E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients who have been treated for a B cell hematological malignancy in a Novartis sponsored or supported study of CD19 directed CAR T-Cell treatment are enrolled in this study following completion or early discontinuation from the Novartis sponsored or supported study of CD19 directed CAR T-Cell treatment. |
Pacientes que han recibido tratamiento para una enfermedad maligna hematológica de células B en un estudio patrocinado por Novartis de CD19 dirigidos al tratamiento de células CAR T serán los reclutados en este estudio después de la terminación o discontinuación temprana del estudio patrocinado por Novartis de CD19 dirigidos al tratamiento de células CAR T |
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E.1.1.1 | Medical condition in easily understood language |
Patients who have been treated in a Novartis sponsored or supported study with a CD19 directed chimeric antigen receptor (CAR) T-cell treatment for a B cell malignancy |
Pacientes que han sido tratados en un estudio patrocinado por Novartis para el tratamiento de las células T con un CD19 receptor de antígeno quimérico (CAR) para una neoplasia maligna de células B |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10003917 |
E.1.2 | Term | B-cell type acute leukaemia |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10003899 |
E.1.2 | Term | B-cell lymphoma |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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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 |
to monitor all patients exposed to CD19 directed CAR T-cells (CD19 CART) for 15 years following last CD19 CART (e.g. CTL019) infusion to assess the risk of delayed adverse events (AEs). |
Monitorizar a todos los pacientes expuestos a células T modificadas con CAR dirigidas a CD19 (CD19 CART) durante 15 años después de la última infusión de CD19 CART (p. ej., CTL019) para evaluar el riesgo de acontecimientos adversos (AAs) tardíos |
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E.2.2 | Secondary objectives of the trial |
- Monitor the persistence of CD19 CAR transgene - Monitor for replication competent lentivirus (RCL) - Assess the long-term efficacy of CD19 CART |
? Monitorizar la persistencia del transgén CD19 CAR en sangre periférica ? Monitorizar la expresión de LCR (lentivirus competentes para replicación) ? Evaluar la eficacia a largo plazo de CD19 CART, incluida la proporción de pacientes que experimentan recidiva o progresión entre los pacientes que no habían experimentado recidiva o progresión en la inclusión/reinclusión en el estudio, y la incidencia de fallecimientos ? Monitorizar los niveles de linfocitos ? Describir el crecimiento, el desarrollo y el estado reproductor de las pacientes que tenían < 18 años en el momento de la infusión inicial de CD19 CART |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- All patients who have received anti-CD19 directed CART therapy and completed or discontinued early from a Novartis sponsored treatment protocol that utilized CD19-directed CART cells or from any CD19 CART trial sponsored by the University of Pennsylvania with which Novartis has a contractual agreement to co-develop the CAR technology. - Patients who have provided informed consent for the long term follow up study prior to their study participation . |
? Todos los pacientes que recibieron terapia CART dirigida a CD19 y que la completaron o interrumpieron prematuramente procedentes de un protocolo de tratamiento patrocinado por Novartis en el que se utilizó terapia celular CART dirigida a CD19 o procedentes de algún ensayo con CD19 CART patrocinado por la Universidad de Pennsylvania con la que Novartis ha firmado un contrato para el codesarrollo de la tecnología CAR. ? Pacientes que han proporcionado el consentimiento informado para el estudio de seguimiento a largo plazo antes de su participación en el estudio. |
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E.4 | Principal exclusion criteria |
- There are no specific exclusion criteria for this study. |
No existen criterios de exclusión específicos para este estudio. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Proportion of patients with listed categories: - New malignancies - Incidence/exacerbation of pre-existing neurologic disorder - New incidence or exacerbation of a prior rheumatologic or other autoimmune disorder - New incidence of a hematologic disorder |
Proporción de pacientes con categorías enumeradas: - Nuevos procesos malignos - Incidencia / exacerbación de la preexistente trastorno neurológico - Nueva incidencia o exacerbación de un trastorno autoinmune reumatológicos u otro antes - Nueva incidencia de un trastorno hematológico |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Time Frame: 3 monthly until year 1, 6 monthly until year 5, yearly until year 15. |
Plazo: 3 mes hasta el primer año, 6 meses hasta 5 años, anualmente hasta el año 15. |
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E.5.2 | Secondary end point(s) |
?Proportion of patients with detectable CD19 CAR transgene levels in peripheral blood by q-PCR at pre- specified post CD19 CART infusion time points - Proportion of patients with detectable RCL by VSV-G - Proportion of patients who relapse or progress among patients who had not relapsed or progressed at study entry/re-entry; Incidence of death. - Absolute B- and T- lymphocyte count - Height and weight, Tanner staging, menstrual cycle status |
? Proporción de pacientes con CD19 detectable niveles transgenes CAR en sangre periférica por q-PCR en pre poste CD19 especificado los puntos de tiempo de infusión Carro - Proporción de pacientes con detectable RCL por VSV-G - Proporción de pacientes que recaen o de progreso entre los pacientes que no habían recaído o progresado al inicio del estudio / reingreso; La incidencia de muerte. - Absolute B y recuento de linfocitos T - Altura y peso, Tanner puesta en escena, el estado del ciclo menstrual |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Time Frame: 3 monthly until year 1, 6 monthly until year 5, yearly until year 15. |
Plazo: 3 mes hasta el primer año, 6 meses hasta 5 años, anualmente hasta el año 15. |
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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 | Yes |
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) | Yes |
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 | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 30 |
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 |
Australia |
Austria |
Belgium |
Canada |
France |
Germany |
Italy |
Netherlands |
Norway |
United States |
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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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The end of the LTFU study for each individual patient will be 15 years after the date of patients? last infusion of CD19 CART. The end of study as a whole is defined as the last patient?s last visit (LPLV), which is the last patient?s last evaluation (visit Year 15), or the time of premature withdrawal. |
El fin del estudio de LTFU para cada paciente individual será de 15 años después de la fecha de la última infusión de CD19 CART del paciente. Debería cumplimentarse una página del CRD de final de la disposición del seguimiento postratamiento, anotando la fecha y el motivo para la interrupción del estudio. |
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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 | 20 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 20 |
E.8.9.2 | In all countries concerned by the trial months | 6 |