Clinical trial NCT02286011: Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis
Official title: Phase I Clinical Trial on Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis.
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: ALS (Amyotrophic Lateral Sclerosis)
Lead sponsor: Red de Terapia Celular
Sponsor class: INDUSTRY
Collaborators: name: Spanish National Health System
name: Hospital Universitario Virgen de la Arrixaca
name: Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
name: Public Health Service, Murcia
name: Instituto Murciano de Investigación Biosanitaria Virgen de la Arrixaca
First posted: 2014-11-07
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

* 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)
* The patient must fulfill all inclusion criteria.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Rate of serious and non-serious adverse events related to the use of bone marrow mononuclear cells in patients with Amyotrophic Lateral Sclerosis.
timeFrame: 24 months from baseline
Secondary outcomes:
measure: Estimated number of motor units (MUNE)
timeFrame: 24 months from baseline
measure: Compound muscle action potential (CMAP)
timeFrame: 24 months from baseline
measure: Fiber density (FD)
timeFrame: 24 months from baseline
measure: Muscle force MRC (Medical Research Council) score
timeFrame: 24 months from baseline
measure: Maximum force developed in an isometric contraction of the tibialis anterior (TA) muscle.
timeFrame: 24 months from baseline
measure: Maximum transversal area of the tibialis anterior (TA)
timeFrame: 24 months from baseline
Other outcomes:
Locations:
city: El Palmar
country: Spain
facility: Clinical Universitary Hospital Virgen de la Arrixaca
state: Murcia
Study officials:
affiliation: Hospital Universitario Virgen de la Arrixaca
name: Joaquín A Gómez Espuch, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.