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Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients

NCT02855112 · Status: UNKNOWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2016-08-04

No results posted yet for this study

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.

Conditions

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

Interventions

BIOLOGICAL

Adipose derived mesenchymal stem cell

Allogeneic Adipose derived Mesenchymal Stem cell transplant

Sponsors & Collaborators

  • Tehran University of Medical Sciences

    lead OTHER

Principal Investigators

  • Mahmoode Reza Ashrafi, MD · Children's Medical Hospital, Tehran University of Medical Sciences

  • Amir Ali Hamidieh, MD · Hematology-Oncology & Stem cell Transplant Research center, Tehran University of Medical Sciences

  • Rashin Mohseni, PhD · School of Advanced Technologies in Medicine, Tehran University of Medical Sciences

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Months
Max Age
12 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-06-30
Primary Completion
2016-12-31
Completion
2017-07-31

Countries

  • Iran

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02855112 on ClinicalTrials.gov