Autologous Stem Cells for Spinal Cord Injury (SCI) in Children
The purpose of this research study is:
- To see if Bone Marrow Cell harvest and transplantation are safe in children with Spinal Cord Injury, and
- To determine if late functional outcome is improved following Bone Marrow Cell transplantation in children with Spinal Cord Injury, using pre-transplantation spinal cord function as the control.
|Spinal Cord Injury||Biological: bone marrow progenitor cells (BMPC) autologous transplantation||Phase 1|
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||Safety of Autologous Stem Cell Treatment for Spinal Cord Injury in Children|
- American Spinal Injury Association (ASIA) - Standard Neurological Classification of Spinal Cord Injury. [ Time Frame: 180 days ]ASIA classification assessment will take place pre-procedure, 1 day, 30 days, and 180 days post-procedure to define changes in functional outcome.
- Standard Neuropathic Pain Rating Scale [ Time Frame: 2 years ]Assessment for improvement, worsening or development of neuropathic pain with an age appropriate standard pain rating scale will be performed pre-procedure, and post-procedure days 1 - 14, and again 30 days, 180 days, 1 year, and 2 year post-procedure.
|Study Start Date:||April 2011|
|Estimated Study Completion Date:||June 2016|
|Primary Completion Date:||June 2014 (Final data collection date for primary outcome measure)|
|Experimental: Biologic; Stem Cells||
Biological: bone marrow progenitor cells (BMPC) autologous transplantation
Not more than 5ml/kg bone marrow will be collected under anesthesia; after the bone marrow has been processed, about 6-8 hours after bone marrow aspiration, subjects will receive the cells via intra-venous (in the vein) infusion and will be monitored for 24 hours for any adverse events.
Of the estimated 11,000 cases of acute spinal cord injury (SCI) which occur each year in North America, 5% involve children. The injury is divided into the primary mechanical event which causes the injury, and the secondary events which follow. Outcome for SCI depends of the severity of the primary injury (complete vs. incomplete) and the spinal cord level of the injury. Current therapy is designed only to minimize the secondary events of SCI and other trauma-associated injuries. Because the current therapy does nothing to reverse the primary insult, significant advances in reducing the disability associated with SCI are unlikely. Recent basic science and animal studies suggest that stem cell treatment can foster functional improvement after SCI by helping repair the primary injury and reducing the secondary injury.
Stem cells are "unspecialized" cells in the body that do not have a specific function yet (for example, they have not become "heart cells" or "brain cells" yet.) Stem cells are able to divide and develop into more mature, function-specific cells and take the place of those cells that die, are injured or can no longer function the way they are supposed to. Stem cells are being studied a lot because of this ability and there is the possibility that they may be used to take the place of cells that are no longer working in different parts of the body because of disease (as in cancer, diabetes, and heart disease). Stem cells can be found throughout the body, but they are most common in the bone marrow, the thick, spongy material inside the bones.
The primary objective of this study is to determine the safety of transplantation of the patient's own (autologous) Bone Marrow Progenitor Cells (BMPC) in children with SCI. The secondary objective is to determine if functional, physiological and anatomic outcome measures are improved after BMPC autologous transplantation in children with SCI.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01328860
|United States, Texas|
|Children's Memorial Hemann Hospital; University of Texas Health Science Center - Houston|
|Houston, Texas, United States, 77030|
|Study Chair:||James E. Baumgartner, MD||MHHS, Houston,TX & FL Hospital for Children, Orlando, FL|
|Principal Investigator:||Rex A. Marco, MD||University of Texas Health Science Center, Houston TX|