Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman] Clinical Trial
Official title:
The Effectiveness of Allogeneic Adipose Derived Mesenchymal Stem Cells (ADMSCs) in the Phenotypic Changes of Werdnig Hoffman Patients
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.
| Status | Recruiting |
| Enrollment | 10 |
| Est. completion date | July 2017 |
| Est. primary completion date | December 2016 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | 5 Months to 12 Months |
| Eligibility |
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 |
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| Iran, Islamic Republic of | Children's Medical Center | Tehran |
| Lead Sponsor | Collaborator |
|---|---|
| Tehran University of Medical Sciences |
Iran, Islamic Republic of,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Other | Change in overall survival (Mortality) | The length of survival after intervention measured by direct observation | 2 Years | No |
| Primary | 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 | Yes |
| Secondary | 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 | No |