Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients

Tehran University of Medical Sciences

Status and phase: Unknown; Phase 2; Phase 1

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

Treatments: Biological: Adipose derived mesenchymal stem cell

Study type: Interventional

Funder types: Other

Identifiers

NCT02855112

94-01-87-28524

Details and patient eligibility

About

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.

Enrollment: 10 estimated patients

Sex: All

Ages: 5 to 12 months old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 2 patient groups

Control

No Intervention group

Description:

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.

Adipose derived Mesenchymal Stem cell

Experimental group

Description:

A group of 10 patients will be take stem cells intra-thecally Dose: 1 million cells/kg for three times Intervals: Every 3 weeks.

Treatment:

Biological: Adipose derived mesenchymal stem cell