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

NCT ID: NCT01328860

Last Updated: 2024-03-20

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE1
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-04-30
• Study Completion Date: 2016-06-30

Brief Summary

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.

Detailed Description

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.

Conditions

Spinal Cord Injury

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Biologic; Stem Cells

Group Type: EXPERIMENTAL

bone marrow progenitor cells (BMPC) autologous transplantation

Intervention Type: BIOLOGICAL

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.

Interventions

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.

Intervention Type: BIOLOGICAL

Other Intervention Names

Autologous Stem Cells; Patient's own Stem Cells

Eligibility Criteria

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.

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 15 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The Institute for Rehabilitaion and Research Foundation

OTHER

Sponsor Role: collaborator

The University of Texas Health Science Center, Houston

OTHER

Sponsor Role: collaborator

M.D. Anderson Cancer Center

OTHER

Sponsor Role: collaborator

Baylor College of Medicine

OTHER

Sponsor Role: collaborator

Florida Hospital for Children

OTHER

Sponsor Role: collaborator

Aryn Knight

OTHER

Sponsor Role: lead

Responsible Party

Aryn Knight

AVP Research

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

James E. Baumgartner, MD

Role: STUDY_CHAIR

MHHS, Houston,TX & FL Hospital for Children, Orlando, FL

Rex A. Marco, MD

Role: PRINCIPAL_INVESTIGATOR

University of Texas Health Science Center, Houston TX

Locations

Children's Memorial Hemann Hospital; University of Texas Health Science Center - Houston

Houston, Texas, United States

Countries

United States

Other Identifiers

BBIND14281

Identifier Type: -

Identifier Source: org_study_id