Safety and Feasibility of Umbilical Cord Blood Cell Transplant Into Injured Spinal Cord

NCT ID: NCT01046786

Last Updated: 2014-01-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 8 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-01-31
• Study Completion Date: 2013-12-31

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.

Conditions

Spinal Cord Injuries

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Group A

Intraspinal injection of 1.6 million cord blood mononuclear cell

Group Type: ACTIVE_COMPARATOR

Umbilical Cord Blood Mononuclear Cell

Intervention Type: BIOLOGICAL

The cord blood mononuclear cells are obtained from thawed units of HLA-matched human umbilical cord blood. The cells will be slowly injected into the posterior gray matter after laminectomy and opening of the dura.

Group B

Intraspinal injection of 3.2 million cord blood mononuclear cell

Group Type: ACTIVE_COMPARATOR

Umbilical Cord Blood Mononuclear Cell

Intervention Type: BIOLOGICAL

The cord blood mononuclear cells are obtained from thawed units of HLA-matched human umbilical cord blood. The cells will be slowly injected into the posterior gray matter after laminectomy and opening of the dura.

Group C

Intraspinal injection of 6.4 million cord blood mononuclear cell

Group Type: ACTIVE_COMPARATOR

Umbilical Cord Blood Mononuclear Cell

Intervention Type: BIOLOGICAL

The cord blood mononuclear cells are obtained from thawed units of HLA-matched human umbilical cord blood. The cells will be slowly injected into the posterior gray matter after laminectomy and opening of the dura.

Group D

Intraspinal injection of 6.4 million cord blood mononuclear cell plus 30 mg/kg methylprednisolone

Group Type: ACTIVE_COMPARATOR

Umbilical Cord Blood Mononuclear Cell

Intervention Type: BIOLOGICAL

The cord blood mononuclear cells are obtained from thawed units of HLA-matched human umbilical cord blood. The cells will be slowly injected into the posterior gray matter after laminectomy and opening of the dura.

Methylprednisolone

Intervention Type: DRUG

30 mg/kg methylprednisolone

Group E

Intraspinal injection of 6.4 million cord blood mononuclear cell plus 30 mg/kg methylprednisolone plus 6 week course of oral lithium, titrated to maintain 0.6-1.0 mM serum level

Group Type: ACTIVE_COMPARATOR

Umbilical Cord Blood Mononuclear Cell

Intervention Type: BIOLOGICAL

The cord blood mononuclear cells are obtained from thawed units of HLA-matched human umbilical cord blood. The cells will be slowly injected into the posterior gray matter after laminectomy and opening of the dura.

Methylprednisolone

Intervention Type: DRUG

30 mg/kg methylprednisolone

Lithium

Intervention Type: DRUG

oral lithium, titrated to maintain 0.6-1.0 mM serum level

Interventions

Umbilical Cord Blood Mononuclear Cell

The cord blood mononuclear cells are obtained from thawed units of HLA-matched human umbilical cord blood. The cells will be slowly injected into the posterior gray matter after laminectomy and opening of the dura.

Intervention Type: BIOLOGICAL

Methylprednisolone

30 mg/kg methylprednisolone

Intervention Type: DRUG

Lithium

oral lithium, titrated to maintain 0.6-1.0 mM serum level

Intervention Type: DRUG

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Chinese University of Hong Kong

OTHER

Sponsor Role: collaborator

The University of Hong Kong

OTHER

Sponsor Role: collaborator

China Spinal Cord Injury Network

NETWORK

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Wai Sang Poon, MD

Role: PRINCIPAL_INVESTIGATOR

The Chinese University of Hong Kong / Prince of Wales Hospital

Gilberto Leung, MD

Role: PRINCIPAL_INVESTIGATOR

The University of Hong Kong / Queen Mary Hospital

Locations

Queen Mary Hospital

Hong Kong, , Hong Kong

Prince of Wales Hospital

Shatin, , Hong Kong

Countries

Hong Kong

Other Identifiers

CN102B

Identifier Type: -

Identifier Source: org_study_id