RWE,NIS,Prospective Study for the Effectiveness, Tolerability and Adherence of Asciminib in Saudi Arabia. (ASC4REAL) (ASC4REAL)

Real-World Evidence Non-interventional Prospective Study for the Effectiveness, Tolerability and Adherence of Asciminib After Two Previous TKIs in Patients With Chronic Myelogenous Leukemia in Chronic Phase (CML-CP) in Saudi Arabia. (ASC4REAL)

This is a non-interventional, prospective, observational single arm study describing the real-world effectiveness, tolerability, adherence, Healthcare Resource Utilization (HCRU) and Patient-Reported Outcome (PRO) data of asciminib in patients with Ph+ Chronic Myelogenous Leukemia - Chronic Phase (CML-CP) previously treated with ≥2 Tyrosine Kinase Inhibitor (TKIs) in routine clinical practice in Kingdom of Saudi Arabia.

Study Overview

• Status: Recruiting
• Conditions: Chronic Myelogenous Leukemia in Chronic Phase
• Intervention / Treatment: Other: asciminib

Detailed Description

Patients who have been receiving asciminib for up to 6 months prior to study start will be enrolled over a period of approximately 6 months and followed up for 12 months to assess study outcomes.

Each patient will be followed from baseline up to the earliest of death, loss to follow-up or end of the 12-month follow-up.

The study aims to enrol approximately 40 patients in a single arm design from a total of 6 centers in Kingdom of Saudi Arabia (KSA) over an enrollment period of approximately 6 months. These patients must have been prescribed asciminib for a maximum of 6 months before enrolling in the study. The prescription of asciminib should align with its approved label use. Importantly, the decision to prescribe asciminib to these patients should have been made independently by the physician and not influenced by the study. Data will be collected from patient electronic medical records in the sites chosen.

• Study Type: Observational
• Enrollment (Estimated): 40

Contacts and Locations

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

Study Locations

• Saudi Arabia
  • Dammam, Saudi Arabia, 15215
    • Recruiting
    • Novartis Investigative Site
  • Jeddah, Saudi Arabia, 21423
    • Recruiting
    • Novartis Investigative Site
  • Riyadh, Saudi Arabia, 11211
    • Recruiting
    • Novartis Investigative Site
  • Riyadh, Saudi Arabia, 11426
    • Recruiting
    • 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

Patients must have been prescribed asciminib for a maximum of 6 months before enrolling in the study. Patients will be included in the study regardless of their MMR status at the initiation of asciminib treatment.

Description

Inclusion Criteria:

Patients eligible for inclusion in this study must meet all the following criteria:

1. Signed and dated Patient Informed Consent Form obtained.

2. Male or female patients with a diagnosis of Ph+ CML-CP ≥18 years of age with the following laboratory values:

   1. <15% blasts in peripheral blood and bone marrow
   2. <30% blasts plus promyelocytes in peripheral blood and bone marrow
   3. <20% basophils in the peripheral blood
   4. ≥50 x 109/L (≥50,000/mm3) platelets

4. Prior treatment with a minimum of 2 prior TKIs: e. First-generation TKI imatinib f. 2G-TKI dasatinib, nilotinib and bosutinib g. 3G-TKI ponatinib 4. Patients are currently receiving asciminib as per approved local label and independently of study participation. Patients must have started asciminib therapy no more than 6 months prior to their enrollment in the study.

Exclusion Criteria:

Patients meeting ANY of the following criteria are not eligible for participation:

1. Asciminib based treatment regimen in first or second line.
2. Patients are currently participating in any other clinical trials.
3. Patients who refused to sign informed consent.
4. According to the investigator's opinion, the patient is an unlikely candidate to provide an accurate medical history and/or to obtain long-term follow-up information for any reasons such as unavailability or severe concomitant illnesses.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Asciminib; Patients who have been receiving asciminib for up to 6 months prior to study start	Other: asciminib; This is an observational study. There is no treatment allocation. The decision to initiate asciminib will be based solely on clinical judgement.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Major molecular response (MMR)	MMR is defined as a breakpoint cluster region-Abelson murine leukemia viral oncogene homolog 1 protein (BCR-ABL1) transcript level ≤0.1%, at 12 months.	Month 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Frequency and severity of hematologic and non-hematologic AEs	Frequency and severity of hematologic and non-hematologic AEs	Up to 12 months
Major Molecular Response (MMR) Rate	MMR is defined as a BCR-ABL1 transcript level ≤0.1%.	Baseline, month 6, month 12
Deep Molecular Response (DMR) Rate	Percentage of participants with MR4.0 and MR4.5: MR4.0 [ BCR-ABL1 transcript level ≤0.01%]; MR4.5 [ BCR-ABL1 transcript level ≤0.0032%]	Month 6, month 12
Cytogenetic Response (CCyR) Rate	Assessment of CCyR and/or [BCR-ABL1 transcript level ≤1%]	Month 6, month 12
Duration of MMR	Assess the duration of MMR in participants.	Up to 12 months
Simplified Medication Adherence Questionnaire (SMAQ) score.	SMAQ assesses adherence to medication. It is a 6-item questionnaire that evaluates different aspects of patient compliance with treatment: forgetfulness, routine, adverse effects, and omissions. A patient is classified as non-compliant if he/she responds to any of the questions with a non-adherence answer, and in terms of quantification, if the patient has lost more than two doses during the last week or has not taken medication during more than two complete days during the last three months.	Month 6, month 12

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): March 24, 2025
• Primary Completion (Estimated): December 30, 2026
• Study Completion (Estimated): December 30, 2026

Study Registration Dates

• First Submitted: November 11, 2024
• First Submitted That Met QC Criteria: November 11, 2024
• First Posted (Actual): November 12, 2024

Study Record Updates

• Last Update Posted (Actual): December 23, 2025
• Last Update Submitted That Met QC Criteria: December 22, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: Asciminib; Chronic Myelogenous Leukemia in chronic phase; CML-CP
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Chronic Disease; Disease Attributes; Neoplasms by Histologic Type; Hematologic Diseases; Bone Marrow Diseases; Myeloproliferative Disorders; Leukemia; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Tyrosine Kinase Inhibitors; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Protein Kinase Inhibitors; asciminib
• Other Study ID Numbers: CABL001ASA01

Plan for Individual participant data (IPD)

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