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

NCT04849065 | Unknown Phase 2

• 1 other identifier: IMB-TCIM/ELAII-2019-01
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 1

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

Conditions

ALS (Amyotrophic Lateral Sclerosis)

Outcome Measures

Primary Outcomes (2)

• 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

• 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 Outcomes (7)

• Motor unit number index" (MUNIX)

  48 months from baseline

• Motor unit size index" (MUSIX)

  48 months from baseline

• Fiber density (FD)

  48 months from baseline

• PAMC amplitude: mV

  48 months from baseline

• PAMC area: mV / ms

  48 months from baseline

Study Arms (2)

MNC (Mononuclear cells)

EXPERIMENTAL

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); Drug: Placebo / Saline

Saline

PLACEBO COMPARATOR

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

Drug: Placebo / Saline

Interventions

MNC (Mononuclear cells) 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)

MNC (Mononuclear cells)

Placebo / Saline DRUG

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

MNC (Mononuclear cells); Saline

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.)
• Inability to understand informed consent.

Sponsors & Collaborators

• Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia: lead

Study Sites (1)

Hospital Clinico Universitario Virgen de la Arrixaca

Murcia, 30003, Spain

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Sodium Chloride

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Central Study Contacts

Miguel Blanquer Blanquer, MD

CONTACT

968 36 95 00; miguelblanquer@um.es

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: double-blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

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

Study Record Dates

• First Submitted: April 15, 2021
• First Posted: April 19, 2021
• Study Start: May 1, 2021
• Primary Completion: May 1, 2023
• Study Completion: September 1, 2023
• Last Updated: April 19, 2021

Record last verified: 2021-04

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1)