Asciminib Prospective Non Interventional Study as 3rd Line Therapy or More to Treat Adult Patients With CML- CP in Real World Setting in France
- Conditions
- Chronic Myeloid Leukemia
- Interventions
- Other: Asciminib
- Registration Number
- NCT06092879
- Lead Sponsor
- Novartis Pharmaceuticals
- 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.
- 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
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 168
- 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
- Patient with CML in accelerated phase (AP) or blastic phase (BP) at enrolment,
- Patient with known history of T315I mutation,
- Patient who previously received asciminib treatment,
- Patient currently participating to an interventional clinical trial,
- Patient with known contra-indication to asciminib according to the SmPC.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Asciminib Asciminib Adult patients with Ph+ CML-CP previously treated with two or more tyrosine kinase inhibitors
- Primary Outcome Measures
Name Time Method 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
Name Time Method 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 reasonComorbidity 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
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
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
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 reasonExposure patterns to asciminib 15 months Exposure patterns to be provided
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
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
Management of patients in real-life 15 months Concomitant medications
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.
Emergence of mutations Up to 15 months If available at end of treatment: mutational analysis, methods and results
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 respectivelyTime 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
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.
Trial Locations
- Locations (1)
Novartis Investigative Site
🇫🇷Vesoul, France