Neural Stem Cells Therapy for Cerebral Palsy

Study Overview
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Brief Summary
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To evaluate the safety and efficacy of neural stem cells (NSCs) therapy for cerebral palsy.

Detailed Description
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Cerebral palsy is the most severe lifelong disability disease in childhood. In China, an epidemiological investigation of cerebral palsy in 2004 has reported that 2.74‰ (about 6.50-9.75 million) of children suffered from cerebral palsy. The costs of medical expenses, living allowance and nursing care can go up to RMB 20000-50000 yuan per year for each child with cerebral palsy and thus China will expend RMB 120-300 billion yuan per year for children with cerebral palsy. With full liberalization of two-child policy, there is an increasing number of second babies born to women who are 35 years or older. Thus, the incidence of cerebral palsy tends to increase. There is currently no cure for cerebral palsy, resulting in a heavy burden for families and on society. Recent clinical trials performed in countries outside China have demonstrated that stem cell therapy is likely the most effective method to treat cerebral palsy. Fifteen trials regarding stem cell therapy for cerebral palsy have been registered with ClinicalTrials.gov. In China, there have been no standardized registered trials regarding stem cell therapy for cerebral palsy, which greatly restricts its clinical application. In October 2016, the National Health and Family Planning Commission and the Food and Drug Administration of China jointly announced the premier stem cell clinical research institutions, making performing standardized trials regarding stem cell therapy for cerebral palsy in China possible.

Official Title
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Neural Stem Cells Therapy for Cerebral Palsy: a Prospective, Randomized, Parallel-controlled Trial

Conditions
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Cerebral Palsy

Intervention / Treatment
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* Procedure: neural stem cells therapy group
* Other: the control group


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Neonates with ischemic/hypoxic cerebral palsy (mainly including neonate with asphyxia and premature infants); Clinical manifestation of spastic tetraplegia; With moderate to severe cerebral palsy, The Gross Motor Function; Classification System (GMFCS) levels IV-V; Age 1-5 years, of either sex; Provision of signed informed consent by one of his/her parents or legal guardians prior to commencement of this study; Exclusion Criteria: Systemic diseases that possibly influence treatment or patient's compliance; Potentially life-threatening diseases involving various organ systems; Brain deformity; Abnormal behaviors or mood disorder; Allergies from blood products; Suffering from infectious disease; Subjected to craniocerebral operations prior to screening.

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 12 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: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: neural stem cells therapy group<br>The patients will be assigned to neural stem cells therapy group for cerebral palsy. | Procedure: neural stem cells therapy group<br>* The patients will be assigned to neural stem cells therapy group for cerebral palsy.<br>|
| Experimental: the control group<br>The patients will be assigned to the control group for cerebral palsy. | Other: the control group<br>* The patients will be assigned to the control group for cerebral palsy.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| GMFM-88 score | To evaluate change in gross motor function in children with cerebral palsy. | changes of month 1, month 3, month 6, month 9 and month 12 after therapy | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Fine Motor Function Measure (FMFM) score | To evaluate change in fine motor function in children with cerebral palsy. | changes of month 1, month 3, month 6, month 9 and month 12 after therapy | 
| Modified Ashworth Scale score | To measure resistance during passive soft-tissue stretching and is used as a simple measure of spasticity. | changes of month 1, month 3, month 6, month 9 and month 12 after therapy |