Phase I/II Clinical Trial on The Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis (Extension CMN/ELA)
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Amyotrophic Lateral Sclerosis
- Sponsor
- Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
- Enrollment
- 63
- Locations
- 1
- Primary Endpoint
- Forced vital capacity
- Status
- Completed
- Last Updated
- 9 years ago
Overview
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.
Investigators
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 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
Outcomes
Primary Outcomes
Forced vital capacity
Time Frame: Every 3 months
Secondary Outcomes
- 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)