Chronic Myeloid Leukemia in Chronic Phase
Conditions
Keywords
CML-CP, Asciminib
Brief summary
This is a multicenter, non-interventional real-world study designed to assess the efficacy and safety of asciminib in patients with newly diagnosed CML.The study uses a prospective data collection design to gather baseline, pre- and post-treatment, and long-term follow-up data, enabling a comprehensive assessment of asciminib's clinical benefits.
Interventions
None listed
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 99 Years
Healthy volunteers
No
Inclusion criteria
Patients eligible for inclusion in this study must meet all the following criteria: 1. 18 years or older at the time of ICF signing; 2. Newly diagnosed with Ph+ CML-CP within 3 months before enrollment; \- The diagnosis documentation must include the type and quantitative level of the BCR-ABL1 transcript. 3. Prior treatment with a maximum of 2 weeks of TKIs; 4. Prior treatment with non-TKI regimens, including interferon and hydroxyurea, is allowed; 5. Patients scheduled to initiate treatment with asciminib; \- Patients beginning asciminib treatment must receive the first dose within 14 days of signing the ICF; 6. Signed ICF.
Exclusion criteria
Patients meeting any of the following criteria are not eligible for inclusion in this study: 1. Previous diagnosis of CML-accelerated phase or blast crisis; 2. Currently participating in an interventional clinical study for CML; 3. Having rare, atypical transcript types that cannot be standardised internationally; 4. Women who are pregnant, lactating or planning to become pregnant during the study; 5. Concurrent other malignancies (refer to the International ICD-11 diagnosis codes, with diagnostic text including carcinoma, malignant neoplasm, etc.); 6. Other conditions that are considered not suitable for the study by the investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Cumulative rate of MMR | Month 12 | Cumulative rate of major molecular response (MMR) (BCR-ABL1 transcript level ≤ 0.1%) in patients receiving asciminib for 12 months |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Cumulative rate of MMR | Month 3, Month 6, Month 9, Month 18 and Month 24 | The cumulative MMR is defined as at least one BCR::ABL1 transcript level ≤ 0.1% during the follow-up period |
| Cumulative rate of deep molecular response (DMR): MR4 and MR4.5 | Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24 | Cumulative rate of MR4 is defined as at least one BCR::ABL1 transcript level ≤ 0.01% during the follow-up period; Cumulative MR4.5 is defined as at least one BCR::ABL1 transcript level ≤0.0032% during the follow-up period |
| Cumulative rate of complete cytogenetic response (CCyR) | Month 3, Month 6, and Month 12 | Cumulative CCyR is defined as at least one cytogenetic examination showing 0 Ph+ cells during the follow-up period |
| Time to first MMR | 24 month follow up period | Time to first MMR is defined as the time interval from baseline to the first occurrence of BCR::ABL1 transcript level ≤0.1% during the follow-up period |
| Time to first MR4 and MR4.5 | 24 month follow up period | Time to first MR4 is defined as the time interval from baseline to the first occurrence of BCR::ABL1 transcript level ≤0.01% during the follow-up period. Time to first MR4 is defined as the time interval from baseline to the first occurrence of BCR::ABL1 transcript level ≤0.0032% during the follow-up period |
| Time to first complete cytogenetic response (CCyR) | 24 month follow up period | Time to first CCyR is defined as the time interval from baseline to the first occurrence of CCyRduring the follow-up period |
| Early molecular response (EMR) rate at 3 months | Month 3 | Proportion of patients achieving BCR::ABL1 transcript level ≤10% at M3 |
| Complete hematologic response (CHR) rate | Month 1, Month 2, and Month 3 | the proportion of patients achieving white blood cell count \< 10 × 109/L, platelet count \< 450 × 109/L, no immature myeloid cells in peripheral blood, peripheral blood basophil percentage \< 5%, absence of symptoms/signs of extramedullary involvement, and non-palpable spleen at M1, M2, and M3 |
| Rate of decline in BCR::ABL1 transcript levels | Baseline, Month 1, Month 2, Month 3, Month 6, Month 9 and Month 12 | indirectly calculated from BCR::ABL1 transcript levels at baseline, M1, M2, M3, M6, M9, and M12; determined by BCR::ABL1 halving time |
| Duration of MMR | 24 month follow up period | Definition of duration: Duration = sum of "intervals"; Interval calculation method: the first visit at which a response is achieved (and previously unmeasured) within the interval is taken as the starting point, then evaluated sequentially in time order; the calculation stops upon encountering a visit that fails to meet the criterion; The duration is calculated as the time interval between the first and last visit at which the response was achieved; Missing visit data points during this period will be defaulted as achieving the response. |
| Duration of MR4 and MR4.5 | 24 month follow-up period | — |
| Event-Free survival (EFS) rate | Month 12 and Month 24 | Lack of efficacy: * Failure to achieve expected molecular response: failure to reach the molecular response milestones recommended by the ELN guidelines at prespecified time points; * Loss of molecular response: loss of confirmed MMR following achievement of MMR, or loss of CCyR. Disease progression: progression from the chronic phase to the accelerated phase or blast crisis. Death: fatal outcome due to any cause during the treatment period. Treatment discontinuation due to adverse events: permanent discontinuation caused by intolerable toxicity, serious adverse events, or other safety-related reasons associated with the study drug. |
| Progression-free survival (PFS) rate | Month 12 and Month 24 | — |
| Overall survival (OS) rate | Month 12 and Month 24 | — |
| Adverse events (AEs) and serious adverse events (SAEs) occurring during asciminib treatment | 24 month follow-up period | — |
| Asciminib persistence rates | Month 6, Month 12, and Month 24 | — |
| Rates of asciminib treatment interruption/dose reduction and discontinuation due to AEs | 24 month follow-up period | — |
| Proportion of patients requiring concomitant medications due to AEs | 24 month follow-up period | — |
| Change in the simplified CML quality of life questionnaire from baseline | Baseline, Month 12 and Month 24 | — |
| Compliance following asciminib treatment | 24 month follow-up period | Medication possession ratio |
| Concomitant medication use associated with AEs related to asciminib | 24 month follow-up period | — |
| Hospitalization rate, outpatient visit rate, emergency department visit rate, and ICU admission rate related to CML treatment | 24 month follow-up period | — |
| Gene mutation rate associated with asciminib treatment | 24 month follow-up period | Gene mutation rate associated with asciminib treatment; types and proportions of positive gene mutations identified via genetic testing. |
Countries
China
Contacts
CONTACTNovartis Pharmaceuticals
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed