Clinical Trial on the Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis

Study Overview
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Brief Summary
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The purpose of this clinical trial is to asses the feasibility and the security of the intraspinal infusion of autologous bone marrow stem cells for the treatment of Amyotrophic Lateral Sclerosis patients.

Detailed Description
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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. Intraspinal injections of bone marrow mononuclear cells (MNC) have been able to ameliorate the course of ALS in murine models, acting as pumps of trophic factors that keep the motoneurons functional. We have designed a phase I/II clinical trial to check the feasibility of this approach in humans.

Official Title
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Phase I/II Clinical Trial on the Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Procedure: Laminectomy and bone marrow stem cells transplantation
* Procedure: Autologous bone marrow cells collection


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: Transplant<br>T3-T4 laminectomy and bone marrow ficoll separated mononuclear autologous cells intraspinal transplantation | Procedure: Laminectomy and bone marrow stem cells transplantation<br>* T3-T4 laminectomy and bone marrow ficoll separated mononuclear autologous cells intraspinal transplantation<br>Procedure: Autologous bone marrow cells collection<br>* Autologous bone marrow cells collection under sedation. Sixty mL are obtained and processed through a ficoll gradient.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Forced vital capacity |  | Every 3 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| ALS-FRS, MRC and Norris scales |  | Every 3 months | 
| Absence of adverse events |  | Every week / month depending on the study phase | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Amyotrophic Lateral Sclerosis, Bone marrow, Stem cells, Autologous, Safety