E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
High risk haematological malignancies
Hemoglobinopathies |
Neoplasias hematológicas de alto riesgo
hemoglobinopatías |
|
E.1.1.1 | Medical condition in easily understood language |
-Cancer affecting blood, bone marrow, lymph nodes
-A kind of genetic defect that results in abnormal structure of one of the globin chains of the hemoglobin molecule |
-cáncer que afecta a la sangre, la médula ósea, los ganglios linfáticos
-A Tipo de defecto genético que resulta en la estructura anormal de una de las cadenas de globina de la molécula de hemoglobina |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10025316 |
E.1.2 | Term | Lymphoma NOS |
E.1.2 | System Organ Class | 100000004864 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10000845 |
E.1.2 | Term | Acute lymphoblastic leukemia |
E.1.2 | System Organ Class | 100000004864 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10000886 |
E.1.2 | Term | Acute myeloid leukemia |
E.1.2 | System Organ Class | 100000004864 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10054658 |
E.1.2 | Term | Thalassemia |
E.1.2 | System Organ Class | 100000004850 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10009015 |
E.1.2 | Term | Chronic myeloid leukemia |
E.1.2 | System Organ Class | 100000004864 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10040645 |
E.1.2 | Term | Sickle cell disease NOS |
E.1.2 | System Organ Class | 100000004850 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10028534 |
E.1.2 | Term | Myelodysplastic syndrome NOS |
E.1.2 | System Organ Class | 100000004864 |
|
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Describe the clinical outcomes of patients who have received a NiCord/CordIn transplantation |
Describir los resultados clínicos de los pacientes que hayan recibido un trasplante de NiCord/CordIn |
|
E.2.2 | Secondary objectives of the trial |
Describe long term sustained engraftment of NiCord/CordIn transplantation
Describe survival and disease free survival in NiCord/CordIn infused patients
Describe characteristics of patients with secondary graft failure or disease relapse
Describe long term immune reconstitution characteristics of patients who receive NiCord/CordIn
Describe incidence of chronic GvHD
|
Describir el injerto prolongado a largo plazo del trasplante de NiCord/CordIn
Describir la supervivencia y la supervivencia libre de enfermedad de pacientes que hayan recibido una perfusión de NiCord/CordIn.
Describir las características de los pacientes con rechazo de injerto secundario o recidiva de la enfermedad.
Describir las características de reconstitución inmune a largo plazo de los pacientes que reciben NiCord/CordIn.
Describir la incidencia de EICH crónica.
|
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Anyone who has received a NiCord/CordIn transplant, signed the consent for this study, and completed study specific follow up. |
Cualquier persona que haya recibido un trasplante de NiCord/CordIn, firmado el consentimiento para este estudio y finalizado el seguimiento específico del estudio. |
|
E.4 | Principal exclusion criteria |
A patient is not eligible if during the NiCord/CordIn transplant protocol, any of the following occurred before or on the completion of the last study visit (d365):
Disease relapse/progression
Autologous Recovery
Graft Failure
|
Un paciente no es elegible si, durante el protocolo de trasplante de NiCord/CordIn, se produce alguno de los siguientes casos, antes o una vez finalizada la última visita del estudio (d365):
Recidiva/progresión de la enfermedad.
Recuperación autóloga.
Rechazo de injerto.
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Describe the clinical outcomes of patients who have received a NiCord/CordIn transplantation |
Describir los resultados clínicos de los pacientes que hayan recibido un trasplante de NiCord/CordIn |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
The study includes 4 timepoints as follows:
Visit #1: 2 years post NiCord/CordIn infusion
Visit #2: 3 years post NiCord/CordIn infusion
Visit #3: 4 years post NiCord/CordIn infusion
Visit #4: 5 years post NiCord/CordIn infusion
|
Visita n.º 1: 2 años después de la perfusión de NiCord/CordIn
Visita n.º 2: 3 años después de la perfusión de NiCord/CordIn
Visita n.º 3: 4 años después de la perfusión de NiCord/CordIn
Visita n.º 4: 5 años después de la perfusión de NiCord/CordIn
|
|
E.5.2 | Secondary end point(s) |
- Describe long term sustained engraftment of NiCord/CordIn transplantation
- Describe survival and disease free survival in NiCord /CordIn infused patients
- Describe characteristics of patients with secondary graft failure or disease relapse
-Describe long term immune reconstitution characteristics of patients who receive NiCord/CordIn
-Describe incidence of chronic GvHD
|
Describir el injerto prolongado a largo plazo del trasplante de NiCord/CordIn
Describir la supervivencia y la supervivencia libre de enfermedad de pacientes que hayan recibido una perfusión de NiCord/CordIn.
Describir las características de los pacientes con rechazo de injerto secundario o recidiva de la enfermedad.
Describir las características de reconstitución inmune a largo plazo de los pacientes que reciben NiCord/CordIn.
Describir la incidencia de EICH crónica.
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
The study includes 4 timepoints as follows:
Visit #1: 2 years post NiCord/CordIn infusion
Visit #2: 3 years post NiCord/CordIn infusion
Visit #3: 4 years post NiCord/CordIn infusion
Visit #4: 5 years post NiCord/CordIn infusion
|
Visita n.º 1: 2 años después de la perfusión de NiCord/CordIn
Visita n.º 2: 3 años después de la perfusión de NiCord/CordIn
Visita n.º 3: 4 años después de la perfusión de NiCord/CordIn
Visita n.º 4: 5 años después de la perfusión de NiCord/CordIn
|
|
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 | No |
E.6.5 | Efficacy | No |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Observational study that will monitor clinical outcomes of patients who have received a NiCord/CordIn transplant |
Se trata de un estudio observacional en el que se monitorizarán los resultados clínicos de los pacientes que hayan recibido un trasplante de NiCord/CordIn |
|
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 6 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
|
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
|
LVLS |
sujeto último visita última |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |