Clinical trial NCT01772810: Safety Study of Human Spinal Cord-derived Neural Stem Cell Transplantation for the Treatment of Chronic SCI
Official title: A Phase 1, Open-label, Single-site, Safety Study of Human Spinal Cord-derived Neural Stem Cell Transplantation for the Treatment of Chronic SCI
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Spinal Cord Injury (SCI)
Keywords: Paralysis, spinal cord injury, chronic spinal cord injury, cspine injury, neck injury
Lead sponsor: Neuralstem Inc.
Sponsor class: INDUSTRY
First posted: 2013-01-21
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a safety study of human spinal cord-derived neural stem cell (HSSC) transplantation for the treatment of chronic spinal cord injury.

Detailed description: Phase I, open-label, single-site, safety study of HSSC transplantation for the treatment of chronic spinal cord injury (SCI). Group A enrolled 4 subjects with a cord injury at T2-T12; Group B will enroll 4 subjects with a C5-C7 cord injury. Study period will be 6 months post-operative. Post-study, subjects will be followed for an additional 54 months.

Eligibility criteria: Inclusion Criteria:

* Lives within 500 miles of study center (For Group B)

  1. Have the ability to understand the requirements of the study, provide written informed consent, understand and provide written authorization for the use and disclosure of Protected Health Information (PHI) \[per Health Insurance Portability and Accountability Act (HIPAA) Privacy Ruling\] and comply with the study procedures
  2. Men and women 18-65 years old
  3. Women must have a negative serum pregnancy test and practice an acceptable method of contraception or be of non-childbearing potential (post-menopausal for at least 2 years or who have undergone hysterectomy or oophorectomy or surgical sterilization)
  4. At least 1 year but no more than 2 years from time of injury at the time of surgery
  5. SCI injury classified as AIS-A complete as confirmed by the PI and a Physical Medicine and Rehabilitation doctor or other SCI Medicine board certified physician based on a complete ISNC SCI examination
  6. Neurologic level of injury:

     1. Group A: injury within cord segments T2-T12;
     2. Group B: injury within cord segments C5-C7
  7. Confirmation of bone fusion by CT scan
  8. Agrees to the visit schedule as outlined in the informed consent
  9. All required vaccinations current at the time of enrollment: tetanus/diptheria (TDAP), herpes zoster/shingles (Zostavax®: within last 10 years and must be prior to surgery), pneumonia (Pneumovax®), seasonal/H1N1 flu vaccines (as appropriate for season). Any missing vaccination will be provided at the screening if consented by the otherwise eligible patient who will then be scheduled for surgery no less than 30 days post vaccination.

Exclusion Criteria:

1. SCI due to penetrating trauma such as gun shot or stabbing wound
2. SCI injuries involving complete spinal cord transection
3. Etiology of paraplegia or weakness related to other or additional neurologic process
4. MRI evidence of syrinx, multiple cysts, or a cyst with greater than 2cm in length or injury site involving greater than 2cm of the cord
5. Current or peak Panel Reactive Antibody (PRA) due to alloantibodies \> 20% receiving their first allograft or presence of antibodies against HLA of the donor cells
6. Receipt of any investigational drug or device within 30 days prior to surgery
7. Receipt of any cell infusion other than blood transfusion
8. Any concomitant medical disease or condition noted below:

   1. Coagulopathy with INR \> 1.4 at the time of screening
   2. Active infection
   3. Active hypotension requiring vasopressor therapy
   4. Skin breakdown over the site of surgery
   5. History of Malignancy (except for non-melanoma skin cancer)
   6. Primary or secondary immune deficiency
   7. Persistent MRI artifact that would prevent imaging post-op h. Creatinine \>1.5, liver function tests (SGOT/SGPT, Bilirubin, Alk Phos) \> 2x upper limit of normal, hematocrit/hemoglobin \< 30/10, total WBC \< 3000, uncontrolled hypertension (systolic \> 180 or diastolic \> 100) or uncontrolled diabetes (defined as hemoglobin A1C \>8), evidence of GI bleeding by hemoccult test, positive tuberculosis, hepatitis B or C, or human immunodeficiency virus (HIV)
9. Presence of any of the following conditions:

   1. Current drug abuse or alcoholism
   2. Unstable medical conditions
   3. Unstable psychiatric illness including psychosis and untreated major depression
10. Any condition that the Investigator or primary physician feels may interfere with participation in the study
11. Any condition that the surgeon feels may pose complications for the surgery
12. Known hypersensitivity to basiliximab, tacrolimus or mycophenolate mofetil
13. Inability to provide informed consent as determined by screening protocol.
14. Stage III or above decubitus wound, other open wound, or active colostomy;
15. Autoimmune diseases, for which chronic corticosteroids or immunosuppression therapy may be needed;
16. Implanted spine stimulator;
17. Uncontrolled spasticity;
18. Uncontrolled neuropathic pain

Primary outcomes:
measure: Adverse events and clinically significant laboratory abnormalities
timeFrame: 6 months
Secondary outcomes:
measure: Graft survival in the transplant site as determined by MRI (for Group A) and via autopsy, if one is completed.
timeFrame: Month 60
Other outcomes:
measure: EMG, SCIM, ISNC SCI, and Bowel and bladder follow-up
timeFrame: 60 months
Locations:
city: San Diego
country: United States
facility: UCSD Medical Center, Division of Neurosurgery
state: California
Study officials:
affiliation: UCSD Medical Center, Division of Neurosurgery
name: Joseph Ciacci, MD
role: PRINCIPAL_INVESTIGATOR
References: