Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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).
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
Not provided
| 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 |
| 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 |
Not provided
Inclusion criteria:
Exclusion criteria:
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:
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.
Patients currently participating in an interventional clinical trial.
Patients unable or unwilling to provide written informed consent.
Patients who are unable to reliably complete patient-reported outcome questionnaires due to cognitive or language limitations relevant to the study assessments.
Patients for whom long-term follow-up is not feasible due to expected relocation or other logistical constraints.
Not provided
Not provided
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.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Novartis Pharmaceuticals | Contact | +41613241111 | novartis.email@novartis.com | |
| Novartis Pharmaceuticals | Contact |
| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Recruiting | Schwäbisch Hall | Baden-Wurttemberg | 74523 | Germany | |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| 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 |
| Novartis Investigative Site |
| Recruiting |
| Augsburg |
| Bavaria |
| 86152 |
| Germany |
| Novartis Investigative Site | Recruiting | Herrsching am Ammersee | Bavaria | 82211 | Germany |
| Novartis Investigative Site | Recruiting | Kempten (Allgäu) | Bavaria | 87439 | Germany |
| Novartis Investigative Site | Recruiting | Munich | Bavaria | 81241 | Germany |
| Novartis Investigative Site | Recruiting | Regensburg | Bavaria | 93053 | Germany |
| Novartis Investigative Site | Recruiting | Bremerhaven | Free Hanseatic City of Bremen | 27576 | Germany |
| Novartis Investigative Site | Recruiting | Kassel | Hesse | 34125 | Germany |
| Novartis Investigative Site | Recruiting | Offenbach | Hesse | 63065 | Germany |
| Novartis Investigative Site | Recruiting | Rotenburg (Wümme) | Lower Saxony | 27356 | Germany |
| Novartis Investigative Site | Recruiting | Cologne | North Rhine-Westphalia | 50674 | Germany |
| Novartis Investigative Site | Recruiting | Cologne | North Rhine-Westphalia | 50677 | Germany |
| Novartis Investigative Site | Recruiting | Solingen | North Rhine-Westphalia | 42653 | Germany |
| Novartis Investigative Site | Recruiting | Zittau | Saxony | 02763 | Germany |
| Novartis Investigative Site | Recruiting | Merseburg | Saxony-Anhalt | 06217 | Germany |
| Novartis Investigative Site | Recruiting | Oldenburg in Holstein | Schleswig-Holstein | 23758 | Germany |
| Novartis Investigative Site | Recruiting | Saalfeld | Thuringia | 07318 | Germany |
| Novartis Investigative Site | Recruiting | Bad Liebenwerda | 04924 | Germany |
| Novartis Investigative Site | Recruiting | Berlin | 12487 | Germany |
| Novartis Investigative Site | Recruiting | Bremerhaven | 27568 | Germany |
| Novartis Investigative Site | Recruiting | Buchholz Nordheide | 21244 | Germany |
| Novartis Investigative Site | Recruiting | Cottbus | 03046 | Germany |
| Novartis Investigative Site | Recruiting | Detmold | 32756 | Germany |
| Novartis Investigative Site | Recruiting | Dorfen | 84405 | Germany |
| Novartis Investigative Site | Recruiting | Dortmund | 44263 | Germany |
| Novartis Investigative Site | Recruiting | Dresden | 01307 | Germany |
| Novartis Investigative Site | Recruiting | Düren | 52353 | Germany |
| Novartis Investigative Site | Recruiting | Hanover | 30161 | Germany |
| Novartis Investigative Site | Recruiting | Hanover | 30625 | Germany |
| Novartis Investigative Site | Recruiting | Hildesheim | 31135 | Germany |
| Novartis Investigative Site | Recruiting | Mutlangen | 73557 | Germany |
| Novartis Investigative Site | Recruiting | Naunhof | 04683 | Germany |
| Novartis Investigative Site | Recruiting | Neuss | 41462 | Germany |
| Novartis Investigative Site | Recruiting | Oldenburg | 26121 | Germany |
| Novartis Investigative Site | Recruiting | Parchim | 19370 | Germany |
| Novartis Investigative Site | Recruiting | Potsdam | 14467 | Germany |
| Novartis Investigative Site | Recruiting | Potsdam | 14482 | Germany |
| Novartis Investigative Site | Recruiting | Schorndorf | 73614 | Germany |
| Novartis Investigative Site | Recruiting | Soest | 59494 | Germany |
| Novartis Investigative Site | Recruiting | Stade | 21680 | Germany |
| Novartis Investigative Site | Recruiting | Wetzlar | 35578 | Germany |
| ID | Term |
|---|---|
| D015464 | Leukemia, Myelogenous, Chronic, BCR-ABL Positive |
| ID | Term |
|---|---|
| D007951 | Leukemia, Myeloid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009196 | Myeloproliferative Disorders |
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided