Bone Marrow-Derived Stem Cell Transplantation for the Treatment of Cerebral Palsy

Study Overview
=================
Brief Summary
-----------------
This study is a single arm, single center trial to study the safety and efficacy of purified autologous bone marrow derived stem cells and mesenchymal stem cells for the treatment of children with cerebral palsy.

Detailed Description
-----------------
Cerebral palsy (CP) is a chronic childhood disorder defined by a range of motor and cognitive impairments and results in a substantial suffering to the patient and a tremendous socio-economic burden to the individual, family, and healthcare system. With no effective treatments or interventions, therapies for CP are currently focused on supportive and management strategies. Stem cell transplantation has been suggested as a putative intervention for neural pathology, as mesenchymal and neural stem cells, as well as olfactory ensheathing glia and Schwann cells, which have shown some regenerative and functional efficacy in experimental central nervous system disorders. In this trial, the investigators study the safety and efficacy of the intrathecal transplantation of specific populations of purified autologous bone marrow-derived stem cells and mesenchymal stem cells in patients with cerebral palsy.

Official Title
-----------------
Safety and Efficacy of Transplantation of Specific Populations of Stem Cells and Mesenchymal Stem Cells for the Treatment of Cerebral Palsy

Conditions
-----------------
Cerebral Palsy

Intervention / Treatment
-----------------
* Biological: Stem Cell Therapy


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Must be more than 2 year of age and less than 12 years of age at the time of screening for inclusion in the study. Clinical evidence of a non-progressive motor disability due to brain dysfunction. Willing to comply with all study procedures. Gross Motor Function Classification Score level II-V Exclusion Criteria: Known history of: Intractable seizures Traumatic brain injury Genetic disorder Current Infection Renal insufficiency Hepatic disease HIV+ (as demonstrated by positive blood test) Immunosuppression Infectious related neurological injury Other etiologies such as degenerative, mitochondrial, and metabolic disorders Normal brain MRI Evidence of acute illness such as fever (temperature > 37.5 C), vomiting, diarrhea, wheezing or crackles Progressing neurological disease (Batten Disease, Leukodystrophies, Neurotransmitter disorders) Microcephaly, macrocephaly, cortical malformations, or genetic disorders of dysgenesis Pulmonary disease requiring ventilator support Unwillingness to return for follow-up visits Contraindications to MRI

Ages Eligible for Study
-----------------
Minimum Age: 2 Years
Maximum Age: 12 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Stem Cells<br>Intravenous and Intrathecal transplantation of specific populations of purified bone marrow-derived stem cells and mesenchymal stem cells. | Biological: Stem Cell Therapy<br>* Intravenous and Intrathecal transplantation of purified autologous bone marrow-derived stem cells and mesenchymal stem cells.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in Motor Performance. | GMPM (Gross Motor Performance Measure) as a standard measurement tool for assessing quality of movement. | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Motor function studies | Total volumes and specific tract lesions will be studied and correlated with functional outcomes. | 1 year | 
| Specific white matter tract analysis using MRI | Changes on brain Diffusion Tensor Image (DTI). | 6 months | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
stem cells