Clinical trial NCT01254539: Clinical Trial on The Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis (Extension CMN/ELA)
Official title: Phase I/II Clinical Trial on The Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis (Extension CMN/ELA)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Keywords: Amyotrophic Lateral Sclerosis
Bone marrow
Stem cells
Autologous
Safety
Lead sponsor: Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
Sponsor class: OTHER
Collaborators: name: Instituto de Salud Carlos III
name: Hospital Universitario Virgen de la Arrixaca
name: Hospital General Universitario Morales Meseguer
name: Diógenes Foundation
First posted: 2010-12-06
Enrollment: 63
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Forced vital capacity
timeFrame: Every 3 months
Secondary outcomes:
measure: Neurological variables: The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALS-FRS), Medical Research Council (MRC) and Norris scales
timeFrame: Every 3 months
measure: Absence of adverse events
timeFrame: Every week / month depending on the study phase
measure: Neurophysiological variables: Electromyography, polysomnography, evoked potentials
timeFrame: Every 3 months
measure: Neuroradiological variables: Spinal Magnetic Resonance Imaging (MRI)
timeFrame: Every 3 months
measure: Respiratory variables: Maximal inspiratory pressure (PIM), Maximal expiratory pressure (PEM), sniff nasal, oxymetry.
timeFrame: Every 3 months
measure: Psychological variables: Health Questionnaire (EuroQol-5D), The Profile of Mood States (POMS)
timeFrame: Every 3 months
Other outcomes:
Locations:
city: El Palmar
country: Spain
facility: Hospital Universitario Virgen de la Arrixaca
state: Murcia
Study officials:
affiliation: Hospital Universitario Virgen de la Arrixaca
name: Jose María Moraleda Jiménez, M.D. PhD.
role: STUDY_DIRECTOR
References: