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

NCT01254539 | Completed Phase 1 DMC

• 3 other identifiers: Extension CMN/ELA2006-003096-12EC07/90762
• Study Type: interventional
• Target: 63
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Forced vital capacity

  Every 3 months

Secondary Outcomes (6)

• Neurological variables: The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALS-FRS), Medical Research Council (MRC) and Norris scales

  Every 3 months

• Absence of adverse events

  Every week / month depending on the study phase

• Neurophysiological variables: Electromyography, polysomnography, evoked potentials

  Every 3 months

• Neuroradiological variables: Spinal Magnetic Resonance Imaging (MRI)

  Every 3 months

• Respiratory variables: Maximal inspiratory pressure (PIM), Maximal expiratory pressure (PEM), sniff nasal, oxymetry.

  Every 3 months

Study Arms (3)

Autologous bone marrow stem cells intraspinal transplantation

EXPERIMENTAL

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

Procedure: Laminectomy and bone marrow stem cells transplantation

Intrathecal infusion of autologous bone marrow stem cells

EXPERIMENTAL

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

Intrathecal infusion of placebo (saline solution).

PLACEBO COMPARATOR

Patients were infused 2 ml of saline solution

Procedure: Intrathecal infusion of placebo (saline solution).

Interventions

Laminectomy and bone marrow stem cells transplantation PROCEDURE

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

Autologous bone marrow stem cells intraspinal transplantation

Intrathecal infusion of autologous bone marrow stem cells PROCEDURE

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.

Intrathecal infusion of autologous bone marrow stem cells

Intrathecal infusion of placebo (saline solution). PROCEDURE

Patients were infused 2 ml of saline solution

Intrathecal infusion of placebo (saline solution).

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• Unability to understand the informed consent

Sponsors & Collaborators

• Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia: lead
• Instituto de Salud Carlos III: collaborator
• Hospital Universitario Virgen de la Arrixaca: collaborator
• Hospital General Universitario Morales Meseguer: collaborator
• Diógenes Foundation: collaborator

Study Sites (1)

Hospital Universitario Virgen de la Arrixaca

El Palmar, Murcia, Spain

Location

Related Links

• Spanish Cell Therapy Network
• Amyotrophic Lateral Sclerosis

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Laminectomy; Saline Solution

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Orthopedic Procedures; Therapeutics; Decompression, Surgical; Surgical Procedures, Operative; Neurosurgical Procedures; Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Study Officials

• Jose María Moraleda Jiménez, M.D. PhD.

  Hospital Universitario Virgen de la Arrixaca

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 3, 2010
• First Posted: December 6, 2010
• Study Start: October 1, 2010
• Primary Completion: November 20, 2015
• Study Completion: November 20, 2015
• Last Updated: March 30, 2017

Record last verified: 2015-11

Locations

ES (1)