A Study on the Tolerability, Safety and Effectiveness of Asciminib in Patients With Philadelphia Chromosome-positive Chronic Myeloid Leukemia in the Chronic Phase in Germany (ASC2ADHERE)

A Non-Interventional Study on the Tolerability, Safety and Effectiveness of Asciminib in Newly Diagnosed and Pre-treated Patients With Philadelphia Chromosome-positive Chronic Myeloid Leukemia in the Chronic Phase in Germany - the ASC2ADHERE Study

The aim of this study is to assess the real-world effectiveness of asciminib in Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) patients who were either newly diagnosed or previously treated with one ATP-competitive tyrosine kinase inhibitor (TKI).

Study Overview

• Status: Recruiting
• Conditions: Leukemia, Chronic Myeloid

• Study Type: Observational
• Enrollment (Estimated): 380

Contacts and Locations

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

Study Locations

• Germany
  • Bad Liebenwerda, Germany, 04924
    • Recruiting
    • Novartis Investigative Site
  • Berlin, Germany, 12487
    • Recruiting
    • Novartis Investigative Site
  • Buchholz Nordheide, Germany, 21244
    • Recruiting
    • Novartis Investigative Site
  • Cottbus, Germany, 03046
    • Recruiting
    • Novartis Investigative Site
  • Detmold, Germany, 32756
    • Recruiting
    • Novartis Investigative Site
  • Hanover, Germany, 30625
    • Recruiting
    • Novartis Investigative Site
  • Parchim, Germany, 19370
    • Recruiting
    • Novartis Investigative Site
  • Potsdam, Germany, 14467
    • Recruiting
    • Novartis Investigative Site
  • Schorndorf, Germany, 73614
    • Recruiting
    • Novartis Investigative Site
  • Stade, Germany, 21680
    • Recruiting
    • Novartis Investigative Site
  • Bavaria
    • Munich, Bavaria, Germany, 81241
      • Recruiting
      • Novartis Investigative Site
  • Free Hanseatic City of Bremen
    • Bremerhaven, Free Hanseatic City of Bremen, Germany, 27576
      • Recruiting
      • Novartis Investigative Site
  • Hesse
    • Offenbach, Hesse, Germany, 63065
      • Recruiting
      • Novartis Investigative Site
  • North Rhine-Westphalia
    • Cologne, North Rhine-Westphalia, Germany, 50677
      • Recruiting
      • Novartis Investigative Site
  • Saxony
    • Zittau, Saxony, Germany, 02763
      • 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

Adult Ph+ CML-CP patients, either newly diagnosed or previously treated with one ATP-competitive TKI, who initiate TKI treatment as recommended by the treating physician in a hematology center in Germany.

Description

Inclusion criteria:

1. Patients who provide written informed consent to participate in the study.
2. Adult patients (≥18 years of age) with a confirmed diagnosis of Ph+ CML-CP.
3. Patients who are either newly diagnosed or have received treatment with exactly one prior TKI. Prior TKI treatment is only permitted for patients in the Asciminib Cohort. Patients in the comparator cohorts (imatinib, dasatinib, bosutinib, nilotinib) must be newly diagnosed and must not have received any prior TKI treatment.
4. Patients for whom the treating physician has made a clinical decision to initiate treatment with asciminib or another TKI (imatinib, dasatinib, bosutinib, nilotinib) as part of routine care. The clinical decision for treatment must have been made prior to enrollment. Treatment must not have started more than 14 days before study inclusion, and treatment may also begin after baseline assessment.
5. Patients willing to participate in routine follow-up visits and complete patient-reported outcome questionnaires over the course of the study.

Exclusion criteria:

1. Patients with contraindications to their respective chronic myeloid leukemia (CML) treatment as per the applicable Summary of Product Characteristics (SmPC) and relevant national treatment guidelines (e.g. Onkopedia CML), including the following asciminib specific considerations:

   • In first- or second-line treatment: presence of BCR::ABL1 fusion transcripts lacking exon a2 (e.g. e13a3, e14a3).
   • In second-line treatment: known BCR::ABL1 mutations associated with partial or complete resistance to asciminib (e.g. M244V, F359I/V/C;T315I).
2. Patients receiving or planned to receive asciminib or other TKIs outside the approved label (off-label use), including use in unapproved dosing regimens or frequency not covered by the respective SmPC.
3. Patients currently participating in an interventional clinical trial.
4. Patients unable or unwilling to provide written informed consent.
5. Patients who are unable to reliably complete patient-reported outcome questionnaires due to cognitive or language limitations relevant to the study assessments.
6. Patients for whom long-term follow-up is not feasible due to expected relocation or other logistical constraints.

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort
Asciminib Cohort; Adult patients with Ph+ CML-CP, either newly diagnosed or previously treated with one TKI, who are treated with asciminib.
Imatinib Cohort; Adult patients newly diagnosed with Ph+ CML-CP treated with imatinib who have not received any prior TKI treatment.
Second-generation TKI Cohort; Adult patients newly diagnosed with Ph+ CML-CP treated with dasatinib, bosutinib, or nilotinib who have not received any prior TKI treatment.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Patients With Major Molecular Response (MMR) at 12 Months	MMR is defined as a BCR::ABL1 level ≤ 0.1% according to the International Scale (IS).	Month 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Patients by Clinical Characteristic	Characteristics include, but are not limited to, detection of Philadelphia chromosome or BCR::ABL1 transcript, prior treatment, and Eastern Cooperative Oncology Group (ECOG) performance status.	Baseline
Percentage of Patients by Reason for TKI Treatment Decision Documented by the Treating Physician		Baseline
Percentage of Patients With Dose Reduction by Reason for Dose Reduction		3, 6, 9, 12, 15, 18, 21, and 24 months
Percentage of Patients With Treatment Interruption by Reason for Interruption		3, 6, 9, 12, 15, 18, 21, and 24 months
Percentage of Patients Who Discontinued Treatment by Reason for Discontinuation		3, 6, 9, 12, 15, 18, 21, and 24 months
Time to Treatment Discontinuation		3, 6, 9, 12, 15, 18, 21, and 24 months
Time to Treatment Interruption		3, 6, 9, 12, 15, 18, 21, and 24 months
Time to Dose Reduction		3, 6, 9, 12, 15, 18, 21, and 24 months
Time to Treatment Discontinuation due to Adverse Events (TTDAE)		3, 6, 9, 12, 15, 18, 21, and 24 months
Percentage of Patients With Early Molecular Response (EMR) at 3 Months	EMR is defined as BCR::ABL1 ≤10% IS.	Month 3
Percentage of Patients With Molecular Response 2 (MR2)	MR2 is defined as BCR::ABL1 ≤1% IS.	3, 6, 9, 12, 15, 18, 21, and 24 months
Percentage of Patients With MMR	MMR is defined as BCR::ABL1 ≤0.1% IS.	3, 6, 9, 15, 18, 21, and 24 months
Percentage of Patients With Deep Molecular Response: MR4.0	MR4.0 is defined as BCR::ABL1 ≤0.01% IS.	3, 6, 9, 12, 15, 18, 21, and 24 months
Percentage of Patients With Deep Molecular Response: MR4.5	MR4.5 is defined as BCR::ABL1 ≤0.0032% IS.	3, 6, 9, 12, 15, 18, 21, and 24 months
Medication Adherence Report Scale (MARS-5) Score	The MARS-5 questionnaire is a validated self-report questionnaire developed to assess patient adherence to prescribed medication, focusing on both intentional and unintentional non-adherence. It consists of 5 items, each rated on a 5-point Likert Scale from 0 (always) to 5 (never). The total score ranges from 5 to 25, with higher scores indicating better adherence to treatment.	3, 6, 9, 12, 15, 18, 21, and 24 months
European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC QLQ-C30) Score	The EORTC QLQ-C30 contains 30 questions answered by the patient. There are 9 multiple-item scales: 5 scales that assess aspects of functioning (physical, role functioning, cognitive, emotional, and social); 3 symptom scales (fatigue, pain, and nausea and vomiting); and a global health status/Quality of Life (QOL) scale. There are 5 single-item measures assessing additional symptoms (i.e., dyspnea, loss of appetite, insomnia, constipation, and diarrhea) and a single item concerning perceived financial impact of the disease. All but 2 questions have 4-point scales ranging from "Not at all" to "Very much." The 2 questions concerning global health status/QOL have 7-point scales with ratings ranging from "Very poor" to "Excellent." For each of the 14 domains, final scores are transformed such that they range from 0-100, where higher scores indicate improvement.	3, 6, 9, 12, 15, 18, 21, and 24 months
European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire for CML (EORTC QLQ-CML24) Score	The EORTC QLQ-CML24 was designed to supplement the QLQ-C30 - the QLQ-CML24 is not a stand-alone instrument but is to be used in conjunction with the QLQ-C30. The EORTC QLQ-CML24 is composed of 4 multi-item scales and 2 single-item scales. The module consists of 24 items assessing symptom burden (13 items), impact on worry/mood (4 items), impact on daily life (3 items), satisfaction with care and information (2 items) body image problems (1 item) and satisfaction with social life (1 item). The items are measured on 4 levels: 1=not at all, 2=a little, 3=quite a bit, 4=very much. For each domain, scores are averaged and transformed to 0 to 100. A higher score in symptom burden, impact on worry/mood, impact on daily life, and body image problems domains indicates a worse outcome. A higher score in satisfaction with social life and satisfaction with care and information domains indicates a higher level of satisfaction.	3, 6, 9, 12, 15, 18, 21, and 24 months
Work Productivity and Activity Impairment - General Health (WPAI-GH) Score	The WPAI-GH is a patient-reported 6 item questionnaire which addresses absenteeism, presenteeism, overall work productivity loss, and activity impairment for the 7 days prior to the assessment. Work productivity and activity impairment outcomes are presented as impairment percentages ranging from 0 to 100 with a higher percentage indicating greater impairment and less productivity.	3, 6, 9, 12, 15, 18, 21, and 24 months

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): April 17, 2026
• Primary Completion (Estimated): September 30, 2030
• Study Completion (Estimated): September 30, 2030

Study Registration Dates

• First Submitted: April 16, 2026
• First Submitted That Met QC Criteria: April 16, 2026
• First Posted (Actual): April 24, 2026

Study Record Updates

• Last Update Posted (Actual): May 15, 2026
• Last Update Submitted That Met QC Criteria: May 13, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Adherence; Non-interventional study; Asciminib; Tyrosine kinase inhibitors; Germany; CML-CP; Real-world evidence; EORTC QLQ-C30; Philadelphia chromosome-positive; BCR::ABL1; MARS-5; Major Molecular Response; EORTC QLQ-CML24; WPAI-GH
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Chronic Disease; Disease Attributes; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia, Myeloid; Bone Marrow Diseases; Leukemia; Myeloproliferative Disorders; Pathological Conditions, Signs and Symptoms; Hemic and Lymphatic Diseases; Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Other Study ID Numbers: CABL001J1DE02

Plan for Individual participant data (IPD)

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