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Our working hypothesis is that the injection of autologous bone marrow mononuclear cells (BMNC) has a positive effect on the natural loss of motor units and on the increase in the size of the motor unit that occurs in patients with ALS during the evolution of the disease
Our working hypothesis is that the injection of autologous bone marrow mononuclear cells (BMNC) has a positive effect on the natural loss of motor units and on the increase in the size of the motor unit that occurs in patients with ALS during the evolution of the disease.
This hypothesis is based on experimental work done in animal models of ALS and the results of our previous phase I clinical trial. In this clinical trial (TCIM / ALS, ClinicalTrials.gov Identifier: NCT02286011) we studied with electrophysiological techniques (in a small number of patients with ALS) the effects of intramuscular injection of a single dose of BMSC in the tibialis anterioris (TA) muscle. . The results indicate that this is a safe procedure and show the presence of a positive and apparently transitory effect on the size and number of motor units of the TA muscle.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MNC (Mononuclear cells) | Experimental | MNC (Mononuclear cells) (patients in which stem cells will be injected into the two muscles on one side and placebo -vehicle- in the two contralateral muscles). This group would consist of 74 patients. |
|
| Saline | Placebo Comparator | (patients in which placebo -vehicle- will be injected into both muscles on both sides). This group would consist of 26 patients. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MNC (Mononuclear cells) | Drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| Rate of serious and non-serious adverse events related to the use of bone marrow mononuclear cells in patients with Amyotrophic Lateral Sclerosis | 48 months from baseline | |
| D50 index obtained from stimulus intensity curves | This parameter quantifies the number and size of the functional motor units of a given muscle. | 48 months from baseline |
| Measure | Description | Time Frame |
|---|---|---|
| Motor unit number index" (MUNIX) | The unit amplitude (or area) of the individual motor units are calculated from the amplitude variations (or area) of the muscle action potential obtained in response to stimuli from a fixed intensity | 48 months from baseline |
| Motor unit size index" (MUSIX) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Miguel Blanquer Blanquer, MD | Contact | 968 36 95 00 | miguelblanquer@um.es |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hospital Clinico Universitario Virgen de la Arrixaca | Murcia | 30003 | Spain |
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Phase II, prospective, multicenter, randomized, parallel, double-blind, placebo-controlled clinical trial
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double-blind
| Placebo / Saline | Drug | - Intramuscular infusion of saline solution (placebo) in the TA muscle of the contralateral lower limb (control side) |
|
The unit amplitude (or area) of the individual motor units are calculated from the amplitude variations (or area) of the muscle action potential obtained in response to stimuli from a fixed intensity |
| 48 months from baseline |
| Fiber density (FD) | Quantifies the average number of muscle fibers per motor unit. It is obtained from single fiber recordings made with electrodes. The average number of motor unit potentials is calculated in 20 different positions in the muscle. | 48 months from baseline |
| PAMC amplitude: mV | Compound muscle action potential (CMAP) | 48 months from baseline |
| PAMC area: mV / ms | Compound muscle action potential (CMAP) | 48 months from baseline |
| Maximum force developed in an isometric contraction of the muscles | The measurement will be made with a dynamometer during dorsiflexion of the foot (from certain angles) and during abduction of the index finger of the hand, respectively. | 48 months from baseline |
| Fuerza muscular (MRC Medical Research Council) score | Muscle strength data will be collected according to the Medical Research Council scale. Muscle strength is graded on a scale of 0 to 5, with 5 being normal muscle strength and 0 being the absence of muscle contraction. | 48 months from baseline |
| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| D012965 | Sodium Chloride |
| ID | Term |
|---|---|
| D002712 | Chlorides |
| D006851 | Hydrochloric Acid |
| D017606 | Chlorine Compounds |
| D007287 | Inorganic Chemicals |
| D017670 | Sodium Compounds |
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