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

• ClinicalTrials.gov Identifier: NCT00855400
• Recruitment Status: Completed
• First Posted: March 4, 2009
• Last Update Posted: December 2, 2010
• Sponsor: Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
• Collaborators: Carlos III Health Institute; Hospital Universitario Virgen de la Arrixaca; Hospital General Universitario Morales Meseguer; Diógenes Foundation
• Information provided by: Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia

Tracking Information

• First Submitted Date: March 3, 2009
• First Posted Date: March 4, 2009
• Last Update Posted Date: December 2, 2010
• Study Start Date: February 2007
• Actual Primary Completion Date: October 2009 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: March 3, 2009): Forced vital capacity [ Time Frame: Every 3 months ]
• Original Primary Outcome Measures: Same as current
• Change History: Complete list of historical versions of study NCT00855400 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures (submitted: March 3, 2009)

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

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Clinical Trial on the Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis
• Official Title: Phase I/II Clinical Trial on the Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis
• 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.
• 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 Type: Interventional
• Study Phase: Phase 1; Phase 2
• Study Design: Allocation: Non-Randomized; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Amyotrophic Lateral Sclerosis

Intervention

• 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.

Study Arms

Experimental: Transplant

T3-T4 laminectomy and bone marrow ficoll separated mononuclear autologous cells intraspinal transplantation

Interventions:

• Procedure: Laminectomy and bone marrow stem cells transplantation
• Procedure: Autologous bone marrow cells collection

• Publications *: 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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 3, 2009): 11
• Original Actual Enrollment: Same as current
• Actual Study Completion Date: February 2010
• Actual Primary Completion Date: October 2009 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 20 Years to 65 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Spain

Administrative Information

• NCT Number: NCT00855400
• Other Study ID Numbers: CMN/ELA; EudraCT number: 2006-003096-12; ISCIII: EC07/90762
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Javier Júdez Gutiérrez, Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
• Study Sponsor: Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia

Collaborators

• Carlos III Health Institute
• Hospital Universitario Virgen de la Arrixaca
• Hospital General Universitario Morales Meseguer
• Diógenes Foundation

Investigators

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

• PRS Account: Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
• Verification Date: December 2010