Amyotrophic lateral sclerosis (ALS) MedDRA version: 14.1 Level: PT Classification code 10002026 Term: Amyotrophic lateral sclerosis System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Product Name: Células madre mesenquimales de tejido adiposo autólogo
Pharmaceutical Form: Suspension for infusion
INN or Proposed INN: NA
CAS Number: NA
Current Sponsor code: NA
Other descriptive name
Sponsors
FUNDACIÓN PROGRESO Y SALUD
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: ?Subjects > 18 years of age. ?Signed informed consent ?Diagnose established following the Escorial criteria from World Federation of Neurology ?Vital Capacity ? 50% ?More than 6 and less than 36 months of evolution of the disease ?Possibility of obtaining at least 50 grams of adipose tisue. ?Treated with riluzole Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 40 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 40
Exclusion criteria
Exclusion criteria: 1.Any concurrent illness that could affect the measure of the clinical trial variables (hepatic, renal, cardiac insufficiency, diabetes mellitus, others) 2.Previous stem cell therapy. 3.Any lymphoproliferative disease. 4.Riluzole with 4 weeks of study entry and at any time during the study. 5.Hemophiliacs or subjects with bleeding disorders. 6.Known hypersensitivity to fetal bovine serum 7.HIV infection. 8.Serum creatinine > 3.0 in subjects not on hemodialysis. 9.Skin infection at the infusion site or systemic infection 10.Active drug or alcohol addiction 11.Pregnant, planning to become pregnant or not on accepted birth control method if subject is of child bearing potential. 12.Subjects that are breast feeding 13.Any condition that the Principal Investigator considers would render the subject unfit for the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary end point(s): -Adverse events (OMS scale): cuantitative variable (number of events); cualitative variable (severity) -Complications at the site of infusion: cuantitative variable (number of events); cualitative variable (severity) -Appearance of new neurological deficit not attributed to the natural progression of the disease: cuantitative variable (number of events); cualitative variable (severity) -Laboratory analysis: cuantitative variables.;Timepoint(s) of evaluation of this end point: Not applicable;Secondary Objective: a) Evaluation of clinical efficacy of intravenous administration of 3 doses of autologous CeTMAd, measured by: ?Progression rate of the neurological dysfunction of the disease ?Muscle strength grading ?Changes in forced vital capacity ?Changes in muscle bulk and circumference of the upper and lower extremities: ?Changes in neurophysiological, neuropsychological and Quality of Life parameters ?Changes in the spasticity and pain measures ?Need of and time to tracheotomy or permanent assisted ventilation ?Need of and time to gastrostomy ?Overall survival, calculating time to death b) Inmmunological effects: ?Regulatory cell populations affecting the inflammatory and tolerogenic status and ?Immunological control mechanisms mediated by TLRs. c) Adquisition of metabolomic profiling data as an alternative tool for monitoring and identification of new markers in ALS.;Main Objective: Safety evaluation and tolerability of the IV administration of 3 doses of autologous CeTMAd, measured by the ABSENCE of: -Complications at the site of infusion -Appearance of new neurological deficit not attributed to the natural progression of the disease: -Adverse events (OMS scale: clinical, haematological, bioquemical) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary variables: Clinical variables ? Sex: cualitative variable variable (male, female). ? Age: cuantitative variable (years) ? Age at beginning of illness: cuantitative variable (years) ? Neurological dysfunction measured by Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) at beginning of the clinical trial: cuantitative variable ?Progression rate of the disease as evidenced by changes in the ALS functional rating scale (ALSFRS-R): cuantitative variable ?Muscle strength grading (measured by Manual Muscular Testing (MMT) and the Maximal Voluntary Isometric Contraction (MVIC): cuantitative variables ?Changes in forced vital capacity (FVC %): cuantitative variable ?Changes in upper and lower extremities circumference (cm): cuantitative variables ?Need of tracheotomy or permanent assisted ventilation: cualitative variable ?Time to tracheotomy or permanent assisted ventilation: cuantitative variable ?Overall survival, calculating time to death: cualitative variable ?Changes in the spasticity Ashworth scale: cuantitative variable ?Changes in the Visual analogue scale (VAS) of pain and in McGill Pain Questionnaire: cuantitative variable Inmmunological análisis: ?Cellular subpopulations ( T CD4+, T CD8+, NK y monocytes): cuantitative variable (nº,%). ?IFN-?, TNF-?, IL-2, IL-12, IL-4, IL-5, IL-10, IL-17A, IL-23 and IL-6: cuantitative variables. Neurophysiological variables: cuantitative variables: ?Changes in Neurophysiological Index (NI) ?in Motor unit number estimation (MUNE) ? in magnetic Motor evoked potentials (MEP) amplitude ?in excitability thereshold of MEP Magnetic Resonance Imaging (MRI) variables: ?Changes in muscle bulk estimated by MRI of the upper and lower extremities: cuantitative variables Neuropsychological variables : ?Escala de Inteligencia de Wechsler para Adultos. WAIS-III: Variables cuantitativas Quality of life variables: ?Changes in the quality of life measured by Sickness Impact Profile | — |
Countries
Spain
Contacts
Public ContactROSARIO MATA ALCAZAR-CABALLERO
FUNDACIÓN PROGRESO Y SALUD
Outcome results
None listed