E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Multiple Myeloma |
Mieloma Múltiple |
|
E.1.1.1 | Medical condition in easily understood language |
Multiple Myeloma |
Mieloma Múltiple |
|
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 | 21.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10028228 |
E.1.2 | Term | Multiple myeloma |
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 |
To collect long-term follow-up data on delayed adverse events after administration of cilta-cel, and to characterize and understand the long-term safety profile of cilta-cel. |
Recoger datos de seguimiento a largo plazo sobre acontecimientos adversos tardíos tras la administración de cilta-cel, así como caracterizar y comprender el perfil de seguridad a largo plazo de cilta-cel. |
|
E.2.2 | Secondary objectives of the trial |
To collect additional long-term data on replication competent lentivirus (RCL), product persistence, efficacy, and overall survival |
Recoger datos adicionales de seguimiento a largo plazo sobre la replicación de Lentivitrus competente (RLC), persistencia del producto, eficacia y supervivencia global. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
All subjects who received cilta-cel in a Janssen-sponsored clinical study are candidates for Study MMY4002. The inclusion criteria for the study are described below: 1. Subjects who have received at least one dose of cilta-cel in a Janssen-sponsored clinical study. 2. Subjects who have provided informed consent for Study MMY4002. |
Todos los sujetos que recibieron cilta-cel en un estudio clínico patrocinado por Janssen son candidatos para el estudio MMY4002. Los criterios de inclusión para el estudio se describen a continuación: 1. Sujetos que hayan recibido al menos una dosis de cilta-cel en un estudio clínico patrocinado por Janssen. 2. Sujetos que hayan dado su consentimiento informado para el estudio MMY4002. |
|
E.4 | Principal exclusion criteria |
No exclusion criteria are applicable in this study. |
No aplican criterios de exclusión en este estudio. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Number of subjects with delayed adverse events associated with administration of Cilta-cel. The following adverse events will be collected: - New malignancies and recurrence of preexisting malignancy (all grades) - New incidence or exacerbation of a preexisting neurologic disorder (all grades) - New incidence or exacerbation of a preexisting rheumatologic or other autoimmune disorder (all grades) - New incidence of Grade ≥3 hematologic disorder (For Years 6-15, only serious hematologic disorder) - New incidence of Grade ≥3 infection (For Years 6-15, only serious infection) - All serious adverse events (For Years 6-15, only related serious adverse events assessed by the Investigator) |
Número de sujetos con eventos adversos tardíos asociados con la administración de Cilta-cel. Se recogerán los siguientes eventos adversos: - Neoplasias malignas nuevas y recurrencia de neoplasias malignas preexistentes (todos los grados) - Nueva incidencia o exacerbación de un trastorno neurológico preexistente (todos los grados) - Nueva incidencia o exacerbación de un trastorno reumatológico u otro autoinmune preexistente (todos los grados) - Nueva incidencia de trastorno hematológico de grado >/=3 (para los años 6 -15, solo trastorno hematológico grave) - Nueva incidencia de infección de grado >/=3 (para los años 6 -15, solo infección grave) - Todos los eventos adversos graves (para los años 6 -15, solo eventos adversos graves relacionados evaluados por el investigador) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
For New malignancies and recurrence of pre-existing malignancy (all grades) and All serious adverse events: Continuous from the time of enrollment
For the others endpoints: At enrollment and Yearly |
Para neoplasias malignas nuevas y recurrencia de neoplasias malignas preexistentes (todos los grados) y todos los eventos adversos graves: continuamente desde el momento de la inscripción
Para los otros criterios de valoración: en la inscripción y anualmente |
|
E.5.2 | Secondary end point(s) |
- Number of subjects with measurable RCL in peripheral blood. - Number of subjects with CAR transgene level >lower limit of quantitation (LLOQ) in peripheral blood cells. - Assessment of the pattern of vector integration sites if >1% of cells in the blood sample or new malignancy are positive for vector sequences. - Long term follow-up on CAR-T therapy efficacy if the subject does not have confirmed disease progression or does not initiate subsequent anti-myeloma therapy at the entry of the study and at any time of during the study. - Overall Survival (OS) |
- Número de sujetos con RCL (lentivirus competente para la replicaicón) medible en sangre periférica. - Número de sujetos con nivel de transgén CAR > límite inferior de cuantificación (LLOQ) en células de sangre periférica. - Evaluación del patrón de los sitios de integración del vector si >1% de las células en la muestra de sangre o neoplasia maligna nueva son positivas para las secuencias del vector. - Seguimiento a largo plazo de la eficacia de la terapia CAR-T si el sujeto no tiene una progresión confirmada de la enfermedad o no inicia una terapia posterior contra el mieloma al inicio del estudio y en cualquier momento durante el estudio. - Supervivencia general (SG) |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Replication competent lentivirus |
Lentivirus competentes para la replicación |
|
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 12 |
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 |
China |
Israel |
Japan |
Saudi Arabia |
Singapore |
United States |
Belgium |
France |
Germany |
Netherlands |
Spain |
|
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
|
Last Patient Last Visit |
Última Visita del Último Paciente |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 18 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 19 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |