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)

Novartis

Status: Enrolling

Conditions: Leukemia, Chronic Myeloid

Study type: Observational

Funder types: Industry

Identifiers

NCT07549516

CABL001J1DE02

Details and patient eligibility

About

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).

Enrollment: 380 estimated patients

Sex: All

Ages: 18 to 100 years old

Volunteers: No Healthy Volunteers

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.

Trial design

380 participants in 3 patient groups

Asciminib Cohort

Description:

Adult patients with Ph+ CML-CP, either newly diagnosed or previously treated with one TKI, who are treated with asciminib.

Imatinib Cohort

Description:

Adult patients newly diagnosed with Ph+ CML-CP treated with imatinib who have not received any prior TKI treatment.

Second-generation TKI Cohort

Description:

Adult patients newly diagnosed with Ph+ CML-CP treated with dasatinib, bosutinib, or nilotinib who have not received any prior TKI treatment.