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

Not yet recruiting

• Conditions: Chronic Myelogenous Leukemia in Chronic Phase

• Registration Number: NCT06684964
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

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.

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 Timeline

• Study First Submitted: 2024-11-11
• Study First Submitted QC: 2024-11-11
• Study First Posted: 2024-11-12
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-08
• Last Update Posted: 2025-01-10
• Study Start: 2025-01-30
• Primary Completion: 2026-07-30
• Study Completion: 2026-07-30

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 40

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

3. 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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Major molecular response (MMR)	Month 12	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.

Secondary Outcome Measures

Name	Time	Method
Frequency and severity of hematologic and non-hematologic AEs	Up to 12 months	Frequency and severity of hematologic and non-hematologic AEs
Major Molecular Response (MMR) Rate	Baseline, month 6, month 12	MMR is defined as a BCR-ABL1 transcript level ≤0.1%.
Deep Molecular Response (DMR) Rate	Month 6, month 12	Percentage of participants with MR4.0 and MR4.5: * MR4.0 \[ BCR-ABL1 transcript level ≤0.01%\]; * MR4.5 \[ BCR-ABL1 transcript level ≤0.0032%\]
Cytogenetic Response (CCyR) Rate	Month 6, month 12	Assessment of CCyR and/or \[BCR-ABL1 transcript level ≤1%\]
Duration of MMR	Up to 12 months	Assess the duration of MMR in participants.
Simplified Medication Adherence Questionnaire (SMAQ) score.	Month 6, month 12	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.