Intrathecal Transplantation of Mesenchymal Stem Cell in Patients With ALS

This study has been completed.
Information provided by (Responsible Party):
Royan Institute Identifier:
First received: January 16, 2013
Last updated: December 31, 2015
Last verified: May 2012
ALS is a debilitating disease with varied etiology characterized by rapidly progressive weakness, muscle atrophy and fasciculations, muscle spasticity, difficulty speaking (dysarthria), difficulty swallowing (dysphagia), and difficulty breathing (dyspnea). ALS is the most common of the five motor neuron diseases.Riluzole (Rilutek) is the only treatment that has been found to improve survival but only to a modest extent. It lengthens survival by several months, and may have a greater survival benefit for those with a bulbar onset. It also extends the time before a person needs ventilation support.Stem cell transplantation is a new hopeful way to improve the patients conditions and reduce the period of disabilities.

Condition Intervention Phase
Amyotrophic Lateral Sclerosis
Biological: intrathecal injection
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Intrathecal Transplantation of Autologous Bone Marrow Derived Mesenchymal Stem Cell in Patients With ALS

Resource links provided by NLM:

Further study details as provided by Royan Institute:

Primary Outcome Measures:
  • Fever [ Time Frame: 48hours ] [ Designated as safety issue: Yes ]
    Evaluation the rate of fever during 48hour after transplantation

  • Unconscious [ Time Frame: 48hours ] [ Designated as safety issue: Yes ]
    Evaluation the rate of unconsciousness during 48hour after transplantation

  • Vomiting [ Time Frame: 48hours ] [ Designated as safety issue: Yes ]
    Evaluation the nausea and vomiting 48hours after transplantation.

Secondary Outcome Measures:
  • ALS-FRS [ Time Frame: 6months ] [ Designated as safety issue: No ]
    Evaluation the improvement of ALS-FRS during 6months after transplantation.

  • FVC [ Time Frame: 6months ] [ Designated as safety issue: No ]
    Evaluation the improvement of FVC by spirometry during 6months after transplantation

Enrollment: 8
Study Start Date: August 2013
Study Completion Date: December 2014
Primary Completion Date: October 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: stem cell reciepient
The patients with ALS that underwent mesenchymal stem cell transplantation
Biological: intrathecal injection
Intrathecal injection of mesenchymal stem cells in patients with ALS

Detailed Description:
In this study our purpose is to evaluate the safety of intraventricular injection of bone marrow derived mesenchymal stem cell.the patients who are eligible,underwent bone marrow aspiration.after cell separation and preparation,the patients underwent mesenchymal stem cell intraventricular injection by stereotaxis.after injection he will be under observed in ICU to monitor the adverse events(allergic and neurological side effects).patients are followed 1th,3th ,6th and 12 months after injection and each time these parameters are checked:ALS-FRS,EMG-NCV,FVC,side effect check list.

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Age:18-65
  • both gender
  • duration of disease<2 years
  • FVC>40% ALS-FRS>26

Exclusion Criteria:

  • neurological and psychiatric concomitant disease
  • concomitant systemic disease
  • treatment with corticosteroid,Ig,immunosuppressive during 12 months.
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Please refer to this study by its identifier: NCT01771640

Iran, Islamic Republic of
Royan Institute
Tehran, Iran, Islamic Republic of
Sponsors and Collaborators
Royan Institute
Study Chair: Hamid Gourabi, PhD Head of Royan Institute
Study Director: Nasser Aghdami, MD,PhD Head of Royan department of degenerative medicine,Head of Royan celltherapy center
Study Director: Seyed Masoud Nabavi, MD Proffessor assistant of Shahed University
  More Information

Additional Information:
No publications provided

Responsible Party: Royan Institute Identifier: NCT01771640     History of Changes
Other Study ID Numbers: Royan-Nerve-006 
Study First Received: January 16, 2013
Last Updated: December 31, 2015
Health Authority: Iran: Ethics Committee
Iran: Ministry of Health

Keywords provided by Royan Institute:
ALS intrathecal injection bone marrow mesenchymal stem cell

Additional relevant MeSH terms:
Amyotrophic Lateral Sclerosis
Central Nervous System Diseases
Metabolic Diseases
Motor Neuron Disease
Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Diseases
Proteostasis Deficiencies
Spinal Cord Diseases
TDP-43 Proteinopathies processed this record on February 08, 2016