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

Chronic Myeloid Leukemia Clinical Trial

— ASSURE-3  

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06092879
• Other study ID #: CABL001AFR04
• Status: Recruiting
• Start date: March 6, 2024
• Est. completion date: December 15, 2026

Study information

• Verified date: May 2024
• Source: Novartis
• Contact: Novartis Pharmaceuticals
• Phone: +41613241111
• Email: novartis.email[@]novartis.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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 information / eligibility

• Status: Recruiting
• Enrollment: 168
• Est. completion date: December 15, 2026
• Est. primary completion date: December 15, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 99 Years

Eligibility

Inclusion Criteria:

1. Patient aged = 18 years at inclusion,

2. Patient with Ph+ CML-CP previously treated with two or more TKIs,

3. 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,

4. Patient having given their non objection to participate to the study

Exclusion Criteria:

1. Patient with CML in accelerated phase (AP) or blastic phase (BP) at enrolment,

2. Patient with known history of T315I mutation,

3. Patient who previously received asciminib treatment,

4. Patient currently participating to an interventional clinical trial,

5. Patient with known contra-indication to asciminib according to the SmPC.

Related Conditions & MeSH terms

• Chronic Myeloid Leukemia
• Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Leukemia, Myeloid

Intervention

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

Locations

Country	Name	City	State
France	Novartis Investigative Site	Bordeaux
France	Novartis Investigative Site	Mulhouse cedex

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of patients remaining on asciminib at 12 months	Proportion of patients remaining on asciminib treatment at 12 months to be provided	Month 12
Secondary	Proportion of patients in major molecular response (MMR) - for patients not in MMR at treatment initiation	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	12 months
Secondary	Proportion of patients in MMR - for patients in MMR at treatment initiation	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	12 months
Secondary	Proportion of patients in MR2, MMR, MR4.0, MR4.5, uMR4.5 - for all patients	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	3 months, 6 months, 9 months, 12 months and 15 months
Secondary	BCR::ABL1 on the International Scale (IS) kinetics along treatment - for all patients	Kinetics of response: BCR::ABL1 on the International Scale (IS) for all patients	15 months
Secondary	Time to MMR from the index date - for patients not in MMR at treatment initiation	Kinetics of response: Time to MMR for patients not in MMR at treatment initiation	Up to 15 months
Secondary	Time from the first MMR to the first loss of MMR - for patients not in MMR at treatment initiation	Molecular response to treatment: Duration of MMR for patients not in MMR at treatment initiation	Up to 15 months
Secondary	Event-Free Surviva (EFS)	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	Up to 15 months
Secondary	Progression-Free Survival (PFS)	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	Up to 15 months
Secondary	Comorbidity profile	Charlson comorbidity index (CCI) at index date	Baseline
Secondary	Disease characteristics	Disease characteristics to be provided	Baseline
Secondary	History of TKI treatment	History of TKI treatment to be provided	Baseline
Secondary	Management of patients in real-life	Concomitant medications	15 months
Secondary	Exposure patterns to asciminib	Exposure patterns to be provided	15 months
Secondary	EORTC QLQ-C30 (European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30)	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.	Baseline, 3months, 6months, 9months, 12months, 15months
Secondary	EORTC QLQ-CML24 questionnaire	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.	Baseline, 3months, 6months, 9months, 12months, 15months
Secondary	Emergence of mutations	If available at end of treatment: mutational analysis, methods and results	Up to 15 months
Secondary	Proportion of patient with an AE described during asciminib treatment among patient reporting the same AE which led to discontinuation with previous TKI treatments	Cross intolerance with previous TKI treatments	Up to 15 months