Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis

NCT02286011 | Unknown Phase 1 DMC

• 2 other identifiers: TCIM/ELA2011-004801-25
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the safety of Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis by a prospective, single-center, randomized, parallel, double-blind, placebo-controlled phase I clinical trial.

Conditions

ALS (Amyotrophic Lateral Sclerosis)

Outcome Measures

Primary Outcomes (1)

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

  24 months from baseline

Secondary Outcomes (6)

• Estimated number of motor units (MUNE)

  24 months from baseline

• Compound muscle action potential (CMAP)

  24 months from baseline

• Fiber density (FD)

  24 months from baseline

• Muscle force MRC (Medical Research Council) score

  24 months from baseline

• Maximum force developed in an isometric contraction of the tibialis anterior (TA) muscle.

  24 months from baseline

Study Arms (2)

MNC (Mononuclear cells)

EXPERIMENTAL

All patients included in the clinical trial will receive an intramuscular infusion of autologous mononuclear cells (MNC) of Bone Marrow (BM) in TA muscle of one of the lower limb (experimental group). The lower limb on the CMN infuse autologous BM will be determined randomly. The average dose is 550 millions of cells (100-1200 million) diluted in 2 ml. saline

Biological: MNC (Mononuclear cells)

Saline

PLACEBO COMPARATOR

All patients included in the clinical trial will receive an intramuscular infusion of 2 mL of saline (placebo) in the TA muscle of the contralateral limb (group control).

Other: Saline

Interventions

MNC (Mononuclear cells) BIOLOGICAL

The cells are infused into the TA muscle of the lower limb randomized as Group A (experimental) intramuscularly. The infusion is made with a needle, 26 gauge, at 4 points of the TA muscle a specific depth given by the neurophysiological study. The total volume infused will be 2 ml, 0.5 ml at each point. For infusion is as painless as possible for the patient to comply exactly with stereotactic indications of neurophysiology, the infusion was made at a uniform controlled rate and for this, the syringe is placed on a Yesargil arm, equipped with a microinjector and controlled infusion device.

MNC (Mononuclear cells)

Saline OTHER

The placebo, 2ml of saline, will be infused like in the experimental arm.

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 definite or probable ALS according to the criteria established by the World Federation of Neurology
• Patient that provides reasonable assurance of adherence to protocol.
• Neurophysiological data confirming affectation of lower motor neurons in the lumbar region.
• Assessment of motor deficits in dorsiflexion of both feet (4 or 5 points on the MRC scale)

You may not qualify if:

• Diabetes Mellitus.
• Other diseases that may present with polyneuropathy.
• Previous history of stroke.
• Prior Pathology of the peripheral nervous system affecting one or both lower limbs with or without clinically evident neurological sequelae.
• Pregnant or breastfeeding patients active.
• Patients physiologically capable of becoming pregnant, unless they are using reliable contraception.
• Patients with cardiac disease, renal, hepatic, systemic, immune that may influence patient survival during the test.
• Positive serology for hepatitis B, hepatitis C or HIV.
• Clinical and anesthesiologic Criteria, contraindicating either sedation or extraction of MO (Altered coagulation system or anticoagulated patient with inability to withdraw anticoagulation, hemodynamic instability, altered skin puncture site, etc.)
• Included in other clinical trials in the last 6 months.

Sponsors & Collaborators

• Red de Terapia Celular: lead
• Spanish National Health System: collaborator
• Hospital Universitario Virgen de la Arrixaca: collaborator
• Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia: collaborator
• Public Health Service, Murcia: collaborator
• Instituto Murciano de Investigación Biosanitaria Virgen de la Arrixaca: collaborator

Study Sites (1)

Clinical Universitary Hospital Virgen de la Arrixaca

El Palmar, Murcia, 30120, Spain

Location

Related Publications (2)

• Blanquer M, Moraleda JM, Iniesta F, Gomez-Espuch J, Meca-Lallana J, Villaverde R, Perez-Espejo MA, Ruiz-Lopez FJ, Garcia Santos JM, Bleda P, Izura V, Saez M, De Mingo P, Vivancos L, Carles R, Jimenez J, Hernandez J, Guardiola J, Del Rio ST, Antunez C, De la Rosa P, Majado MJ, Sanchez-Salinas A, Lopez J, Martinez-Lage JF, Martinez S. Neurotrophic bone marrow cellular nests prevent spinal motoneuron degeneration in amyotrophic lateral sclerosis patients: a pilot safety study. Stem Cells. 2012 Jun;30(6):1277-85. doi: 10.1002/stem.1080.

  PMID: 22415951 RESULT

• Geijo-Barrientos E, Pastore-Olmedo C, De Mingo P, Blanquer M, Gomez Espuch J, Iniesta F, Iniesta NG, Garcia-Hernandez A, Martin-Estefania C, Barrios L, Moraleda JM, Martinez S. Intramuscular Injection of Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis Patients: A Randomized Clinical Trial. Front Neurosci. 2020 Mar 24;14:195. doi: 10.3389/fnins.2020.00195. eCollection 2020.

  PMID: 32265627 DERIVED

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

Study Officials

• Joaquín A Gómez Espuch, MD

  Hospital Universitario Virgen de la Arrixaca

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 24, 2014
• First Posted: November 7, 2014
• Study Start: November 1, 2014
• Primary Completion: December 1, 2017
• Study Completion: December 1, 2017
• Last Updated: March 30, 2017

Record last verified: 2017-03

Locations

ES (1)