A Study of Asciminib Safety and Efficacy in Young Adults With Chronic Myeloid Leukemia in the Gulf Region (ASC4Young)

ASC4Young: Real-World Study of Asciminib Safety and Efficacy in Young Adults With Chronic Myeloid Leukemia in the Gulf Region

The aim of this study is to evaluate the real-world effectiveness and safety of asciminib among young adults with chronic myeloid leukemia (CML) across the Gulf region. The data source for this study will consist of routinely collected clinical information documented within the electronic health records (EHR) of participating centers.

Study Overview

• Status: Not yet recruiting
• Conditions: Chronic Myeloid Leukemia

• Study Type: Observational
• Enrollment (Estimated): 80

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: +41613241111; Email: novartis.email@novartis.com
• Study Contact Backup: Name: Novartis Pharmaceuticals

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Non-Probability Sample

Study Population

CML patients who initiate asciminib between 01 May 2023 and 31 May 2026 in participating centers across the Gulf region.

Description

Inclusion Criteria

• Confirmed diagnosis of Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP)
• Adult patients aged ≥ 18 years at the index date (or adult as defined by applicable national regulations)
• Documented exposure to asciminib during the identification period (May 2023 to May 2026)
• Provision of signed informed consent for secondary use of data, or an ethics committee-approved waiver of consent, where applicable (including for deceased patients or where data were previously collected within the EHR system)

Exclusion Criteria

• Patients who do not meet the eligibility criteria specified above
• Patients with insufficient medical record documentation to determine asciminib exposure or key study outcomes

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
Young Adults Cohort; CML patients aged ≤ 40 years at the first documented asciminib administration.
Older Adults Cohort; CML patients aged > 40 years at the first documented asciminib administration.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Major Molecular Response (MMR) Rate in Young Adults	MMR is defined as a BCR::ABL1 level on the International Scale (BCR::ABL1 IS) ≤ 0.1%.	Approximately 12 Months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Adverse Events (AEs)		Approximately 3, 6, and 12 months
Number of Patients by Type and Severity of AEs	Number of patients by type and severity of AEs including serious AEs and grade ≥ 3 AEs according to the Common Terminology Criteria for Adverse Events (CTCAE) v6.0.	Approximately 3, 6, and 12 months
Incidence of AEs Leading to Dose Modifications	Dose modifications include treatment interruptions and dose reductions.	Approximately 3, 6, and 12 months
Number of Treatment Interruptions Due to AEs		Approximately 3, 6, and 12 months
Duration of Treatment Interruptions Due to AEs		Approximately 3, 6, and 12 months
Time to Treatment Discontinuation due to Adverse Events (TTDAE)		Approximately 3, 6, and 12 months
Proportion of Patients who Achieve an Early Molecular Response Milestone of BCR::ABL1 IS ≤ 10% After 3 Months of Treatment		3 months
Proportion of Patients who Achieve an Early Molecular Response Milestone of BCR::ABL1 IS ≤ 1% After 6 Months of Treatment		6 months
MMR in Patients Aged > 40 years	MMR is defined as BCR::ABL1 IS ≤ 0.1%.	Approximately 12 months
Rate of Deep Molecular Response	Deep molecular response includes: Molecular response (MR) 4.0 (BCR::ABL1 IS ≤ 0.01%), and; MR4.5 (BCR::ABL1 IS ≤ 0.0032%), where available.	Approximately 12 months
Number of Patients With Prior Tyrosine Kinase Inhibitor (TKI) Treatment		Up to 12 months prior to Baseline
Duration of CML at Time of Asciminib Treatment Initiation		Baseline
Asciminib Starting Dose		Baseline
Number of Patients With Dose Modifications	Number of patients with dose modifications including up titration or dose reduction.	Approximately 3, 6, and 12 months
Number of Treatment Interruptions		Approximately 3, 6, and 12 months
Duration of Treatment Interruptions		Approximately 3, 6, and 12 months
Number of Patients by Reasons for Treatment Interruptions and Subsequent Treatment Switching		Approximately 3, 6, and 12 months
Number of Patients by Reason for Treatment Switch		Approximately 3, 6, and 12 months
Number of Patients by Clinicopathological Characteristics	Clinicopathological characteristics include: Demographics (gender, ethnicity, nationality, smoking status, and insurance status); Clinical presentation (signs and symptoms of disease); Laboratory parameters (lipid panel, complete blood count, clinical chemistry, kidney, liver and pancreas function tests); Molecular findings (BCR::ABL1 transcript types, additional cytogenetic abnormalities, and other reported mutations)	Up to 12 months pre-Baseline, Baseline, and approximately 3, 6 , and 12 months post-Baseline

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Estimated): June 30, 2026
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): June 30, 2027

Study Registration Dates

• First Submitted: June 26, 2026
• First Submitted That Met QC Criteria: June 26, 2026
• First Posted (Actual): July 2, 2026

Study Record Updates

• Last Update Posted (Actual): July 2, 2026
• Last Update Submitted That Met QC Criteria: June 26, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Keywords: Efficacy and Safety; Young Adults; Chronic Myeloid Leukemia; Asciminib; Gulf Region; Real-world Evidence; Major Molecular Response; Unmet Medical Need
• 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
• Other Study ID Numbers: CABL001J1AE01

Plan for Individual participant data (IPD)

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