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

This study is currently recruiting participants.
Verified December 2010 by Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
Sponsor:
Collaborators:
Instituto de Salud Carlos III
Hospital Universitario Virgen de la Arrixaca
Hospital General Universitario Morales Meseguer
Fundación Diógenes
Information provided by:
Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
ClinicalTrials.gov Identifier:
NCT01254539
First received: December 3, 2010
Last updated: December 7, 2010
Last verified: December 2010
History of Changes
  Purpose

The purpose of this clinical trial is to assess the feasibility and the security of the intraspinal and intrathecal infusion of autologous bone marrow stem cells for the treatment of Amyotrophic Lateral Sclerosis patients.


Condition Intervention Phase
Amyotrophic Lateral Sclerosis
 Procedure: Laminectomy and bone marrow stem cells transplantation
Procedure: Intrathecal infusion of autologous bone marrow stem cells
Procedure: Intrathecal infusion of placebo (saline solution).
 Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: Phase I/II Clinical Trial on The Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis (Extension CMN/ELA)

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia:

Primary Outcome Measures:
  • Forced vital capacity [ Time Frame: Every 3 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Neurological variables: The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALS-FRS), Medical Research Council (MRC) and Norris scales [ Time Frame: Every 3 months ] [ Designated as safety issue: No ]
  • Absence of adverse events [ Time Frame: Every week / month depending on the study phase ] [ Designated as safety issue: Yes ]
  • Neurophysiological variables: Electromyography, polysomnography, evoked potentials [ Time Frame: Every 3 months ] [ Designated as safety issue: No ]
  • Neuroradiological variables: Spinal Magnetic Resonance Imaging (MRI) [ Time Frame: Every 3 months ] [ Designated as safety issue: No ]
  • Respiratory variables: Maximal inspiratory pressure (PIM), Maximal expiratory pressure (PEM), sniff nasal, oxymetry. [ Time Frame: Every 3 months ] [ Designated as safety issue: No ]
  • Psychological variables: Health Questionnaire (EuroQol-5D), The Profile of Mood States (POMS) [ Time Frame: Every 3 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 63
Study Start Date: October 2010
Estimated Study Completion Date: November 2014
Estimated Primary Completion Date: October 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Autologous bone marrow stem cells intraspinal transplantation
T3-T4 laminectomy and bone marrow ficoll separated mononuclear autologous cells intraspinal transplantation
 Procedure: Laminectomy and bone marrow stem cells transplantation

Autologous bone marrow cells collection under sedation. Sixty ml are obtained and processed through a ficoll gradient.

T3-T4 laminectomy and bone marrow ficoll separated mononuclear autologous cells intraspinal transplantation
Experimental: Intrathecal infusion of autologous bone marrow stem cells
Patients were drawn 2 ml of cerebrospinal fluid and infused 2 ml (two 1 ml syringes) of Autologous Stem Cells.
 Procedure: Intrathecal infusion of autologous bone marrow stem cells

Autologous bone marrow cells collection under sedation. Sixty ml are obtained and processed through a ficoll gradient.

Patients were drawn 2 ml of cerebrospinal fluid and infused 2 ml (two 1 ml syringes) of Autologous Stem Cells.
Placebo Comparator: Intrathecal infusion of placebo (saline solution).
Patients were infused 2 ml of saline solution
 Procedure: Intrathecal infusion of placebo (saline solution).
Patients were infused 2 ml of saline solution

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. Moreover, the clinical trial (Study NCT00855400 on www.ClinicalTrials.gov) conducted by our research group to determine the safety and efficacy of Autologous Stem Cell transplantation in Amyotrophic Lateral Sclerosis in humans, found that this procedure is feasible and safe. Continuing with that study, we have designed a phase I/II clinical trial to check the feasibility of the intraspinal and intrathecal infusion of autologous bone marrow stem cells.

  Eligibility

Ages Eligible for Study:   18 Years to 70 Years
Genders Eligible for Study:   Both
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 18 and less than 70 years old
  • Forced Vital Capacity ≥ 50%
  • Total time of oxygen saturation <90% inferior to 5% 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01254539

Contacts
Contact: Paqui Iniesta Martínez iniesmar@yahoo.es

Locations
Spain
Hospital Universitario Virgen de la Arrixaca Recruiting
El Palmar, Murcia, Spain
Contact: Paqui Iniesta Martínez            
Principal Investigator: Jose María Moraleda Jiménez, M.D. PhD.            
Sponsors and Collaborators
Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
Instituto de Salud Carlos III
Hospital Universitario Virgen de la Arrixaca
Hospital General Universitario Morales Meseguer
Fundación Diógenes
Investigators
Study Director: Jose María Moraleda Jiménez, M.D. PhD. Hospital Universitario Virgen de la Arrixaca
  More Information

Additional Information:
Spanish Cell Therapy Network  This link exits the ClinicalTrials.gov site
Amyotrophic Lateral Sclerosis  This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party: Javier Júdez Gutiérrez, Fundación para la Formación e Investigación Sanitarias de la Región de Murcia
ClinicalTrials.gov Identifier: NCT01254539     History of Changes
Other Study ID Numbers: Extension CMN/ELA, 2006-003096-12, EC07/90762
Study First Received: December 3, 2010
Last Updated: December 7, 2010
Health Authority: Spain: Spanish Agency of Medicines

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
Spinal Cord Diseases
Central Nervous System Diseases
Nervous System Diseases
 Neurodegenerative Diseases
TDP-43 Proteinopathies
Neuromuscular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on June 17, 2013