Clinical Trial on the Use of Cell Therapy in the Treatment of Patients With Amyotrophic Lateral Sclerosis (TCIM-ELAII)

Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia

Status and phase: Unknown; Phase 2

Conditions: ALS (Amyotrophic Lateral Sclerosis)

Treatments: Drug: MNC (Mononuclear cells); Drug: Placebo / Saline

Study type: Interventional

Funder types: Other

Identifiers

NCT04849065

IMB-TCIM/ELAII-2019-01

Details and patient eligibility

About

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

Full 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.

Enrollment: 100 estimated patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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100 participants in 2 patient groups, including a placebo group

MNC (Mononuclear cells)

Experimental group

Description:

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.

Treatment:

Drug: MNC (Mononuclear cells)

Drug: Placebo / Saline

Saline

Placebo Comparator group

Description:

(patients in which placebo -vehicle- will be injected into both muscles on both sides). This group would consist of 26 patients.

Treatment:

Drug: Placebo / Saline