Clinical trial NCT04849065: Clinical Trial on the Use of Cell Therapy in the Treatment of Patients With Amyotrophic Lateral Sclerosis
Official title: Clinical Trial in Phase II of Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis.
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: ALS (Amyotrophic Lateral Sclerosis)
Lead sponsor: Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
Sponsor class: OTHER
First posted: 2021-04-19
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Phase II, prospective, multicenter, randomized, parallel, double-blind, placebo-controlled clinical trial
Primary purpose: TREATMENT

Brief summary: 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

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of ALS defined or probable according to the criteria established by the World Federation of Neurology
* Age between 18 and 70 years.
* Patient who offers sufficient guarantees of adherence to the protocol.
* Neurophysiological data confirming lower motor neuron involvement at the lumbar and cervical level.
* Assessment of the motor deficit in the dorsal flexion of both feet (between 3 and 5 points on the MRC scale).

Exclusion Criteria:

* Mellitus diabetes.
* Other diseases that may be associated with polyneuropathies.
* Previous history of cerebral stroke.
* Previous pathology of the peripheral nervous system that affected one or both lower or upper limbs, with or without clinically evident neurological sequelae.
* Pregnant or actively breastfeeding patients
* Patients physiologically capable of becoming pregnant, unless they are using a reliable contraceptive method (Annex III)
* Patients with cardiac, renal, hepatic, systemic, immune disease that may influence the survival of the patient during the test.
* Positive serology for hepatitis B, hepatitis C or HIV.
* Clinical and anesthesiological criteria that contraindicate either sedation or the extraction of BM itself (Alteration of the coagulation system or anticoagulated patient with inability to withdraw anticoagulation, hemodynamic instability, skin alteration in the puncture site, etc.)
* Inclusion in other clinical trials in the last 6 months.
* Inability to understand informed consent.

Primary outcomes:
measure: Rate of serious and non-serious adverse events related to the use of bone marrow mononuclear cells in patients with Amyotrophic Lateral Sclerosis
timeFrame: 48 months from baseline
measure: D50 index obtained from stimulus intensity curves
timeFrame: 48 months from baseline
Secondary outcomes:
measure: Motor unit number index" (MUNIX)
timeFrame: 48 months from baseline
measure: Motor unit size index" (MUSIX)
timeFrame: 48 months from baseline
measure: Fiber density (FD)
timeFrame: 48 months from baseline
measure: PAMC amplitude: mV
timeFrame: 48 months from baseline
measure: PAMC area: mV / ms
timeFrame: 48 months from baseline
measure: Maximum force developed in an isometric contraction of the muscles
timeFrame: 48 months from baseline
measure: Fuerza muscular (MRC Medical Research Council) score
timeFrame: 48 months from baseline
Other outcomes:
Locations:
city: Murcia
country: Spain
facility: Hospital Clinico Universitario Virgen de la Arrixaca
Study officials:
References: