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Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients

This study is currently recruiting participants.
Verified August 2016 by Amir Ali Hamidieh, Tehran University of Medical Sciences
Sponsor:
ClinicalTrials.gov Identifier:
NCT02855112
First Posted: August 4, 2016
Last Update Posted: August 4, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Amir Ali Hamidieh, Tehran University of Medical Sciences
  Purpose
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.

Condition Intervention Phase
Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman] Biological: Adipose derived mesenchymal stem cell Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: The Effectiveness of Allogeneic Adipose Derived Mesenchymal Stem Cells (ADMSCs) in the Phenotypic Changes of Werdnig Hoffman Patients

Resource links provided by NLM:


Further study details as provided by Amir Ali Hamidieh, Tehran University of Medical Sciences:

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


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


Other Outcome Measures:
  • Change in overall survival (Mortality) [ Time Frame: 2 Years ]
    The length of survival after intervention measured by direct observation


Estimated Enrollment: 10
Study Start Date: June 2015
Estimated Study Completion Date: July 2017
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Name: Cell Therapy

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Months to 12 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02855112


Contacts
Contact: Rashin Mohseni, PhD +989123230627 rashin_mohseni@yahoo.com

Locations
Iran, Islamic Republic of
Children's Medical Center Recruiting
Tehran, Iran, Islamic Republic of, 14194
Contact: Rashin Mohseni, PhD    +989123430627    rashin_mohseni@yahoo.com   
Sponsors and Collaborators
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
  More Information

Responsible Party: Amir Ali Hamidieh, Head department of Pediatrics Stem cell Transplantation Research center, Tehran University of Medical Sciences
ClinicalTrials.gov Identifier: NCT02855112     History of Changes
Other Study ID Numbers: 94-01-87-28524
First Submitted: July 22, 2016
First Posted: August 4, 2016
Last Update Posted: August 4, 2016
Last Verified: August 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Additional relevant MeSH terms:
Spinal Muscular Atrophies of Childhood
Atrophy
Muscular Atrophy
Muscular Atrophy, Spinal
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn