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

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Chronic Myeloid Leukemia
• Intervention / Treatment: 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 Type: Observational
• Enrollment (Actual): 201

Contacts and Locations

Study Locations

• France
  • Aix-en-Provence, France, 13616
    • Novartis Investigative Site
  • Amiens, France, 80054
    • Novartis Investigative Site
  • Antibes, France, 06600
    • Novartis Investigative Site
  • Avignon, France, 84000
    • Novartis Investigative Site
  • Besançon, France, 25030
    • Novartis Investigative Site
  • Bobigny, France, 93009
    • Novartis Investigative Site
  • Bordeaux, France, 33000
    • Novartis Investigative Site
  • Bordeaux, France, 33076
    • Novartis Investigative Site
  • Brest, France, 29609
    • Novartis Investigative Site
  • Brive-la-Gaillarde, France, 19100
    • Novartis Investigative Site
  • Caen, France, 14033
    • Novartis Investigative Site
  • Cagnes-sur-Mer, France, 06800
    • Novartis Investigative Site
  • Cannes, France, 06414
    • Novartis Investigative Site
  • Castelnau-le-Lez, France, 34170
    • Novartis Investigative Site
  • Cesson-Sévigné, France, 35576
    • Novartis Investigative Site
  • Chambéry, France, 73000
    • Novartis Investigative Site
  • Clermont-Ferrand, France, 63003
    • Novartis Investigative Site
  • Corbeil-Essonnes, France, 91100
    • Novartis Investigative Site
  • Dunkirk, France, 59240
    • Novartis Investigative Site
  • Essey-lès-Nancy, France, 54270
    • Novartis Investigative Site
  • Grenoble, France, 38043
    • Novartis Investigative Site
  • Le Kremlin-Bicêtre, France, 94275
    • Novartis Investigative Site
  • Le Mans, France, 72000
    • Novartis Investigative Site
  • Lens, France, 62307
    • Novartis Investigative Site
  • Libourne, France, 33505
    • Novartis Investigative Site
  • Lorient, France, 56100
    • Novartis Investigative Site
  • Lyon, France, 69373
    • Novartis Investigative Site
  • Montpellier, France, 34295
    • Novartis Investigative Site
  • Montpellier, France, 34070
    • Novartis Investigative Site
  • Mulhouse, France, 68070
    • Novartis Investigative Site
  • Nantes, France, 44277
    • Novartis Investigative Site
  • Nevers, France, 58000
    • Novartis Investigative Site
  • Nîmes, France, 30029
    • Novartis Investigative Site
  • Paris, France, 75013
    • Novartis Investigative Site
  • Paris, France, 75015
    • Novartis Investigative Site
  • Paris, France, 75475
    • Novartis Investigative Site
  • Perpignan, France, 66046
    • Novartis Investigative Site
  • Pierre-Bénite, France, 69495
    • Novartis Investigative Site
  • Poitiers, France, 86021
    • Novartis Investigative Site
  • Quimper, France, 29000
    • Novartis Investigative Site
  • Rennes, France, 35033
    • Novartis Investigative Site
  • Roubaix, France, 59100
    • Novartis Investigative Site
  • Strasbourg, France, 67085
    • Novartis Investigative Site
  • Tarbes, France, 65100
    • Novartis Investigative Site
  • Toulouse, France, 31059
    • Novartis Investigative Site
  • Tours, France, 37044
    • Novartis Investigative Site
  • Valence, France, 26953
    • Novartis Investigative Site
  • Vandœuvre-lès-Nancy, France, 54511
    • Novartis Investigative Site
  • Vesoul, France, 70014
    • Novartis Investigative Site
  • France
    • Angers, France, France, 49933
      • Novartis Investigative Site
  • Haute Vienne
    • Limoges, Haute Vienne, France, 87000
      • Novartis Investigative Site
  • Pays de la Loire Region
    • Saint Priest Jarez, Pays de la Loire Region, France, 42270
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) previously treated with two or more tyrosine kinase inhibitors

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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
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
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
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
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
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
Comorbidity profile	Charlson comorbidity index (CCI) at index date	Baseline
Disease characteristics	Disease characteristics to be provided	Baseline
History of TKI treatment	History of TKI treatment to be provided	Baseline
Management of patients in real-life	Concomitant medications	15 months
Exposure patterns to asciminib	Exposure patterns to be provided	15 months
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
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
Emergence of mutations	If available at end of treatment: mutational analysis, methods and results	Up to 15 months
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

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): March 6, 2024
• Primary Completion (Estimated): December 15, 2026
• Study Completion (Estimated): December 15, 2026

Study Registration Dates

• First Submitted: October 16, 2023
• First Submitted That Met QC Criteria: October 16, 2023
• First Posted (Actual): October 23, 2023

Study Record Updates

• Last Update Posted (Actual): May 29, 2026
• Last Update Submitted That Met QC Criteria: May 27, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: France; CML; Chronic myeloid leukemia; asciminib
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Chronic Disease; Disease Attributes; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia, Myeloid; Bone Marrow Diseases; Leukemia; Myeloproliferative Disorders; Pathological Conditions, Signs and Symptoms; Hemic and Lymphatic Diseases; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Tyrosine Kinase Inhibitors; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Protein Kinase Inhibitors; asciminib
• Other Study ID Numbers: CABL001AFR04

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO