E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Traumatic or ischemic spinal cord injury, chronically established and considered irreversible |
Lesión medular de causa traumática o isquémica, crónicamente establecida y considerada irreversible |
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E.1.1.1 | Medical condition in easily understood language |
Traumatic or ischemic spinal cord injury, chronically established and considered irreversible |
Lesión medular de causa traumática o isquémica, crónicamente establecida y considerada irreversible |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To analyze the potential clinical efficacy of intrathecal administration, in the subarachnoid space, of in vitro expanded autologous adult bone marrow mesenchymal troncal cells in the treatment of a homogeneous group of patients with established chronic spinal cord injury in the lower segments of the backbone (lumbar region) |
Analizar la posible eficacia clínica de la administración intratecal, en espacio subaracnoideo, de células mesenquimales troncales adultas autólogas de la médula ósea expandidas “in vitro” en el tratamiento de un grupo homogéneo de pacientes con lesión medular (LEM) crónicamente establecida en los segmentos inferiores de la columna vertebral (región lumbar). |
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E.2.2 | Secondary objectives of the trial |
To confirm the treatment safety to the dose raised in the present study |
Confirmar la seguridad del tratamiento, a las dosis planteadas en el presente estudio |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Clinically stable spinal injury in the low level of the cord (underneath the dorsal region) at least in the 6 months prior to the recruitment. 2. Clinical scale studies, as well as neurophysiology, urodynamic, of stool function and of the functioning studies that would allow to have useful baseline values with the purpose of being able to be compared with the same examinations after the study and obtain objective data of possible efficacy. 3. Aged between 18 and 70 years old. 4. Men and women in childbearing age must be committed to using birth control measures from the moment in which the cell removal of their bone marrow is performed up to 6 months after the last administration of CME through lumbar puncture for safety. 5. Possibility of monitoring progress and commitment of performing outpatient physiotherapy throughout the whole treatment period. 6. Written informed consent according to the current legislation. 7. Hematologic parameters and of creatinine, SGOT and SGPT within normal range according to the laboratory standards, accepting, nonetheless, modifications that are considered not significant in the context of the treatment to be carried out according to the clinical criteria of the research team. |
1. Lesión medular en nivel bajo de la médula (por debajo de la región dorsal), clínicamente estable al menos en los 6 meses previos al reclutamiento. 2. Estudios de escalas clínicas, así como estudios de neurofisiología, urodinámicos, de función defecatoria y de la marcha que permitan contar con valores basales útiles, al objeto de que puedan ser comparados con las mismas exploraciones tras el tratamiento, y poder obtener datos objetivos de posible eficacia. 3. Edad entre 18 y 70 años. 4. Mujeres y hombres en edad fértil, por seguridad, deberán comprometerse a utilizar medidas de anticoncepción desde el momento en que se le realice la extracción de células de su médula ósea hasta 6 meses después de la última administración de CME por punción lumbar. 5. Posibilidad de seguimiento evolutivo y compromiso de realizar fisioterapia ambulatoria, durante todo el periodo de tratamiento. 6. Consentimiento informado escrito, conforme a la legislación vigente. 7. Parámetros hematológicos y de creatinina, SGOT y SGPT, en rango de normalidad, de acuerdo a los estándares del laboratorio, aceptándose, no obstante, modificaciones que se consideren no significativas en el contexto del tratamiento a realizar, según criterio clínico del equipo investigador. |
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E.4 | Principal exclusion criteria |
1. Age below 18 or over 70. 2. Pregnancy or lactation. 3. Patients with systemic disease that is considered by the research team to represent an added risk to the treatment. 4. Alterations in the performed genetic study to rule out risk of cell transformation in the expansion process. 5. Patients with doubts regarding their possible cooperation in the physiotherapy maintenance or controls during the study. 6. Added neurodegenerative disease. 7. Current or past drug addiction or psychiatric disease, as well as current or past cancer disease that may interfere in the study to the investigators’ opinion. 8. HIV Positive serology and/or syphillis or allergy to protein products used in the cell expansion process. 9. Active Hepatitis B or Hepatitis C, according to the serology analysis. 10. If in the opinion of the investigator there is some other cause for which the patient is not considered to be a candidate for the study. |
1. Edad inferior a 18 años o superior a 70. 2. Embarazo o lactancia. 3. Pacientes con enfermedad sistémica que se considere por el equipo investigador que puede representar un riesgo añadido al tratamiento. 4. Alteraciones en el estudio genético realizado para descartar riesgo de transformación celular en el proceso de expansión. 5. Pacientes con dudas acerca de su posible cooperación en el mantenimiento de fisioterapia o de controles durante el estudio. 6. Enfermedad neurodegenerativa añadida. 7. Drogadicción o enfermedad psiquiátrica, actual o pasada, así como enfermedad neoplásica actual o pasada, que a juicio de los investigadores pueda interferir en el estudio. 8. Serología positiva a HIV y/o sífilis o alergia a los productos proteicos utilizados en el proceso de expansión celular. 9. Hepatitis B o Hepatitis C activa, de acuerdo al análisis de serología. 10. Si en la opinión del investigador existe alguna otra causa por la cual el paciente no se considere candidato al estudio. |
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E.5 End points |
E.5.1 | Primary end point(s) |
- Changes in ASIA scales and its subsections, as well as in IANR-SCIFRS, PENN, ASHWORTH, EVA, GEFFNER and BDS scales. - Changes in the neurophysiological records (somato-sensory evoked potentials, motor evoked potentials and EMG). - Changes in urodynamic, defaecatory funtion and gait records. |
- Modificaciones en las escalas ASIA y sus subapartados, asi como en las escalas IANR-SCIFRS, PENN, ASHWORTH, EVA, GEFFNER y BDS. - Modificaciones en los registros neurofisiológicos (Potenciales evocados somato- sensoriales, Potenciales evocados motores y EMG). - Modificaciones en registros urodinámicos, de función defecatoria y de la marcha. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Efficacy will be assessed taking into account the changes in score of the different scales and records along the study, comparing the final values with those ones which were obtained before the treatment initiation. |
La eficacia se evaluará teniendo en cuenta la variación en la puntuación de las diferentes escalas y registros a lo largo del estudio, comparando los valores finales con los obtenidos antes de iniciar el tratamiento. |
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E.5.2 | Secondary end point(s) |
Will be evaluated the possible adverse effects during CME administration, development of complications and other adverse effects after it and during the follow up period |
Se evaluarán los posibles efectos adversos durante la administración de las CME, aparición de complicaciones y otros efectos adversos tras la misma y durante el periodo de seguimiento. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
During follow up period. |
Durante el periodo de seguimiento. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |