Real-world Study of Scemblix in the Treatment of Chronic Myeloid Leukemia in China (ASC4CN)

Explore the Effectiveness and Safety of Scemblix (Asciminib) for Newly Diagnosed CML-CP Patients in China Real World Setting (ASC4CN)

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.

Study Overview

• Status: Recruiting
• Conditions: Chronic Myeloid Leukemia in Chronic Phase

• Study Type: Observational
• Enrollment (Estimated): 200

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: +41613241111; Email: novartis.email@novartis.com
• Study Contact Backup: Name: Novartis Pharmaceuticals

Study Locations

• China
  • Hubei
    • Wuhan, Hubei, China, 430022
      • Recruiting
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

patients newly diagnosed with CML-CP and confirmed with Ph+ CML in medical records

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Study Group; Asciminib treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cumulative rate of MMR	Cumulative rate of major molecular response (MMR) (BCR-ABL1 transcript level ≤ 0.1%) in patients receiving asciminib for 12 months	Month 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cumulative rate of MMR	The cumulative MMR is defined as at least one BCR::ABL1 transcript level ≤ 0.1% during the follow-up period	Month 3, Month 6, Month 9, Month 18 and Month 24
Cumulative rate of deep molecular response (DMR): MR4 and MR4.5	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	Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24
Cumulative rate of complete cytogenetic response (CCyR)	Cumulative CCyR is defined as at least one cytogenetic examination showing 0 Ph+ cells during the follow-up period	Month 3, Month 6, and Month 12
Time to first MMR	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	24 month follow up period
Time to first MR4 and MR4.5	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	24 month follow up period
Time to first complete cytogenetic response (CCyR)	Time to first CCyR is defined as the time interval from baseline to the first occurrence of CCyRduring the follow-up period	24 month follow up period
Early molecular response (EMR) rate at 3 months	Proportion of patients achieving BCR::ABL1 transcript level ≤10% at M3	Month 3
Complete hematologic response (CHR) rate	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	Month 1, Month 2, and Month 3
Rate of decline in BCR::ABL1 transcript levels	indirectly calculated from BCR::ABL1 transcript levels at baseline, M1, M2, M3, M6, M9, and M12; determined by BCR::ABL1 halving time	Baseline, Month 1, Month 2, Month 3, Month 6, Month 9 and Month 12
Duration of MMR	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.	24 month follow up period
Duration of MR4 and MR4.5		24 month follow-up period
Event-Free survival (EFS) rate	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.	Month 12 and Month 24
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	Medication possession ratio	24 month follow-up period
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	Gene mutation rate associated with asciminib treatment; types and proportions of positive gene mutations identified via genetic testing.	24 month follow-up period

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): February 12, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2028

Study Registration Dates

• First Submitted: January 21, 2026
• First Submitted That Met QC Criteria: January 21, 2026
• First Posted (Actual): January 29, 2026

Study Record Updates

• Last Update Posted (Actual): April 1, 2026
• Last Update Submitted That Met QC Criteria: March 27, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: Asciminib; CML-CP
• Other Study ID Numbers: CABL001J1CN01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO