Clinical Trial on The Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis (Extension CMN/ELA)
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- Procedure: Intrathecal infusion of autologous bone marrow stem cellsProcedure: Intrathecal infusion of placebo (saline solution).Procedure: Laminectomy and bone marrow stem cells transplantation
- Registration Number
- NCT01254539
- Lead Sponsor
- Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
- 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.
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 63
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Intrathecal infusion of autologous bone marrow stem cells 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. Intrathecal infusion of placebo (saline solution). Intrathecal infusion of placebo (saline solution). Patients were infused 2 ml of saline solution Autologous bone marrow stem cells intraspinal transplantation Laminectomy and bone marrow stem cells transplantation T3-T4 laminectomy and bone marrow ficoll separated mononuclear autologous cells intraspinal transplantation
- Primary Outcome Measures
Name Time Method Forced vital capacity Every 3 months
- Secondary Outcome Measures
Name Time Method 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 Psychological variables: Health Questionnaire (EuroQol-5D), The Profile of Mood States (POMS) Every 3 months Neurological variables: The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALS-FRS), Medical Research Council (MRC) and Norris scales Every 3 months
Trial Locations
- Locations (1)
Hospital Universitario Virgen de la Arrixaca
🇪🇸El Palmar, Murcia, Spain