Overview

This trial is active, not recruiting.

Condition amyotrophic lateral sclerosis
Treatments laminectomy and bone marrow stem cells transplantation, intrathecal infusion of autologous bone marrow stem cells, intrathecal infusion of placebo (saline solution).
Phase phase 1/phase 2
Sponsor Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
Collaborator Instituto de Salud Carlos III
Start date October 2010
End date November 2015
Trial size 63 participants
Trial identifier NCT01254539, 2006-003096-12, EC07/90762, Extension CMN/ELA

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, investigator)
Primary purpose treatment
Arm
(Experimental)
T3-T4 laminectomy and bone marrow ficoll separated mononuclear autologous cells intraspinal transplantation
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)
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
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)
Patients were infused 2 ml of saline solution
intrathecal infusion of placebo (saline solution).
Patients were infused 2 ml of saline solution

Primary Outcomes

Measure
Forced vital capacity
time frame: Every 3 months

Secondary Outcomes

Measure
Neurological variables: The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALS-FRS), Medical Research Council (MRC) and Norris scales
time frame: Every 3 months
Absence of adverse events
time frame: Every week / month depending on the study phase
Neurophysiological variables: Electromyography, polysomnography, evoked potentials
time frame: Every 3 months
Neuroradiological variables: Spinal Magnetic Resonance Imaging (MRI)
time frame: Every 3 months
Respiratory variables: Maximal inspiratory pressure (PIM), Maximal expiratory pressure (PEM), sniff nasal, oxymetry.
time frame: Every 3 months
Psychological variables: Health Questionnaire (EuroQol-5D), The Profile of Mood States (POMS)
time frame: Every 3 months

Eligibility Criteria

Male or female participants from 18 years up to 70 years old.

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

Additional Information

Official title Phase I/II Clinical Trial on The Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis (Extension CMN/ELA)
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.
Trial information was received from ClinicalTrials.gov and was last updated in March 2015.
Information provided to ClinicalTrials.gov by Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia.