E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Allogeneic post-transplant cytopenias. |
Citopenia/s post-transplante alogénico. |
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E.1.1.1 | Medical condition in easily understood language |
Allogeneic transplantation peripheral cytopenias are those which occur after the proper graft hematopoietic progenitors. |
Las citopenias periféricas postrasplante alogénico son aquellas que se producen tras el injerto adecuado de los progenitores hematopoyéticos. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
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 |
Analyze the safety of the infusion of allogeneic mesenchymal stem cells (MSCs) expanded "in vitro" with platelet lysate in absence of animal products in the treatment of patients undergoing allo-TPH having developed one or more cytopenias posttransplant. Safety is measured in terms of: ? Adverse effects upon infusion ? Emergence of infectious complications after infusion of MSC. |
Analizar la seguridad de la infusión de células stem mesenquimales (CSM) alogénicas expandidas ?in vitro? con lisado plaquetario en ausencia de productos derivados de animales en el tratamiento de pacientes sometidos a un alo-TPH que hayan desarrollado una o varias citopenias postrasplante. La seguridad se medirá en términos de: ? Efectos adversos en el momento de la infusión ? Aparición de complicaciones infecciosas tras la infusión de CSM. |
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E.2.2 | Secondary objectives of the trial |
Analyze the efficacy of the procedure measured in terms of clinical response: ? Recovery hemoperiféricas figures ? Duration of response / relapse incidence To analyze the influence of the infusion of MSC in the risk of relapse after transplantation of the underlying disease. To study the influence of the infusion of MSC in overall survival and disease-free survival. |
Analizar la eficacia del procedimiento medida en términos de respuesta clínica: ? Recuperación de cifras hemoperiféricas ? Duración de la respuesta / incidencia de recaídas Analizar la influencia de la infusión de CSM en el riesgo de recaída postrasplante de la enfermedad de base. Estudiar la influencia de la infusión de CSM en la supervivencia global y en la supervivencia libre de enfermedad. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patients with hematologic malignancies who have undergone allo-HSCT and are diagnosed with one or more cytopenias peripheral with full chimerism in bone marrow. They may be included: a. Patients who have received BM or PS as cell source b. Patients who have received cells from a family donor or HLA-matched unrelated c. Patients transplanted with myeloablative or nonmyeloablative conditioning. 2. Patients must have adequate cardiac function without evidence of uncontrolled hypertension, congestive heart failure, angina pectoris or myocardial infarction within 6 months prior to the process. 3. Adequate pulmonary function with no evidence of obstructive or restrictive pulmonary disease severe. 4. Patients between 18 and 70 years 5. Signature of informed consent |
1. Pacientes con hemopatías malignas que hayan sido sometidos a alo-TPH y que sean diagnosticados de una o más citopenias periféricas con quimerismo completo en médula ósea. Podrán incluirse a. Pacientes que hayan recibido como fuente de células MO o SP b. Pacientes que hayan recibido células de un donante familiar o no emparentado HLA compatible c. Pacientes trasplantados con acondicionamiento mieloablativo o no mieloablativo. 2. Los pacientes deben tener una adecuada función cardiaca sin evidencia de hipertensión incontrolada, Insuficiencia cardiaca congestiva, angor pectoris o IAM en los 6 meses previos al proceso. 3. Adecuada función pulmonar sin evidencia de enfermedad pulmonar obstructiva o restrictiva severa. 4. Pacientes entre 18 y 70 años 5. Firma del consentimiento informado |
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E.4 | Principal exclusion criteria |
1. Patients whose blood disease has not been controlled by the transplantation or is in progress at the time of treatment. 2. Patients who do not have full chimerism in bone marrow 3. Patients with bacterial, viral or fungal infection that is not being controlled with proper treatment. 4. Patients with poor cardiac function and / or pulmonary 5. Patients that on investigator judgment are not in a good position to tolerate treatment 6. Patients who do not have the required donor. 7. Pregnant women or with risk of pregnancy due to inadequate contraceptive measures. 8. Patients <18 or> 70 years. 9. Patients who do not sign the consent form. |
1. Pacientes cuya hemopatía no haya sido controlada por el trasplante o esté en progresión en el momento del tratamiento. 2. Pacientes que no tengan quimerismo completo en médula ósea 3. Pacientes con infección bacteriana, vírica o fúngica que no esté siendo controlada con el tratamiento adecuado. 4. Pacientes con mala función cardiaca y/o pulmonar 5. Pacientes que a juicio del investigador no estén en una situación adecuada para tolerar el tratamiento 6. Pacientes que no tengan el donante requerido. 7. Mujeres gestantes o con riesgo de embarazo por medidas anticonceptivas inadecuadas. 8. Pacientes < 18 o > 70 años. 9. Pacientes que no firmen el consentimiento informado. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety of the procedure. |
Seguridad del procedimiento. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Safety is measured in terms of: ? Adverse effects upon infusion ? Emergence of infectious complications after infusion of MSC. |
La seguridad se medirá en términos de: ? Efectos adversos en el momento de la infusión ? Aparición de complicaciones infecciosas tras la infusión de CSM. |
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E.5.2 | Secondary end point(s) |
Efficacy of the procedure |
Eficaia del procedimiento |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Efficacy od the procedure is measured in terms of clinical response: ? Recovery hemo-peripheral figures (depending on the cytopenia original): - Complete response: ? Hb> 11 g / dL ? Neutrophils> 1.0x10e9 / L ? Platelets> 100 x10e9 / L Maintained at least 7 days - Partial response: ? Hb 10-11 g / dL ? Neutrophils > 0.5 and <1.0 x10e9 / L ? Platelets > 50 and <100 x10e9 / L Maintained at least 7 days |
La eficacia del procedimiento es medida en términos de respuesta clínica: ? Recuperación de cifras hemoperiféricas (dependiendo de la citopenia original): - Respuesta completa: ? Hb>11 g/dL ? Neutrófilos >1.0x10e9/L ? Plaquetas >100 x10e9/L Mantenido al menos 7 días - Respuesta parcial: ? Hb 10-11 g/dL ? Neutrófilos >0.5 y <1.0 x10e9/L ? Plaquetas >50 y <100 x10e9/L Mantenido al menos 7 días |
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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 | Yes |
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
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
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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Since the last infusion of the MSC to last patient recruited and until the end of the follow-up, it will be 90 days post-administration. In the first two years 15 patients plannedwill be included in the study and they will be monitor (follow-up) for a period of 90 days after the last dose of mesenchymal cells. |
Desde la última infusión de las CSM al último paciente reclutado y hasta el fin del seguimiento, será de 90 días post-administración. En los primeros 2 años serán incluidos en el estudio los 15 enfermos previstos, y se realizará un seguimiento de los mismos durante un periodo de 90 días desde la última dosis de células mesenquimales. |
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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 | 3 |
E.8.9.1 | In the Member State concerned days | |