Efficacy of Stem Cell Transplantation Compared to Rehabilitation Treatment of Patients With Cerebral Paralysis

Study Overview
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Brief Summary
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Cerebral palsy (CP) is described as a group of permanent disorders affecting motor development and posture, resulting in activity limitation attributed to nonprogressive disturbances of the fetal or infant brain. The prevalence of cerebral palsy has increased among the children with low birth-weight, jaundice, respiratory distress and intrauterine infection and so on. The incidence of cerebral palsy is increasing gradually with increased neonatal survival rate. Although there are many kinds of functional therapy programs especially the rehabilitation treatment for cerebral palsy, their effects are limited. Increasing cerebral palsy patients become a heavy burden to the family and society. Stem cell based therapy, a new prospective therapy for central nervous system disorders, has the potential to repair the damaged brain tissue in patients with cerebral palsy. In this study, 300 patients with cerebral palsy will be divided into three groups and the investigators will use mesenchymal stem cells derived from umbilical cord to treat 100 CP patients of them randomly. We will also follow up the other 100 patients who only receive rehabilitation treatment and another 100 patients who accept neither stem cell therapy nor rehabilitation treatment. On this basis, as the investigators we can compare the efficacy of cell therapy and rehabilitation treatments for cerebral palsy patients. Multiple sources of assessment were used to ascertain and classify all cases of cerebral palsy. Particularly the Gross Motor Function Measure (GMFM) as an important valid and reliable outcome measure, has made it possible to evaluate the severity of movement disability，change over time and the effects of clinical interventions. It also will be the primary outcome measure in follow-up analysis of this study.

Detailed Description
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Patients enrolled in this study need to finish our whole follow-up survey for 12 months, which is carried out by clinical doctors, rehabilitators and epidemiologist.

Official Title
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Efficacy of Stem Cell Transplantation Compared to Rehabilitation Treatment of Children With Cerebral Paralysis

Conditions
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Cerebral Palsy

Intervention / Treatment
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* Other: rehabilitation
* Biological: stem cell injection


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with diagnosis of cerebral palsy. Patients' curator must be able to give voluntary consent. Exclusion Criteria: Intracranial infection. Severe respiratory and circulatory system diseases. Hematologic malignancies. Positive serological tests such as AIDS, hepatitis B virus, hepatitis C virus and syphilis （antigen or antibody）. Tumors. Genetic and metabolic diseases.

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 14 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: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: rehabilitation<br>Patients in the group accept rehabilitation for three weeks in hospital and other eleven months in their home under the guidance of physical therapist. | Other: rehabilitation<br>* Patients only receive rehabilitation of physical therapy and occupational therapy.<br>* Other names: phsical exercise rehabilitation;|
| No Intervention: control<br>Patients receive no professional treatment in hospital or rehabilitation centre. | |
| Experimental: stem cell injection<br>Patients in the group accept cell therapy including four times stem cells transplant via intrathecal injection. | Biological: stem cell injection<br>* Mesenchymal stem cells derived from umbilical cord are transplanted directly into subarachnoid by Lumbar puncture.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Gross Motor Function Measure Score | Gross Motor Function Measure-88 and Gross Motor Function Measure-66 | Change from baseline at 12 monthes after enrollment or transplantation | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Routine Blood Test and Biochemical Test | red blood cell white blood cell platelet count glutamic pyruvic transaminase glutamic oxaloacetic transaminase | Change from baseline at 12 monthes after enrollment or transplantation | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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cerebral palsy, cell transplantation, rehabilitation