Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients

Study Overview
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Brief Summary
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Spinal Muscular Atrophy (SMA) is an autosomal recessive disease of motor neurons. In the early 1980s, Werdnig from Vienna University and Hoffman from Heidelberg University described this disorder. So SMA type 1 was named Werdnig- Hoffman disease. This is the first genetic disorder that cause death after cystic fibrosis in infants with the prevalence of 1 in 6000 birth. Mutation in the SMN1 gene (Survival Motor Neuron) is the reason for the disease that cause decrease in the SMN protein production. So the alpha motor neurons in the spinal cord ventricle horn will be destroyed and it cause progressive paralysis and defenite death.No specific therapy is yet available for the treatment of Werdnig-Hoffmann disease. Treatment is not disease-modifying and just is supportive. SMA type 1 is diagnosed within the early 6 month after birth and accompanied with breath disorders and definite death in 2 years. The affected infants have a weak muscle tone and they couldn't even hold their head up. Perhaps the only open way for these patients is the application of stem cells that could deliver trophic factor to the apoptotic cells. So this study focuses on the effectivness of cell therapy via adipose derived mesenchymal stem cells on the probable phenotypic changes in these patients.

Official Title
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The Effectiveness of Allogeneic Adipose Derived Mesenchymal Stem Cells (ADMSCs) in the Phenotypic Changes of Werdnig Hoffman Patients

Conditions
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Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman]

Intervention / Treatment
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* Biological: Adipose derived mesenchymal stem cell


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age under 12 month, Weak muscle tone, Weakness in mobility, Patients sitting without full conduction of nerve Existence of home senses, Normal Brain function Exclusion Criteria: Age beyound 12 month, Brain abnormality, Loss of sensory functions Malignancies

Ages Eligible for Study
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Minimum Age: 5 Months
Maximum Age: 12 Months

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: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| No Intervention: Control<br>A group of 10 patients only will be subjected to electro-myogram test every 3 month and then follow up for their survival time without any cell therapy intervention. | |
| Experimental: Adipose derived Mesenchymal Stem cell<br>A group of 10 patients will be take stem cells intra-thecally Dose: 1 million cells/kg for three times Intervals: Every 3 weeks. | Biological: Adipose derived mesenchymal stem cell<br>* Allogeneic Adipose derived Mesenchymal Stem cell transplant<br>* Other names: Cell Therapy;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in action potential of muscles on ElectroMyoGram (EMG) test | Measure the electrical activity of muscles by Electromyography | Change from Baseline of intervention at 3 month | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Changes in Motility on Modified Barthel Index Score | Measure any phenotypic changes in patients motion by direct Observation on Modified Barthel Index Score | Change from Baseline of intervention at 1 year |