Clinical trial NCT01046786: Safety and Feasibility of Umbilical Cord Blood Cell Transplant Into Injured Spinal Cord
Official title: Safety and Feasibility of Umbilical Cord Blood Cell Transplant Into Injured Spinal Cord: an Open-Labeled, Dose-Escalating Clinical Tiral
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Spinal Cord Injuries
Keywords: spinal cord injury
cord blood mononuclear cell
Methylprednisolone
Lithium Carbonate
Lead sponsor: China Spinal Cord Injury Network
Sponsor class: NETWORK
Collaborators: name: Chinese University of Hong Kong
name: The University of Hong Kong
First posted: 2010-01-12
Enrollment: 8
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: To investigate the feasibility, safety, efficacy and optimal dose of umbilical cord blood mononuclear cell transplant in the treatment of chronic spinal cord injuries.

Detailed description: This is an open-label, dose-escalating clinical trial. Three groups of four patients will receive transplants of increasing doses of HLA-matched umbilical cord blood mononuclear cell into the spinal cord. In the fourth group, we will transplant the highest volume of cells that did not increase neurological deficits along with a single bolus of 30mg/kg methylprednisolone sodium succinate. In the fifth group of four subjects, we will inject that volume of cells plus a bolus intravenous dose of 30mg/kg methylprednisolone sodium succinate and a 6-week course of oral lithium carbonate titrated to 0.6-1.0 mM serum levels. All the subjects are encouraged to stand or walk for one hour a day after the cell transplantation.

The neurological and walking outcomes will be assessed 1, 2, 6, 24 and 48 weeks after transplantation. The outcomes of the five treatment groups will be compared by analysis of variance and, if possible, correlation with cell dose. Efficacy and safety will be analyzed comparing neurological change scores amongst the five different treatment groups. Loss of motor (\>5 points) or sensor scores (\>2 points) from baseline pre-treatment levels would be considered deleterious. Increases in scores above baseline would be considered beneficial.

Eligibility criteria: Inclusion Criteria:

* Subjects of either gender and 18-60 years old
* Subjects with chronic spinal cord injury (defined as ≧12 months post-initial SCI surgery) with stable neurological findings for at least 6 months
* Subject with a current neurological status of ASIA A
* The neurological level of the subjects is between C5 and T11
* The MRI shows that the injured site of the spinal cord is within three vertebral levels and there is no cyst
* Subjects must be able to read, understand, and complete the Visual Analog Scale
* Subjects who have voluntarily signed and dated an informed consent form, approved by the appropriate IRB, prior to any study-specific procedures

Exclusion Criteria:

* Significant renal, cardiovascular, hepatic and psychiatric diseases
* Significant medical diseases or infection (including but not limited to the carrier of hepatitis B virus or HIV)
* Pregnant or lactating woman
* Female of childbearing potential and who is unwilling to use an effective contraceptive method while enrolled in the study
* The MRI shows that the length of spinal cord lesion exceeds three segments or there is cyst in the spinal cord
* The lesion edge of the spinal cord cannot be determined by imaging technology
* Unavailability of HLA matched umbilical cord blood cells
* Any contraindication of laminectomy operation, MPSS and/or lithium carbonate
* Subject who is currently participating in another investigational study or has been taking any investigational drug within the last 4 weeks prior to screening of this study and finally
* Any criteria, which, in the opinion of the investigator, suggests that the subject would not be compliant with the study protocol and/or would not be suitable to participant this study

Primary outcomes:
measure: ASIA motor scores and sensory scores
timeFrame: 0, 1, 2, 6 24 and 48 weeks
measure: ASIA Impairment Scale grade
timeFrame: 0, 1, 2, 6 24 and 48 weeks
Secondary outcomes:
measure: MRI and Diffusion Tensor Imaging of spinal cord
timeFrame: 0, 1, 24 and 48 weeks
measure: Spinal Cord Independence Measure (SCIM) score
timeFrame: 0, 6, 24 and 48 weeks
measure: Walking Index of Spinal Cord Injury (WISCI) level
timeFrame: 0, 6, 24 and 48 weeks
measure: Modified Ashworth Scale (MAS) of Spasticity
timeFrame: 0, 1, 2, 6, 24 and 48 weeks
measure: Visual Analog Scale (VAS) of pain
timeFrame: 0, 1, 2, 6, 24 and 48 weeks
Other outcomes:
Locations:
city: Hong Kong
country: Hong Kong
facility: Queen Mary Hospital
city: Shatin
country: Hong Kong
facility: Prince of Wales Hospital
Study officials:
affiliation: The Chinese University of Hong Kong / Prince of Wales Hospital
name: Wai Sang Poon, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: The University of Hong Kong / Queen Mary Hospital
name: Gilberto Leung, MD
role: PRINCIPAL_INVESTIGATOR
References: