E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
CML-CP (at the end of parent study) who are currently participating in an asciminb Novartis sponsored study (parent study) |
Pacientes con LMC en FC (al final del estudio principal) que estén participando actualmente en un estudio de asciminb promocionado por Novartis (estudio principal). |
|
E.1.1.1 | Medical condition in easily understood language |
Chronic Myelogenous Leukemia is a cancer of the blood characterized by a gene mutation (Philadelphia chromosome) which causes proliferation of white blood cells in the bone marrow. |
La leucemia mielógena crónica es un cáncer de la sangre caracterizado por una mutación genética (cromosoma Filadelfia) que provoca la proliferación de leucocitos en la médula ósea. |
|
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 | 10009012 |
E.1.2 | Term | Chronic myelogenous leukemia |
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 |
The primary objective of this study is to assess long term safety data (i.e SAEs and AEs) |
El objetivo principal de este estudio es evaluar los datos de seguridad a largo plazo (es decir, AAG y AA). |
|
E.2.2 | Secondary objectives of the trial |
The secondary objective of this study is to evaluate ongoing clinical benefit as assessed by the Investigator. |
El objetivo secundario de este estudio es evaluar el beneficio clínico en curso según el criterio del investigador. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Signed informed consent must be obtained prior to participation in the study. 2. Participant with CML-CP (according to ELN guidelines 2013) is currently receiving treatment with asciminib (or asciminib in combination with imatinib, or imatinib alone or nilotinib alone for CABL001E2201 participants, or bosutinib for CABL001A2301 participants) within a Novartis-sponsored study, which has fulfilled the requirements for the primary objective and has completed treatment phase and, in the opinion of the Investigator, would benefit from continued treatment. 3. Participant has demonstrated compliance, as assessed by the investigator, with the parent study protocol requirements and is willing and able to comply with scheduled visits, treatment plans and any other study procedures. 4. Other protocol-defined inclusion criteria may apply |
1. Se deberá obtener el consentimiento informado firmado antes de la participación en el estudio. 2.Participantes con LMC-FC (según las guías de la ELN de 2013) que estén recibiendo actualmente tratamiento con asciminib (o asciminib en combinación con imatinib, o imatinib en monoterapia o nilotinib en monoterapia para participantes del CABL001E2201, o bosutinib para participantes del CABL001A2301) en un estudio promocionado por Novartis, que hayan cumplido los requisitos para el objetivo principal y hayan completado la fase de tratamiento y que, según el investigador, obtendrían beneficio del tratamiento continuado. 3. El participante ha demostrado que reúne, a criterio del investigador, los requisitos del protocolo del estudio principal y desea y es capaz de cumplir con las visitas programadas, los planes de tratamiento y otros procedimientos del estudio. 4. Se podrían aplicar otros criterios de inclusión definidos en el protocolo |
|
E.4 | Principal exclusion criteria |
1. Participant has been discontinued from parent study treatment due to any reason. 2. Participant currently has unresolved toxicities of grade 3 or 4 reported as possibly related to study treatment in the parent study, which have: - not resolved to Grade 2 or lower within 42 days and /or require dose interruption for longer than 42 days for hematological toxicities, - not resolved to Grade 2 or lower within 28 days and /or require dose interruption for longer than 28 days in case of non-hematological toxicities. 3. Participant’s ongoing treatment is currently approved and reimbursed at country level. 4. Pregnant or nursing (lactating) women. 5. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception while taking study treatment and for 3 days after stopping asciminib, 14 days after stopping imatinib or nilotinib and one month after stopping bosutinib medication. 6. For participants in the USA and on asciminib treatment only: Sexually active males unwilling to use a condom during intercourse while taking study treatment and for 3 days after stopping study treatment. In addition, male participants must not donate sperm for the time period specified above. 7. Applicable only for participants on bosutinib treatment at the end of the ABL001A2301 study that switch to asciminib treatment at enrollment: -Asymptomatic (grade 2) pancreatitis if not resolved within 28 days. -QTcF > 480 msec or inability to determine QTc interval 8. Other protocol-defined exclusion criteria may apply |
1. Participantes que hayan discontinuado el tratamiento del estudio principal por cualquier motivo. 2. Participantes que actualmente tengan toxicidades no resueltas de grado 3 o 4 notificadas como posiblemente relacionadas con el tratamiento del estudio l, que: • No hayan pasado a grado 2 o inferior en un periodo de 42 días o que requieran interrupción de la dosis durante más de 42 días por toxicidades hematológicas. • No hayan pasado a grado 2 o inferior durante un periodo de 28 días o que requieran interrupción de la dosis durante más de 28 días en caso de toxicidades no hematológicas. 3. El tratamiento en curso del participante está actualmente aprobado y se reembolsa en su país. 4. Mujeres embarazadas o en periodo de lactancia. 5. Mujeres con posibilidad de quedarse embarazadas, definidas como toda mujer fisiológicamente capaz de quedarse embarazada, salvo que esté utilizando métodos anticonceptivos altamente eficaces mientras toma el tratamiento del estudio y durante 3 días después de dejar de tomar asciminib, 14 días después de dejar de tomar imatinib o nilotinib y un mes después de dejar de tomar bosutinib. 6. Solo para participantes en los EE. UU. y que reciban tratamiento con asciminib: Los varones sexualmente activos que no deseen utilizar un preservativo durante el coito mientras estén tomando el tratamiento del estudio ni durante 3 días después de haber suspendido el tratamiento. Además, los participantes varones no deben donar esperma durante el periodo de tiempo especificado anteriormente. 7. Aplica solo a participantes en tratamiento con bosutinib al final del estudio ABL001A2301 que cambien al tratamiento con asciminib en el reclutamiento: -Pancreatitis asintomática (grado 2) si no se resuelve en 28 días. -QTcF >480 mseg o incapacidad de determinar el intervalo QTc 8. Se podrían aplicar otros criterios de exclusión definidos en el protocolo. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
The frequency and severity of AEs/SAEs |
La frecuencia y gravedad de los AA/AAG. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
End of Study of 5 years |
Fin de estudio de 5 años |
|
E.5.2 | Secondary end point(s) |
Proportion of participants with clinical benefit as assessed by the investigator at scheduled visits |
La proporción de participantes con beneficio clínico según el criterio del investigador en las visitas programadas. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
End of Study of 5 years |
Fin de estudio de 5 años |
|
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 | 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 | 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) | No |
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 | Yes |
E.8.1.7.1 | Other trial design description |
Estudio de extensión, los pacientes que reciban bosutinib tienen la opción de cambiar a asciminib |
Roll-over study, Bosutinib patients have the option to switch to asciminib during the study |
|
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.8.2.4 | Number of treatment arms in the trial | 7 |
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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 31 |
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 |
Brazil |
Canada |
China |
Japan |
Korea, Republic of |
Lebanon |
Mexico |
Russian Federation |
Saudi Arabia |
Taiwan |
Turkey |
United States |
Austria |
Bulgaria |
Denmark |
France |
Germany |
Italy |
Netherlands |
Poland |
Portugal |
Romania |
Spain |
United Kingdom |
Czechia |
Argentina |
|
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
|
Study is expected to remain open for 5 years or until one of the below scenario occurs for all participants, whichever comes first: •ongoing treatment becomes available following product launch and/or subsequent reimbursement (where applicable) •benefit-risk profile of ongoing treatment for participant is no longer positive •participant meets treatment discontinuation criteria •other access program becomes available or is identified (i.e. compassionate use, MAP, etc.) for ongoing treatment |
Se prevé que el estudio esté abierto durante 5 años o hasta que se produzca uno de los siguientes casos y afecte a todos los participantes, lo que suceda primero: • El tratamiento en curso se encuentra disponible tras el lanzamiento del producto o reembolso posterior (cuando corresponda). • El perfil beneficio-riesgo del tratamiento en curso para el participante ya no es positivo. • El participante cumple los criterios de discontinuación del tratamiento. Para más información ver protocolo. |
|
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 | 8 |
E.8.9.1 | In the Member State concerned days | 28 |
E.8.9.2 | In all countries concerned by the trial years | 5 |