Clinical Trial on the Use of Cell Therapy in the Treatment of Patients With Amyotrophic Lateral Sclerosis

NCT ID: NCT04849065

Last Updated: 2021-04-19

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 100 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-05-01
• Study Completion Date: 2023-09-01

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.

Conditions

ALS (Amyotrophic Lateral Sclerosis)

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

MNC (Mononuclear cells)

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.

Group Type: EXPERIMENTAL

MNC (Mononuclear cells)

Intervention Type: DRUG

• Intramuscular infusion of autologous BM CMN into the TA muscle of one of the lower limbs (experimental side)
• Intramuscular infusion of saline solution (placebo) in the TA muscle of the contralateral lower limb (control side)

Placebo / Saline

Intervention Type: DRUG

\- Intramuscular infusion of saline solution (placebo) in the TA muscle of the contralateral lower limb (control side)

Saline

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

Group Type: PLACEBO_COMPARATOR

Placebo / Saline

Intervention Type: DRUG

\- Intramuscular infusion of saline solution (placebo) in the TA muscle of the contralateral lower limb (control side)

Interventions

MNC (Mononuclear cells)

• Intramuscular infusion of autologous BM CMN into the TA muscle of one of the lower limbs (experimental side)
• Intramuscular infusion of saline solution (placebo) in the TA muscle of the contralateral lower limb (control side)

Intervention Type: DRUG

Placebo / Saline

\- Intramuscular infusion of saline solution (placebo) in the TA muscle of the contralateral lower limb (control side)

Intervention Type: DRUG

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

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

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Hospital Clinico Universitario Virgen de la Arrixaca

Murcia, , Spain

Countries

Spain

Central Contacts

Miguel Blanquer Blanquer, MD

Role: CONTACT

miguelblanquer@um.es

968 36 95 00

Facility Contacts

Miguel Blanquer Blanquer

Role: primary

968359767

Other Identifiers

IMB-TCIM/ELAII-2019-01

Identifier Type: -

Identifier Source: org_study_id