ASSURE-3: Asciminib Prospective Non Interventional Study as 3rd Line Therapy or More to Treat Adult Patients With CML- CP in Real World Setting in France

Sponsor

Novartis Pharmaceuticals (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT06092879

Collaborator

(none)

168

Enrollment

Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

The purpose of this study is to enhance the knowledge on asciminib treatment in a broader and real-life population by collecting additional data to characterize the treatment patterns of patients treated with asciminib, with a primary objective represented by maintenance on treatment at 12 months.

Condition or Disease	Intervention/Treatment	Phase
Chronic Myeloid Leukemia	Other: Asciminib

Detailed Description

The ASSURE-3 study is a national, multicentric, non-interventional, prospective study in real-life conditions with primary data collection in adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) previously treated with two or more Tyrosine Kinase Inhibitors (TKIs). It will be conducted in France with hematologists, onco-hematologists, physicians with documented involvement in managing Ph+ CML-CP patients in routine practice, practicing in public or private health care institutions. Each patient will be followed during 15 months at M0, M1 and then every 3 months (rhythm of visits according to the routine clinical care), or until premature discontinuation of asciminib treatment.

Historical data will be abstracted retrospectively by the participating physicians from patient files, to collect information using an electronic case report form (eCRF). Primary data will be collected during inclusion and follow-up visits

Study Design

Study Type:

Observational

Anticipated Enrollment :

168 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Scemblix® (Asciminib): Prospective Non Interventional Study as 3rd Line Therapy or More to Treat Adult Patients With CML-CP in Real World Setting in France

Anticipated Study Start Date :

Dec 15, 2023

Anticipated Primary Completion Date :

Sep 15, 2026

Anticipated Study Completion Date :

Sep 15, 2026

Arms and Interventions

Arm	Intervention/Treatment
Asciminib Adult patients with Ph+ CML-CP previously treated with two or more tyrosine kinase inhibitors	Other: Asciminib There is no treatment allocation. Patients administered Asciminib by prescription will be enrolled Other Names: Scemblix

Arm

Intervention/Treatment

Asciminib

Adult patients with Ph+ CML-CP previously treated with two or more tyrosine kinase inhibitors

Other: Asciminib

There is no treatment allocation. Patients administered Asciminib by prescription will be enrolled

Other Names:

Scemblix

Outcome Measures

Primary Outcome Measures

Proportion of patients remaining on asciminib at 12 months [Month 12]
Proportion of patients remaining on asciminib treatment at 12 months to be provided

Secondary Outcome Measures

Proportion of patients in major molecular response (MMR) - for patients not in MMR at treatment initiation [12 months]
Molecular response to treatment: Response to treatment (MMR, BCR::ABL1 ≤ 0.1% on the International Scale, IS) at 12 months (+/- 1 month) for patients not in MMR at treatment initiation
Proportion of patients in MMR - for patients in MMR at treatment initiation [12 months]
Molecular response to treatment: Maintenance of molecular response (MMR, BCR::ABL1 ≤ 0.1% on the International Scale, IS) at 12 months (+/- 1 month) for patients in MMR at treatment initiation
Proportion of patients in MR2, MMR, MR4.0, MR4.5, uMR4.5 - for all patients [3 months, 6 months, 9 months, 12 months and 15 months]
Molecular response to treatment: Kinetics of response: MR2, MMR, MR4.0, MR4.5, undetectable MR4.5 for all patients. MR2, MR4, MR4.5 are defined as the transcript ratio of BCR-ABL1/ABL1 being 1% or less, 0.01% or less and 0.0032% or less respectively
BCR::ABL1 on the International Scale (IS) kinetics along treatment - for all patients [15 months]
Kinetics of response: BCR::ABL1 on the International Scale (IS) for all patients
Time to MMR from the index date - for patients not in MMR at treatment initiation [Up to 15 months]
Kinetics of response: Time to MMR for patients not in MMR at treatment initiation
Time from the first MMR to the first loss of MMR - for patients not in MMR at treatment initiation [Up to 15 months]
Molecular response to treatment: Duration of MMR for patients not in MMR at treatment initiation
Event-Free Surviva (EFS) [Up to 15 months]
EFS: time from index date to occurrence of one the events listed among variables: lack of efficacy, i.e., BRC::ABL1>1% or loss of CCyR if assessed disease progression (CML-AP/BP, CML death) death from any cause definitive treatment discontinuation due to any reason
Progression-Free Survival (PFS) [Up to 15 months]
PFS: time from index date to occurrence of one of the progression markers listed among variables: disease progression (CML-AP/BP, CML death), death from any cause, definitive treatment discontinuation due to any reason
Comorbidity profile [Baseline]
Charlson comorbidity index (CCI) at index date
Disease characteristics [Baseline]
Disease characteristics to be provided
History of TKI treatment [Baseline]
History of TKI treatment to be provided
Management of patients in real-life [15 months]
Concomitant medications
Exposure patterns to asciminib [15 months]
Exposure patterns to be provided
EORTC QLQ-C30 (European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30) [Baseline, 3months, 6months, 9months, 12months, 15months]
The EORTC QLQ-C30 contains 30 questions assessed by the participant. 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 two questions have 4-point scales ranging from "Not at all" to "Very much." The two 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.
EORTC QLQ-CML24 questionnaire [Baseline, 3months, 6months, 9months, 12months, 15months]
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-CML 24 is composed of four multi-item scales and two single-item scales. The module consists of 24 items assessing symptoms 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 were measured on four levels: 1=not at all, 2=a little, 3=quite a bit, 4=very much. For each domain, scores were averaged and transformed to 0 to 100. A higher score in satisfaction with social life domain indicates a higher level of satisfaction. A positive change from baseline indicates increasing satisfaction.
Emergence of mutations [Up to 15 months]
If available at end of treatment: mutational analysis, methods and results
Proportion of patient with an AE described during asciminib treatment among patient reporting the same AE which led to discontinuation with previous TKI treatments [Up to 15 months]
Cross intolerance with previous TKI treatments

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 99 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Patient aged ≥ 18 years at inclusion,
Patient with Ph+ CML-CP previously treated with two or more TKIs,
Patient for whom a decision has been taken by the treating physician (investigator) to initiate treatment with asciminib according to his own practice, the drug label / Summary of Product Characteristics (SmPC), and regardless of study participation,
Patient having given their non objection to participate to the study