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

Primary Purpose

ALS (Amyotrophic Lateral Sclerosis)

Status

Unknown status

Phase

Phase 2

Locations

Spain

Study Type

Interventional

Intervention

MNC (Mononuclear cells)

Placebo / Saline

About this trial

This is an interventional treatment trial for ALS (Amyotrophic Lateral Sclerosis)

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept 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.

Sites / Locations

Hospital Clinico Universitario Virgen de la Arrixaca

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

MNC (Mononuclear cells)

Saline

Arm 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.

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

Outcomes

Primary Outcome Measures

Rate of serious and non-serious adverse events related to the use of bone marrow mononuclear cells in patients with Amyotrophic Lateral Sclerosis

D50 index obtained from stimulus intensity curves

This parameter quantifies the number and size of the functional motor units of a given muscle.

Secondary Outcome Measures

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

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

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.

PAMC amplitude: mV

Compound muscle action potential (CMAP)

PAMC area: mV / ms

Compound muscle action potential (CMAP)

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.

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.

Full Information

NCT ID

NCT04849065

First Posted

April 15, 2021

Last Updated

April 15, 2021

Sponsor

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

1. Study Identification

Unique Protocol Identification Number

NCT04849065

Brief Title

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

Acronym

TCIM-ELAII

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

2. Study Status

Record Verification Date

April 2021

Overall Recruitment Status

Unknown status

Study Start Date

May 1, 2021 (Anticipated)

Primary Completion Date

May 1, 2023 (Anticipated)

Study Completion Date

September 1, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

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

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

ALS (Amyotrophic Lateral Sclerosis)

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Model Description

Phase II, prospective, multicenter, randomized, parallel, double-blind, placebo-controlled clinical trial

Masking

ParticipantInvestigator

Masking Description

double-blind

Allocation

Randomized

Enrollment

100 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

MNC (Mononuclear cells)

Arm Type

Experimental

Arm Description

Arm Title

Saline

Arm Type

Placebo Comparator

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

MNC (Mononuclear cells)

Intervention Description

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

Intervention Name(s)

Placebo / Saline

Intervention Description

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

Primary Outcome Measure Information:

Title

Rate of serious and non-serious adverse events related to the use of bone marrow mononuclear cells in patients with Amyotrophic Lateral Sclerosis

Time Frame

48 months from baseline

Title

D50 index obtained from stimulus intensity curves

Description

This parameter quantifies the number and size of the functional motor units of a given muscle.

Time Frame

48 months from baseline

Secondary Outcome Measure Information:

Title

Motor unit number index" (MUNIX)

Description

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

Time Frame

48 months from baseline

Title

Motor unit size index" (MUSIX)

Description

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

Time Frame

48 months from baseline

Title

Fiber density (FD)

Description

Time Frame

48 months from baseline

Title

PAMC amplitude: mV

Description

Compound muscle action potential (CMAP)

Time Frame

48 months from baseline

Title

PAMC area: mV / ms

Description

Compound muscle action potential (CMAP)

Time Frame

48 months from baseline

Title

Maximum force developed in an isometric contraction of the muscles

Description

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.

Time Frame

48 months from baseline

Title

Fuerza muscular (MRC Medical Research Council) score

Description

Time Frame

48 months from baseline

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

70 Years

Accepts Healthy Volunteers

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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Miguel Blanquer Blanquer, MD

Phone

968 36 95 00

miguelblanquer@um.es

Facility Information:

Facility Name

Hospital Clinico Universitario Virgen de la Arrixaca

City

Murcia

ZIP/Postal Code

30003

Country

Spain

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Miguel Blanquer Blanquer

Phone

968359767

First Name & Middle Initial & Last Name & Degree

Miguel Blanquer Blanquer, MD

First Name & Middle Initial & Last Name & Degree

Carmen Díaz Marín, MD

First Name & Middle Initial & Last Name & Degree

Teresa Gavilá Lattur, MD

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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