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

Официальное название

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

Обзор

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).

Первичные конечные точки

• Percentage of Patients With Major Molecular Response (MMR) at 12 Months [Срок оценки: Month 12]

Вторичные конечные точки (12)

• Percentage of Patients by Clinical Characteristic [Срок оценки: Baseline]
• Percentage of Patients by Reason for TKI Treatment Decision Documented by the Treating Physician [Срок оценки: Baseline]
• Percentage of Patients With Dose Reduction by Reason for Dose Reduction [Срок оценки: 3, 6, 9, 12, 15, 18, 21, and 24 months]
• Percentage of Patients With Treatment Interruption by Reason for Interruption [Срок оценки: 3, 6, 9, 12, 15, 18, 21, and 24 months]
• Percentage of Patients Who Discontinued Treatment by Reason for Discontinuation [Срок оценки: 3, 6, 9, 12, 15, 18, 21, and 24 months]
• Time to Treatment Discontinuation [Срок оценки: 3, 6, 9, 12, 15, 18, 21, and 24 months]
• Time to Treatment Interruption [Срок оценки: 3, 6, 9, 12, 15, 18, 21, and 24 months]
• Time to Dose Reduction [Срок оценки: 3, 6, 9, 12, 15, 18, 21, and 24 months]
• Time to Treatment Discontinuation due to Adverse Events (TTDAE) [Срок оценки: 3, 6, 9, 12, 15, 18, 21, and 24 months]
• Percentage of Patients With Early Molecular Response (EMR) at 3 Months [Срок оценки: Month 3]
• Percentage of Patients With Molecular Response 2 (MR2) [Срок оценки: 3, 6, 9, 12, 15, 18, 21, and 24 months]
• Percentage of Patients With MMR [Срок оценки: 3, 6, 9, 15, 18, 21, and 24 months]

Критерии участия

Критерии включения

• Patients who provide written informed consent to participate in the study.
• Adult patients (≥18 years of age) with a confirmed diagnosis of Ph+ CML-CP.
• 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.
• 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.
• Patients willing to participate in routine follow-up visits and complete patient-reported outcome questionnaires over the course of the study.

Критерии исключения

• 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).
• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Германия · 54 центра

• Novartis Investigative Site — Baden-Baden
• Novartis Investigative Site — Heidenheim
• Novartis Investigative Site — Schwäbisch Hall
• Novartis Investigative Site — Aschaffenburg
• Novartis Investigative Site — Augsburg
• Novartis Investigative Site — Donauwörth
• Novartis Investigative Site — Herrsching am Ammersee
• Novartis Investigative Site — Kempten (Allgäu)
• … и ещё 46 центров

Идентификаторы

NCT: NCT07549516 · CABL001J1DE02

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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