Ignite Creation Date:
2024-05-06 @ 8:55 AM
Last Modification Date:
2024-10-26 @ 12:07 PM
Study NCT ID:
NCT02855112
Status:
UNKNOWN
Last Update Posted:
2016-08-04
First Post:
2016-07-22
Brief Title:
Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients
Sponsor:
Tehran University of Medical Sciences
Organization:
Tehran University of Medical Sciences
Study Overview
Official Title:
The Effectiveness of Allogeneic Adipose Derived Mesenchymal Stem Cells ADMSCs in the Phenotypic Changes of Werdnig Hoffman Patients
Status:
UNKNOWN
Status Verified Date:
2016-08
Last Known Status:
RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 deathNo 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 couldnt 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
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None