The Clinical Trial on the Use of Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis

Study Overview
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Brief Summary
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Patients with Amyotrophic Lateral Sclerosis (ALS) typically endure a progressive paralysis due to the continued loss of motoneurons that leads them to death in less than 5 years. No treatment has changed its natural history. Intrathecal injection of umbilical cord mesenchymal stem cells can secret trophic factors that keep the motorneurons functional. The investigators have designed a phase I/II clinical trial to check the feasibility of this approach in humans.

Detailed Description
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A total of 30 diagnosed ALS patients. The patients would got these symptoms such as gait difficulty and tremor, hand incoordination or speech difficulties.

Official Title
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The Clinical Study on the Use of Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Procedure: stem cell transplantation


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnose established following the World Federation of Neurology criteria More than 6 and less than 36 months of evolution of the disease Medullar onset of the disease More than 20 and less than 65 years old Forced Vital Capacity equal or superior to 50% Total time of oxygen saturation <90% inferior to 2% of the sleeping time Signed informed consent Exclusion Criteria: Neurological or psychiatric concomitant disease Need of parenteral or enteral nutrition through percutaneous endoscopic gastrostomy or nasogastric tube Concomitant systemic disease Treatment with corticosteroids, immunoglobulins or immunosuppressors during the last 12 months Inclusion in other clinical trials Unability to understand the informed consent

Ages Eligible for Study
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Minimum Age: 20 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: stem cell transplantation<br>After stem cell prepared, the patients accepted 4 times stem cell transplantations through lumbar puncture, the time is 3-5days between two treatments. The patient would have to be in the bed at least 6 hours and removed the pillow. | Procedure: stem cell transplantation<br>* after stem cell prepared, the patients accepted 4 times stem cell transplantations through lumbar puncture, the time is 3-5days between two treatments. The patient would have to be in the bed at least 6 hours and removed the pillow.<br>* Other names: the stem cell treatment of ALS;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Nerve functional evaluation | Medical Research Council scale：To determine the strength of bilateral little finger abductor muscle and anterior tibial. To evaluate the situation of upper motor neuron impairment through the Ellis reflex scale The severity evaluation: NorrisALS score and ALS functional rating scale, ALSFRS Speed of disease progression by the following formula：Progress rate = (40-ALSFRS score) / course Questionnaires related to sleep, depression (Beck Depression Inventory) (BDI) and memory (Mini Mental State Examination) | within one week before,1month,6months,12months and 24months after transplantation | 
| Forced vital capacity | vital capacity(VC）、forced vital capacity( FVC）、forced expiratory volune(FEV1）、FEV1/FVC、maximal voluntary ventilation(MVV）、peak expiratory flow(PEF） | within one week before,1month,6months,12months and 24months after transplantation | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Blood test | white blood cell、neutrophilic granulocyte、leukomonocyte；glutamic pyruvic transaminase(GPT)、glutamic oxalacetic transaminase、lactate dehydrogenase(LDH)、;hydroxybutyrate dehydrogenase(HBDH)、phosphocreatine kinase(CK)；acidum uricum(UA)、creatinine(Cr)、α1- microglobulin、β2- microglobulin；lymphotoxin(LCT). Tumor markers , Lymphocytes classification, cholesterol total、Triglyceride、low density lipoprotein、Glycosylated serum protein glycosylated hemoglobin、Islet function，Na+、K+ | within one week before,1month,6months,12months and 24months after transplantation | 
| Urinal test | proteinum、akaryocyte、α1- microglobulin、β2- microglobulin. CSF test:：IgA, IgG quantitation | within one week before,1month,6months,12months and 24months after transplantation | 
| Electrophysiology examination | motor evoked potential(MEP)、nerve conduction and electromyologram(EMG) | within one week before,1month,6months,12months and 24months after transplantation | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ALS, stem cell