Special Drug Use-results Surveillance of Scemblix Tablets

Study Overview
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Brief Summary
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Uncontrolled, central registration system, all-case, multicenter, special drug use-results surveillance.

Detailed Description
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The objective of this study is to collect data on the occurrence, severity, clinical courses of the safety specifications of asciminib, identify factors etc. involved in occurrence and assess its clinical safety inresistant/intolerant chronic myelogenous leukemia patients during an observational period of 48 weeks from the start of treatment with asciminib.

Official Title
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Special Drug Use-results Surveillance of Scemblix Tablets (Resistant or Intolerant Chronic Myeloid Leukemia , CABL001A1401)

Conditions
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Chronic Myeloid Leukemia

Intervention / Treatment
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* Other: Asciminib


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: patients treated with asciminib in Japan. Exclusion Criteria: NA

Ages Eligible for Study
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Maximum Age: 99 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Asciminib<br>Patients prescribed with Asciminib | Other: Asciminib<br>* Prospective observational study. There is no treatment allocation. Patients prescribed with asciminib are eligible to enroll into this study.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Type, frequency, seriousness and severity of adverse event (AE)/treatment-related AE of the safety specifications | For the safety specifications (myelosuppression, infections, QT interval prolongation, pancreatitis, vascular occlusive events, photosensitivity), type, frequency AE, seriousness, severity of adverse event (AE)/treatment-related AE will be collected | Up to 48 Weeks | 
| AEs leading to interruption/discontinuation of the safety specifications | For the safety specifications (myelosuppression, infections, QT interval prolongation, pancreatitis, vascular occlusive events, photosensitivity), AEs leading to interruption/discontinuation will be collected | Up to 48 Weeks | 
| Number of patients with changes in relevant laboratory parameters for the safety specifications | For the safety specifications (myelosuppression, infections, QT interval prolongation, pancreatitis, vascular occlusive events, photosensitivity), number of patients with changes in relevant laboratory parameters will be collected | Up to 48 Weeks | 
| Frequency of AEs/Treatment-related AEs by patient characteristic factor | Frequency of AEs/Treatment-related AEs by patient characteristic factor will be collected | Up to 48 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Type, frequency, seriousness, severity of AEs/treatment-related AEs of the safety analysis set | Type, frequency, seriousness, severity of AEs/treatment-related AEs of the safety analysis set will be collected | Up to 48 Weeks | 
| AEs leading to interruption/discontinuation in the safety analysis set | AEs leading to interruption/discontinuation in the safety analysis set will be collected | Up to 48 Weeks | 
| Frequency of AEs/treatment-related AEs summarized by patient characteristic factor | Frequency of AEs/treatment-related AEs summarized by patient characteristic factor will be collected | Up to 48 Weeks | 
| Type, frequency, seriousness, severity of AEs/treatment-related AEs in patients with special characteristics | Type, frequency, seriousness, severity of AEs/treatment-related AEs in patients with special characteristics (patients with concurrent renal impairment/hepatic impairment/cardiac impairment, elderly, children, pregnant/parturient women) will be collected | Up to 48 Weeks | 
| AEs leading to interruption/discontinuation in patients with special characteristics | AEs leading to interruption/discontinuation in patients with special characteristics (patients with concurrent renal impairment/hepatic impairment/cardiac impairment, elderly, children, pregnant/parturient women) will be collected | Up to 48 Weeks | 
| Type, frequency, seriousness, severity and outcome of AEs/treatment-related AEs by treatment line | Type, frequency, seriousness, severity and outcome of AEs/treatment-related AEs by treatment line will be collected | Up to 48 Weeks | 
| Factors affecting occurrence of AEs by treatment line | Factors affecting occurrence of AEs by treatment line will be collected | Up to 48 Weeks | 
| AEs leading to interruption/discontinuation by treatment line | AEs leading to interruption/discontinuation by treatment line will be collected | Up to 48 Weeks | 
| Major molecular response (MMR) rates | Major molecular response is defined as BCR-ABL1 International Scale value ≤ 0.1%. BCR-ABL1: translocation-produced fusion gene | Week 12, Week 24, Week 48 | 
| MMR rates by Week 48 by patient characteristics factor | Major molecular response (MMR) is defined as BCR-ABL1 International Scale value ≤ 0.1%. BCR-ABL1: translocation-produced fusion gene | Up to 48 Weeks | 
| MR4.0 and MR4.5 rates | MR4.0 and MR4.5 rates are defined as : MR4.0: BCR-ABL1 International Scale value ≤ 0.01% MR4.5: BCR-ABL1 International Scale value ≤ 0.0032% BCR-ABL1: translocation-produced fusion gene | Week 12, Week 24 and Week 48 | 
| Complete cytogenetic response (CCyR) rates | This study will collect complete cytogenetic response (CCyR), which is defined as a state of Ph+ metaphase cell disappearance, i.e. Ph+ cell = 0%. | Week 12, Week 24 and Week 48 | 
| Complete hematological response (CHR) rates | This study will collect complete hematological response (CHR), which is defined as meeting the following 6 criteria. White blood cell count < 10,000/µL Platelet count < 450,000/µL No blast cell and promyelocyte in peripheral blood Myelocyte + metamyelocyte in peripheral blood = 0% Basophil < 5% No spleen and liver swelling, and no extramedullary lesion | Week 12, Week 24 and Week 48 | 
| Rate of patients with BCR-ABL1 gene mutations | This study will collect the rate of patients with BCR-ABL1 gene mutations | Up to 48 Weeks | 
| MMR rates by Week 48 in patients with special characteristics | This study will collect major molecular response (MMR) rates by Week 48 in patients with special characteristics (patients with concurrent renal impairment/hepatic impairment/cardiac impairment, elderly, children, pregnant/parturient women) | Week 48 | 
| MMR rates by treatment line | This study will collect major molecular response (MMR) rates by treatment line | Week 12, Week 24 and Week 48 | 
| MR4.0 and MR4.5 rates by treatment line | MR4.0 and MR4.5 rates are defined as : MR4.0: BCR-ABL1 International Scale value ≤ 0.01% MR4.5: BCR-ABL1 International Scale value ≤ 0.0032% BCR-ABL1: translocation-produced fusion gene | Week 12, Week 24 and Week 48 | 
| CCyR rates by treatment line | This study will collect complete cytogenetic response (CCyR), which is defined as a state of Ph+ metaphase cell disappearance, i.e. Ph+ cell = 0%. | Week 12, Week 24 and Week 48 | 
| CHR rates by treatment line | This study will collect complete hematological response (CHR), which is defined as meeting the following 6 criteria. White blood cell count < 10,000/µL Platelet count < 450,000/µL No blast cell and promyelocyte in peripheral blood Myelocyte + metamyelocyte in peripheral blood = 0% Basophil < 5% No spleen and liver swelling, and no extramedullary lesion | Week 12, Week 24 and Week 48 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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chronic myeloid leukemia, CML, Scemblix Tablets, Asciminib, resistant or intolerant CML