Clinical Effect and Safety of Autologous Umbilical Cord Blood Transfusion in the Treatment of Cerebral Palsy

Study Overview
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Brief Summary
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To study the clinical efficacy and safety of autologous umbilical cord blood transfusion in the treatment of cerebral palsy.

Detailed Description
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This is a Phase 1 clinical trial that constitutes two time points cohorts with participants who will receive intravenous autologous umbilical cord blood and 0.9% sodium chloride respectively. The timing of treatment is any time after the diagnosis of cerebral palsy. The investigator will proceed the groups during the same period. Demographic Data and Baseline Characteristics of the Studied Group were collected: Basic patient's information survey Medical history Physical examination Basic blood test result Children's developmental disorders evaluation before the treatment Brain Magnetic Resonance Imaging-Diffusion Tensor Imaging (MRI-DTI) before the treatment Neurocognitive function test before the treatment Assessment of clinical condition in the course by measurement of blood pressure, heart and respiratory rates, temperature and adverse events was recorded. Autologous cord blood doses is 20-30ml (total Mononuclear cells>1*10^7/kg)，the infusion speed is 1ml/min, and with same volume of 0.9% sodium chloride as placebo. The follow-up: clinical test until 30th month in 3 month gaps.

Official Title
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Clinical Effect and Safety of Autologous Umbilical Cord Blood Transfusion in the Treatment of Cerebral Palsy

Conditions
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Safety Issues, Effect of Drugs

Intervention / Treatment
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* Drug: 0.9% Sodium Chloride
* Biological: Autologous Umbilical Cord Blood


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: A patient who was diagnosed with a mild case, and severity case of cerebral palsy or developmental disability through developmental assessment from more than two specialists including a specialist in pediatrics and rehabilitation medicine.. In case there is a sign of the general decrease in white matter, or periventricular leukomalacia near cerebral ventricles in MRI DTI test. A subject who did a written consent to participation in this clinical trial through the very person and a legal representative. Exclusion Criteria: In case a patient underwent a surgical procedure, or was administered autologous cord blood within one year before participating in a clinical trial. Accompanied by a serious disease, such as chromosome abnormality, etc. In case where a patient's medical condition is judged to be maladapted by a researcher. In case a patient or his or her legal representative doesn't agree to participation in a clinical trial. A patient having a predisposition to allergies. A patient having serious disorders in the liver, kidney, and cardiopulmonary function.

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 10 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: Non-Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Parallel assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Placebo<br>0.9% sodium chloride infusion any time after entering the Placebo Group, doses is 20-30ml. The infusion speed is 1ml/min. | Drug: 0.9% Sodium Chloride<br>* 0.9% Sodium Chloride in control group<br>|
| Experimental: Cord Blood Mononuclear Cells(CBMNC)<br>Autologous Umbilical Cord Blood Mononuclear Cells Therapy any time after the diagnosis of cerebral palsy, doses is 20-30ml (total Mononuclear cells>1*10^7/kg). The infusion speed is 1ml/min. | Biological: Autologous Umbilical Cord Blood<br>* Autologous Umbilical Cord Blood Mononuclear Cells Therapy in Cerebral Palsy for safety and effect evaluation.<br>* Other names: Autologous Umbilical Cord Blood Mononuclear Cells Therapy;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The Change of Gross Motor Function Classification System(GMFCS) Score | GMFCS is used to assess the gross motor function of the children who were diagnosised as cerebral palsy.The results of GMFCS are divided into five levels. Grade I represents the best outcome while grade Ⅴ represents the worst. | up to 30 months after therapy at a 3-month interval | 
| The Change of Gesell Developmental Scale(GDS) Score | The GDS is used to measure the gross motor,fine action,adaptability，language and individual-society function of the children whose age ranges from 1-month to 6-year-old. The score less than 75 for each subscale represent the worse outcome and more than 75 represent the better. | every 3-month after therapy until 30 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cerebral palsy, Autologous Umbilical Cord Blood, safety, effect