Compassionate Treatment : An Exploratory Clinical Trial to Assess Treatment of Amyotrophic Lateral Sclerosis

Study Overview
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Brief Summary
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The purpose of the study is to determine the safety and possible effectiveness of Autologous Adipose-Tissue Derived Stem Cells treatment in a Amyotrophic Lateral Sclerosis(ALS) patient.

Detailed Description
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This pilot clinical trial is design to see the safety and possible efficiency of ADSCs treatment in a ALS patient. one subjects will be treated (n=1). The patient will receive brain transplantation of ADSCs and combines intravenous infusion ADSCs 4 times. Subjects will be assessed by adverse effects and functional outcomes of clinical: PI max , PE max,and Haloscale respiration, ALS functional rating scale (ALS-FRS) are analyzed at 1 month, 3 months, 6 months 9 months and 12 months. Brain MRI scans will be obtained before and after the treatment. Long term follow-up will be performed at 1, 6 , and 12months for safety reasons. Brain CT for check operation safety before and after brain implantation. Timing of Study: 12months recruitment period after approvals

Official Title
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An Exploratory Clinical Trial to Assess Treatment of Amyotrophic Lateral Sclerosis With Brain Transplants of Autologous Adipose-Tissue Derived Stem Cells (ADSCs)

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Procedure: Autologous Adipose-Tissue Derived Stem Cells


Participation Criteria
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Eligibility Criteria
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Compassionate Treatment The number of participants for a person. The patients (Guo XX) of 28% of lung function assessment (normal lung function was greater than 80%), the daily life of motor function score was 17 points (normal function score of 40 points).

Ages Eligible for Study
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Minimum Age: 35 Years
Maximum Age: 50 Years

Sexes Eligible for Study
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Male

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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Compassionate Treatment<br>ALS patients are received Intravenous and intracerebral implantation of Autologous Adipose-Tissue Derived Stem Cells (ADSCs), Rehabilitation. | Procedure: Autologous Adipose-Tissue Derived Stem Cells<br>* Intravenous and Intracerebral implantation of Autologous Adipose-Tissue Derived Stem Cells Patient will receive brain implantation of approximately 100 -400million Autologous Adipose-Tissue Derived Stem Cells. Patient will receive Intravenous of approximately 100-400 million Autologous Adipose-Tissue Derived Stem Cells twice , before brain implantation, and will receive Intravenous of approximately 100-400 million Autologous Adipose-Tissue Derived Stem Cells twice, after brain implantation .<br>* Other names: ADSCs;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| changes of ALS-FRS score | using ALS-FRS score to assess patient's function after transplantation. | Baseline-Day 28-Day 44-Day 60-6 months-9months-12months | 
| changes of respiratory function | check the changes of respiratory functions after intervention . | Baseline-Day 28-Day 44-Day 60-6 mouths-9mouths-12mouths | 
| changes of neurologic function | check any changes of neurologic functions after intervention . | Baseline-Day 28-Day 44-Day 60-6 mouths-9mouths-12mouths | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| changes of brain structure | check changes of MRI after intervention | Baseline-Day 60- 6 mounths-12 mounths | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Amyotrophic Lateral Sclerosis, Autologous Adipose-Tissue Derived Stem Cells (ADSCs)