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

ALS (Amyotrophic Lateral Sclerosis) Clinical Trial

— TCIM-ELAII  

Official title:

Clinical Trial in Phase II of Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis.

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT04849065
• Other study ID #: IMB-TCIM/ELAII-2019-01
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: May 1, 2021
• Est. completion date: September 1, 2023

Study information

• Verified date: April 2021
• Source: Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
• Contact: Miguel Blanquer Blanquer, MD
• Phone: 968 36 95 00
• Email: miguelblanquer[@]um.es
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 100
• Est. completion date: September 1, 2023
• Est. primary completion date: May 1, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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.

Related Conditions & MeSH terms

• ALS (Amyotrophic Lateral Sclerosis)
• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• 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)
• Placebo / Saline
- Intramuscular infusion of saline solution (placebo) in the TA muscle of the contralateral lower limb (control side)

Locations

Country	Name	City	State
Spain	Hospital Clinico Universitario Virgen de la Arrixaca	Murcia

Sponsors (1)

Lead Sponsor	Collaborator
Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Secondary	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
Secondary	Motor unit size index" (MUSIX)	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
Secondary	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
Secondary	PAMC amplitude: mV	Compound muscle action potential (CMAP)	48 months from baseline
Secondary	PAMC area: mV / ms	Compound muscle action potential (CMAP)	48 months from baseline
Secondary	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
Secondary	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