Clinical Trial on the Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis (CMN/ELA)

Study Description

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Amyotrophic Lateral Sclerosis	Procedure: Laminectomy and bone marrow stem cells transplantation; Procedure: Autologous bone marrow cells collection	Phase 1; Phase 2

Detailed Description:

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.

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	11 participants
Allocation:	Non-Randomized
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
Official Title:	Phase I/II Clinical Trial on the Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis
Study Start Date :	February 2007
Actual Primary Completion Date :	October 2009
Actual Study Completion Date :	February 2010

Arms and Interventions

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Arm	Intervention/treatment
Experimental: Transplant; T3-T4 laminectomy and bone marrow ficoll separated mononuclear autologous cells intraspinal transplantation	Procedure: Laminectomy and bone marrow stem cells transplantation; T3-T4 laminectomy and bone marrow ficoll separated mononuclear autologous cells intraspinal transplantation Procedure: Autologous bone marrow cells collection; Autologous bone marrow cells collection under sedation. Sixty mL are obtained and processed through a ficoll gradient.

Outcome Measures

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Primary Outcome Measures :

1. Forced vital capacity [ Time Frame: Every 3 months ]

Secondary Outcome Measures :

1. ALS-FRS, MRC and Norris scales [ Time Frame: Every 3 months ]
2. Absence of adverse events [ Time Frame: Every week / month depending on the study phase ]

Eligibility Criteria

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Ages Eligible for Study:	20 Years to 65 Years (Adult)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

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Locations

Spain
Hospital Universitario Virgen de la Arrixaca
El Palmar, Murcia, Spain, 30120

Sponsors and Collaborators

Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia

Carlos III Health Institute

Hospital Universitario Virgen de la Arrixaca

Hospital General Universitario Morales Meseguer

Fundación Diógenes

Investigators

Study Director:	Jose Maria Moraleda Jiménez, M.D. Ph.D.	Hospital Universitario Virgen de la Arrixaca

More Information

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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Blanquer M, Moraleda JM, Iniesta F, Gómez-Espuch J, Meca-Lallana J, Villaverde R, Pérez-Espejo MÁ, Ruíz-López FJ, García Santos JM, Bleda P, Izura V, Sáez M, De Mingo P, Vivancos L, Carles R, Jiménez J, Hernández J, Guardiola J, Del Rio ST, Antúnez C, De la Rosa P, Majado MJ, Sánchez-Salinas A, López J, Martínez-Lage JF, Martínez S. Neurotrophic bone marrow cellular nests prevent spinal motoneuron degeneration in amyotrophic lateral sclerosis patients: a pilot safety study. Stem Cells. 2012 Jun;30(6):1277-85. doi: 10.1002/stem.1080.

Responsible Party:	Javier Júdez Gutiérrez, Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
ClinicalTrials.gov Identifier:	NCT00855400 History of Changes
Other Study ID Numbers:	CMN/ELA; EudraCT number: 2006-003096-12; ISCIII: EC07/90762
First Posted:	March 4, 2009
Last Update Posted:	December 2, 2010
Last Verified:	December 2010

Keywords provided by Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia:

Amyotrophic Lateral Sclerosis; Bone marrow; Stem cells; Autologous; Safety

Additional relevant MeSH terms:

Amyotrophic Lateral Sclerosis; Sclerosis; Motor Neuron Disease; Pathologic Processes; Neurodegenerative Diseases; Nervous System Diseases	Neuromuscular Diseases; Spinal Cord Diseases; Central Nervous System Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases