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Autologous Stem Cells for Spinal Cord Injury (SCI) in Children

This study has been terminated.
(Principal Investigator relocated to Orlando, Florida.)
Sponsor:
Collaborators:
The Institute for Rehabilitaion and Research Foundation
The University of Texas Health Science Center, Houston
M.D. Anderson Cancer Center
Baylor College of Medicine
Florida Hospital for Children Orlando FL
Information provided by (Responsible Party):
James E.Baumgartner, MD, Memorial Hermann Healthcare System
ClinicalTrials.gov Identifier:
NCT01328860
First received: April 1, 2011
Last updated: June 6, 2014
Last verified: June 2014

April 1, 2011
June 6, 2014
April 2011
June 2014   (final data collection date for primary outcome measure)
American Spinal Injury Association (ASIA) - Standard Neurological Classification of Spinal Cord Injury. [ Time Frame: 180 days ] [ Designated as safety issue: No ]
ASIA classification assessment will take place pre-procedure, 1 day, 30 days, and 180 days post-procedure to define changes in functional outcome.
Same as current
Complete list of historical versions of study NCT01328860 on ClinicalTrials.gov Archive Site
Standard Neuropathic Pain Rating Scale [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
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.
Same as current
Not Provided
Not Provided
 
Autologous Stem Cells for Spinal Cord Injury (SCI) in Children
Safety of Autologous Stem Cell Treatment for Spinal Cord Injury in Children

The purpose of this research study is:

  1. To see if Bone Marrow Cell harvest and transplantation are safe in children with Spinal Cord Injury, and
  2. 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.

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.

Interventional
Phase 1
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Spinal Cord Injury
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.
Other Names:
  • Autologous Stem Cells
  • Patient's own Stem Cells
Experimental: Biologic; Stem Cells
Intervention: Biological: bone marrow progenitor cells (BMPC) autologous transplantation
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Terminated
10
June 2016
June 2014   (final data collection date for primary outcome measure)

Inclusion Criteria:

  1. Between 1 year and 15 years of age on the day of study BMPC infusion.
  2. Survived at least six months with PSCI, but are less than 4 years post injury (± 30 days), and have fixed neurologic deficits related to their injury at the time of enrollment.
  3. Ability of child to understand and speak English.
  4. Ability of child and caregiver to travel to Houston, Texas, and stay for at least 4 days, and to return for all Follow-up visits (patient is responsible for cost of travel and lodging while in Houston).
  5. Pediatric patients with any type of spinal cord injury as long as their spinal cords are continuous on MRI evaluation. This includes paraplegic and quadriplegic patients with complete or incomplete spinal cord injuries. This includes patients with ASIA impairment scales from A to D. The clinical classification will be described by the level below which motor/sensory function is impaired, and the degree of that impairment. (i.e. C-5 if the deltoid muscle is intact but the biceps and other muscle groups below the C-5 level are weak [incomplete motor injury] or paralyzed [complete motor injury]. A similar evaluation of sensory function will be established clinically.

Exclusion Criteria:

  1. Lack of informed consent.
  2. Uncorrected coagulopathy during the baseline period defined as: INR > 1.4; PTT > 35 sec; PLT < 100,000.
  3. Pre-injury history of seizure disorder and/or neurological impairment where the patient would not be able to participate in age appropriate pain rating scales.
  4. A history of prior SCI or severe traumatic brain injury.
  5. Known history of:

    • Recently diagnosed infection (within past 2 weeks) requiring treatment and/or medical intervention.
    • Renal disease or altered renal function as defined by serum creatinine > 1.5 mg/dL.
    • Hepatic disease or altered liver function as defined by SGPT > 150 U/L, and/or T. Bilirubin > 1.3 mg/dL.
    • Malignancy.
    • Immunosuppression as defined by WBC < 3 (10x3) at screening and/or baseline evaluation lab.
    • HIV.
    • Hepatitis B or C.

8. Unhealed fractures or wounds including osteomyelitis.

9. Pneumonia, or chronic lung disease requiring oxygen.

10. An anatomically discontinuous spinal cord diagnosed by CT or MRI imaging.

11. Positive urine pregnancy test (urine pregnancy test will be routinely performed on females of childbearing potential, age 11 or older.

12. Participation in a concurrent intervention study.

13. Desire for organ-donation in the event of death.

14. Unwillingness or inability to stay for at least four days following BMPC infusion (should any problems arise following the infusion) and to return for a 30 day, and 6 month follow-up visit, and be available for 1 year, and 2 year follow-up phone calls.

Both
1 Year to 15 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01328860
BBIND14281
Yes
James E.Baumgartner, MD, Memorial Hermann Healthcare System
James E.Baumgartner, MD
  • The Institute for Rehabilitaion and Research Foundation
  • The University of Texas Health Science Center, Houston
  • M.D. Anderson Cancer Center
  • Baylor College of Medicine
  • Florida Hospital for Children Orlando FL
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
Memorial Hermann Health System
June 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP