Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients

The Effectiveness of Allogeneic Adipose Derived Mesenchymal Stem Cells (ADMSCs) in the Phenotypic Changes of Werdnig Hoffman Patients

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.

Study Overview

• Status: Unknown
• Conditions: Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman]
• Intervention / Treatment: Biological: Adipose derived mesenchymal stem cell

• Study Type: Interventional
• Enrollment (Anticipated): 10
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Iran, Islamic Republic of
  • Tehran, Iran, Islamic Republic of, 14194
    • Recruiting
    • Children's Medical Center
    • Contact: Rashin Mohseni, PhD; Phone Number: +989123430627; Email: rashin_mohseni@yahoo.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 11 months to 4 months (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
No Intervention: Control; 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.
Experimental: 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.	Biological: Adipose derived mesenchymal stem cell; Allogeneic Adipose derived Mesenchymal Stem cell transplant; Other Names: Cell Therapy

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in overall survival (Mortality)	The length of survival after intervention measured by direct observation	2 Years

Collaborators and Investigators

• Sponsor: Tehran University of Medical Sciences
• Investigators: Principal Investigator: Mahmoode Reza Ashrafi, MD, Children's Medical Hospital, Tehran University of Medical Sciences; Study Chair: Amir Ali Hamidieh, MD, Hematology-Oncology & Stem cell Transplant Research center, Tehran University of Medical Sciences; Study Director: Rashin Mohseni, PhD, School of Advanced Technologies in Medicine, Tehran University of Medical Sciences

Study record dates

Study Major Dates

• Study Start: June 1, 2015
• Primary Completion (Anticipated): December 1, 2016
• Study Completion (Anticipated): July 1, 2017

Study Registration Dates

• First Submitted: July 22, 2016
• First Submitted That Met QC Criteria: August 1, 2016
• First Posted (Estimate): August 4, 2016

Study Record Updates

• Last Update Posted (Estimate): August 4, 2016
• Last Update Submitted That Met QC Criteria: August 1, 2016
• Last Verified: August 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Genetic Diseases, Inborn; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Pathological Conditions, Anatomical; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Atrophy; Motor Neuron Disease; Muscular Atrophy; Muscular Atrophy, Spinal; Spinal Muscular Atrophies of Childhood
• Other Study ID Numbers: 94-01-87-28524

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided