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) |
CML-CP (alla fine dello studio degli adulti) che stanno attualmente partecipando a uno studio sponsorizzato da Novartis con asciminb (studio degli adulti) |
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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 mieloide cronica è un cancro del sangue caratterizzato da una mutazione genetica (cromosoma Philadelphia) che causa la proliferazione dei globuli bianchi nel midollo osseo. |
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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 | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10009013 |
E.1.2 | Term | Chronic myeloid 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 | 24.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10082178 |
E.1.2 | Term | Philadelphia positive chronic myeloid leukemia |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 24.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10082180 |
E.1.2 | Term | Philadelphia positive chronic myeloid leukaemia |
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 |
The primary objective of this study is to assess long term safety data (i.e SAEs and AEs) |
L'obiettivo primario di questo studio è quello di valutare i dati di sicurezza a lungo termine (cioè SAEs e AEs) |
|
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. |
L'obiettivo secondario di questo studio è quello di valutare il beneficio clinico in corso come valutato dallo sperimentatore. |
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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. Il consenso informato firmato deve essere ottenuto prima della partecipazione allo studio. 2. Il partecipante con CML-CP (secondo le linee guida ELN 2013) è attualmente in trattamento con asciminib (o asciminib in combinazione con imatinib, o imatinib da solo o nilotinib da solo per i partecipanti CABL001E2201, o bosutinib per i partecipanti CABL001A2301) all'interno di uno studio sponsorizzato da Novartis, che ha soddisfatto i requisiti per l'obiettivo primario e ha completato la fase di trattamento e, a giudizio dello sperimentatore, trarrebbe beneficio dal proseguimento del trattamento. 3. Il partecipante ha dimostrato la conformità, come valutato dallo sperimentatore, con i requisiti del protocollo dello studio madre ed è disposto e in grado di rispettare le visite programmate, i piani di trattamento e qualsiasi altra procedura dello studio. 4. Possono essere applicati altri criteri di inclusione definiti dal protocollo |
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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. Il partecipante è stato interrotto dal trattamento dello studio dei genitori per qualsiasi motivo. 2. Il partecipante ha attualmente tossicità irrisolte di grado 3 o 4 segnalate come possibilmente correlate al trattamento nello studio di riferimento, che: - non si sono risolte al grado 2 o inferiore entro 42 giorni e / o richiedono l'interruzione della dose per più di 42 giorni per tossicità ematologiche, - non si sono risolti al grado 2 o inferiore entro 28 giorni e / o richiedono l'interruzione della dose per più di 28 giorni in caso di tossicità non ematologiche. 3. Il trattamento in corso del partecipante è attualmente approvato e rimborsato a livello nazionale. 4. Donne incinte o che allattano. 5. Donne in età fertile, definite come tutte le donne fisiologicamente in grado di rimanere incinte, a meno che non stiano usando metodi contraccettivi altamente efficaci durante il trattamento dello studio e per 3 giorni dopo l'interruzione di asciminib, 14 giorni dopo l'interruzione di imatinib o nilotinib e un mese dopo l'interruzione del farmaco bosutinib. 6. Solo per i partecipanti negli USA e in trattamento con asciminib: Maschi sessualmente attivi non disposti a usare il preservativo durante i rapporti sessuali mentre assumono il trattamento dello studio e per 3 giorni dopo l'interruzione del trattamento dello studio. Inoltre, i partecipanti maschi non devono donare lo sperma per il periodo di tempo specificato sopra. 7. Applicabile solo ai partecipanti in trattamento con bosutinib alla fine dello studio ABL001A2301 che passano al trattamento con asciminib all'arruolamento: -Pancreatite asintomatica (grado 2) se non risolta entro 28 giorni. -QTcF > 480 msec o incapacità di determinare l'intervallo QTc 8. Possono essere applicati altri criteri di esclusione definiti dal protocollo |
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E.5 End points |
E.5.1 | Primary end point(s) |
The frequency and severity of AEs/SAEs |
La frequenza e la gravità degli AEs/SAEs |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
End of Study of 5 years |
Fine dello studio di 5 anni |
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E.5.2 | Secondary end point(s) |
Proportion of participants with clinical benefit as assessed by the investigator at scheduled visits |
Percentuale di partecipanti con beneficio clinico valutato dallo sperimentatore alle visite programmate |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
End of Study of 5 years |
Fine dello studio di 5 anni |
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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 | 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 | No |
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 |
Aperto, i pazienti trattati con Bosutinib hanno la possibilità di passare ad asciminib durante lo s |
Open, 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 | 3 |
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 | Information not present in EudraCT |
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 |
Brazil |
Canada |
China |
Japan |
Korea, Republic of |
Lebanon |
Mexico |
Russian Federation |
Saudi Arabia |
Taiwan |
Turkey |
United States |
Bulgaria |
France |
Germany |
Italy |
Netherlands |
Poland |
Romania |
Spain |
United Kingdom |
Czechia |
Argentina |
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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
|
Study is expected to remain open for 5 years or until one of the below scenario occurs, 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 for ongoing treatment |
Lo studio ci si aspetta che rimarrà aperto per 5 anni o finché non si verifichi uno dei seguenti scenari, a seconda di quale si verifichi per primo: -il trattamento in corso diventa disponibile dopo il lancio del prodotto e/o il successivo rimborso (se applicabile) -il profilo beneficio-rischio del trattamento in corso non è più positivo -il partecipante soddisfa i criteri di interruzione -si rende disponibile o viene identificato un altro programma di accesso per il trattamento in corso |
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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 | 5 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 5 |
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 |