries
Wounds and Injuries
Spinal Cord Diseases
 Central Nervous System Diseases
Nervous System Diseases
Trauma, Nervous System