Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients

Phase 1

• Conditions: Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman]
• Interventions: Biological: Adipose derived mesenchymal stem cell

• Registration Number: NCT02855112
• Lead Sponsor: Tehran University of Medical Sciences

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2015-06
• Study First Submitted: 2016-07-22
• Status Verified: 2016-08
• Study First Submitted QC: 2016-08-01
• Last Update Submitted: 2016-08-01
• Study First Posted: 2016-08-04
• Last Update Posted: 2016-08-04
• Primary Completion: 2016-12
• Study Completion: 2017-07

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 10

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Adipose derived Mesenchymal Stem cell	Adipose derived mesenchymal stem cell	A group of 10 patients will be take stem cells intra-thecally Dose: 1 million cells/kg for three times Intervals: Every 3 weeks.

Primary Outcome Measures

Name	Time	Method
Changes in action potential of muscles on ElectroMyoGram (EMG) test	Change from Baseline of intervention at 3 month	Measure the electrical activity of muscles by Electromyography

Secondary Outcome Measures

Name	Time	Method
Changes in Motility on Modified Barthel Index Score	Change from Baseline of intervention at 1 year	Measure any phenotypic changes in patients motion by direct Observation on Modified Barthel Index Score

Trial Locations

Locations (1)

Children's Medical Center; 🇮🇷 Tehran, Iran, Islamic Republic of