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

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ClinicalTrials.gov Identifier: NCT04849065
Recruitment Status : Not yet recruiting
First Posted : April 19, 2021
Last Update Posted : April 19, 2021
Sponsor:
Information provided by (Responsible Party):
Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia

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

Condition or disease Intervention/treatment Phase
ALS (Amyotrophic Lateral Sclerosis) Drug: MNC (Mononuclear cells) Drug: Placebo / Saline Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Phase II, prospective, multicenter, randomized, parallel, double-blind, placebo-controlled clinical trial
Masking: Double (Participant, Investigator)
Masking Description: double-blind
Primary Purpose: Treatment
Official Title: Clinical Trial in Phase II of Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis.
Estimated Study Start Date : May 1, 2021
Estimated Primary Completion Date : May 1, 2023
Estimated Study Completion Date : September 1, 2023


Arm Intervention/treatment
Experimental: 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.

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)

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

Placebo Comparator: Saline
(patients in which placebo -vehicle- will be injected into both muscles on both sides). This group would consist of 26 patients.
Drug: Placebo / Saline
- Intramuscular infusion of saline solution (placebo) in the TA muscle of the contralateral lower limb (control side)




Primary Outcome Measures :
  1. 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 ]
  2. D50 index obtained from stimulus intensity curves [ Time Frame: 48 months from baseline ]
    This parameter quantifies the number and size of the functional motor units of a given muscle.


Secondary Outcome Measures :
  1. Motor unit number index" (MUNIX) [ Time Frame: 48 months from baseline ]
    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

  2. Motor unit size index" (MUSIX) [ Time Frame: 48 months from baseline ]
    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

  3. Fiber density (FD) [ Time Frame: 48 months from baseline ]
    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.

  4. PAMC amplitude: mV [ Time Frame: 48 months from baseline ]
    Compound muscle action potential (CMAP)

  5. PAMC area: mV / ms [ Time Frame: 48 months from baseline ]
    Compound muscle action potential (CMAP)

  6. Maximum force developed in an isometric contraction of the muscles [ Time Frame: 48 months from baseline ]
    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.

  7. Fuerza muscular (MRC Medical Research Council) score [ Time Frame: 48 months from baseline ]
    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.



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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04849065


Contacts
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Contact: Miguel Blanquer Blanquer, MD 968 36 95 00 miguelblanquer@um.es

Locations
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Spain
Hospital Clinico Universitario Virgen de la Arrixaca
Murcia, Spain, 30003
Contact: Miguel Blanquer Blanquer    968359767      
Principal Investigator: Miguel Blanquer Blanquer, MD         
Principal Investigator: Carmen Díaz Marín, MD         
Principal Investigator: Teresa Gavilá Lattur, MD         
Sponsors and Collaborators
Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
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Responsible Party: Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
ClinicalTrials.gov Identifier: NCT04849065    
Other Study ID Numbers: IMB-TCIM/ELAII-2019-01
First Posted: April 19, 2021    Key Record Dates
Last Update Posted: April 19, 2021
Last Verified: April 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases