amyotrophic lateral sclerosis MedDRA version: 15.1 Level: PT Classification code 10002026 Term: Amyotrophic lateral sclerosis System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Product Name: células troncales hematopoyéticas autólogas adultas extraidas de médula ósea
Pharmaceutical Form: Solution for injection/infusion in pre-filled syringe
INN or Proposed INN: autologous bo
Sponsors
Fundación Sanitaria para el Fomento de la Investigación Sanitaria y Biomédica de la Comunidad Valenciana (FISABIO)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Aged between 18 and 70 years. - Diagnosis of definite or probable ALS according to the criteria established by the World Federation of Neurology. - Patient providing sufficient guarantees of adherence to protocol. - Neurophysiological data confirming lower motor neuron involvement of lumbar level. - No motor deficit in dorsiflexion of both feet (4 + or 5 points on the MRC) 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 15 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
Exclusion criteria: - Diabetes Mellitus. - Other diseases that may occur with polyneuropathy. - A previous history of stroke. - A previous pathology of peripheral nervous system that affects one or both lower limbs with or without clinically evident neurologic sequelae. - Patients who are pregnant or breastfeeding active - Patients physiologically capable of becoming pregnant, unless they are using reliable contraception (Annex III) - Patients with heart, kidney, liver, systemic immune that may influence patient survival during the test. - Positive serology for hepatitis B, hepatitis C or HIV. - Clinical criteria and anesthesia sedation contraindicating either itself or extraction MO (Altered coagulation system or anticoagulated patient inability to withdraw anticoagulation, hemodynamic instability, altered skin puncture area, etc.) - Included in other clinical trials in the last 6 months. - Inability to understand informed consent.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the safety of intramuscular infusion of stem cells from autologous bone marrow in patients with amyotrophic lateral sclerosis. To determine the ability of stem cells from autologous bone marrow to modify the time course of progressive loss of motor units that characterizes amyotrophic lateral sclerosis in tibialis anterior muscle.;Secondary Objective: Determine the usefulness of ultrasound for monitoring muscle muscle morphology in the evolution of patients with ALS. Correlate the maximum force of isometric contraction muscle and muscle morphology (obtained from ultrasound) with the estimated number of motor units in the evolution of patients with ALS.;Primary end point(s): 1)Absence of serious adverse events (SAE) related to the procedure. 2) Rate of non-serious adverse events regarding possible, probable or defined with the procedure. 3) Neurophysiological parameters (measured in both TA muscles): - Motor units number estimation (MUNE) - fiber density (FD) - Amplitude and area of the compound muscular action potential (CMAP). 4) Neurological: - Maximum strength developed in an isometric contraction in both TA muscles. - The measurement will be performed with a digital dynamometer during dorsiflexion of the foot. Neurophysiological and neurological assessments were made in the times that are specified in the protocol: at baseline and at 30, 90 and 180 days of infusion.Also, there will be a neurological assessment at 7 days of infusion. See Appendix B (Protocol);Timepoint(s) of evaluation of this end point: Number of serious adverse events related to the procedure. Follow-up: Visit 0 (Day 0) up to visit 6 (day +180) ? neurophysiological parameters: MUNE, DF, PAMC. Follow-up: baseline visit (day -14), visit 2 (day +30), visit 4 (day +90) and visit 6 (day +180) ? Muscular strength developed in an isometric contraction. Follow-up: baseline visit (day -14), visit 2 (day +30), visit 4 (day +90) and visit 6 (day +180) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1)Neurological variables: Assessment of motor function. The muscle force data are collected according to the scale of the Medical Research Council (1943). Muscle strength is graded on a scale of 0 to 5, with 5 being normal muscle strength and 0 absence of muscle contraction. 2) Sonographic variables: - Presence of local complications related to the process (edema, abscesses, muscle necrosis, etc.): Yes / No - Maximum transverse area of both TA muscles. The ultrasound study was performed using a linear probe of 12 to 15 MHz These assessments will be made in the times that are specified in the protocol: at baseline, at 7, 30, 90 and 180 days of infusion;Timepoint(s) of evaluation of this end point: ? Variables Neurological Evaluation of the TA muscle motor function of both lower limbs using the MRC scale. ? Ultrasound Variables: Analysis of the TA muscle of both legs to assess muscle mass and maximum cross-sectional area, analyzing the appearance of edema, abscess, or necrosis. | — |
Countries
Spain
Contacts
Public ContactRocío Caño Alameda
CRO-Fundación de la C.Valenciana para la Investigación en el Hospital General Universitario de Alicante
Outcome results
None listed