Intrathecal Stem Cells for Cerebral Palsy Phase II

Study Overview
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Brief Summary
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The purpose of this study is to determine whether the infusion of intrathecal autologous bone marrow total nucleated cells would improve the neurologic evolution of pediatric patients with quadriparetic cerebral palsy.

Detailed Description
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There is accumulated evidence that shows that the administration of bone marrow total nucleated cells (TNC) into the brain may produce some benefits by different mechanisms like cytokine production, in several neurological areas such as motor, social, adaptative and cognitive. It has been found that after introducing TNC in the subarachnoid space of the spinal cord, these cells may be transported through the cerebrospinal fluid and can be delivered more efficiently to the injured area, compared with intravenous route in patients with neurologic injury. Patients will be stimulated for 3 consecutive days with subcutaneous granulocyte colony stimulating factor (G-CSF) and then their bone marrow will be harvested according to their weight. Bone marrow will be processed in order to obtain the buffy coat and minimize the amount of red blood cells. An inoculum of 10mL of this TNC will be infused intrathecally. Patients will be evaluated with the Gross Motor Functional Classification System before the procedure and one, three and six months after that.

Official Title
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Intrathecal Autologous Stem Cells for Children With Cerebral Palsy Phase II

Conditions
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Cerebral Palsy

Intervention / Treatment
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* Biological: Autologous Stem Cell Transplantation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with quadriplegic cerebral palsy, with an age from 7 to 9 years of age, regardless the age at the time of injury, time post-injury, or previously received therapies, different from ours Exclusion Criteria: Patients with neurodegenerative or autoimmune diseases Patients with active infection in any organ or tissue at the time of entering the study, the onset of stimulation with G-CSF or at the procedure Patients who do not sign the informed consent form

Ages Eligible for Study
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Minimum Age: 7 Years
Maximum Age: 9 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Supportive Care
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Intrathecal Autologous Bone Marrow TNC<br>Procedure/Surgery: Intrathecal Autologous Bone Marrow TNC. Other Names: Autologous Stem Cell Transplantation Patients will be stimulated with Granulocyte Colony Stimulating Factor (G-CSF) (10mcgr/kg of body weight) for 3 consecutive days. Bone marrow will be harvested under sedation and, after being processed in the laboratory, the autologous TNC concentrate of 10 mL will be infused intrathecally. | Biological: Autologous Stem Cell Transplantation<br>* Patients will be stimulated with Granulocyte Colony Stimulating Factor (G-CSF) 3 consecutive days. Bone marrow will be harvested under sedation and, after being processed in the laboratory, the buffy coat (TNC) of 10 mL will be infused intrathecally. Intrathecal Autologous Bone Marrow TNC<br>|
| No Intervention: Control group<br>Patients will be evaluated with the Gross Motor Functional Classification System initially, at one, three and six months, and then cross to the intervention arm. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants who show progress in GMFCS scale after autologous bonemarrow TNC intrathecal infusion. | Patients, in an outpatient basis, will be stimulated with Granulocyte Colony Stimulating Factor (G-CSF) for 3 consecutive days. Their bone marrow will be harvested under sedation and, after being processed in the laboratory, the buffy coat of 10 mL will be infused intrathecally. Patient will be monitored during acute phase to register any adverse effect (malaise, headache, fever, and nausea or vomit, etc).Gross Motor Function Classification System (GMFCS) scale is going to be monitored at baseline, one, three and six months to measured differences between the scales. GMFCS is based on patient's self-initiated movement with particular emphasis on sitting, walking, and wheeled mobility. Distinctions between levels are based on functional abilities, the need for assistive technology, including hand-held mobility devices (walkers, crutches, or canes) or wheeled mobility, and to a much lesser extent, quality of movement. | six months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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brain, children, cerebral palsy, bone marrow stem cells, intrathecal autologous total nucleated cells, quadriparesis