Clinical trial NCT02855112: Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients
Official title: The Effectiveness of Allogeneic Adipose Derived Mesenchymal Stem Cells (ADMSCs) in the Phenotypic Changes of Werdnig Hoffman Patients
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman]
Lead sponsor: Tehran University of Medical Sciences
Sponsor class: OTHER
First posted: 2016-08-04
Enrollment: 10
Sex: ALL
Minimum age (years): 5
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Changes in action potential of muscles on ElectroMyoGram (EMG) test
timeFrame: Change from Baseline of intervention at 3 month
Secondary outcomes:
measure: Changes in Motility on Modified Barthel Index Score
timeFrame: Change from Baseline of intervention at 1 year
Other outcomes:
measure: Change in overall survival (Mortality)
timeFrame: 2 Years
Locations:
city: Tehran
country: Iran, Islamic Republic of
facility: Children's Medical Center
Study officials:
affiliation: Children's Medical Hospital, Tehran University of Medical Sciences
name: Mahmoode Reza Ashrafi, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Hematology-Oncology & Stem cell Transplant Research center, Tehran University of Medical Sciences
name: Amir Ali Hamidieh, MD
role: STUDY_CHAIR
affiliation: School of Advanced Technologies in Medicine, Tehran University of Medical Sciences
name: Rashin Mohseni, PhD
role: STUDY_DIRECTOR
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