Real-world Effectiveness of Asciminib and Treatment Patterns in Patients With Chronic Myeloid Leukemia With T315I Mutation - a Chart Review Study of Patients Treated in the Asciminib Managed Access Program (MAP)

This non-interventional study (NIS) was a retrospective chart review analyzing existing data from patients participating in the asciminib MAP.

Study Overview

• Status: Completed
• Conditions: Chronic Myeloid Leukemia With T315I Mutation

• Study Type: Observational
• Enrollment (Actual): 31

Contacts and Locations

Study Locations

• Switzerland
  • Basel, Switzerland, 4056
    • Novartis

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Non-Probability Sample

Study Population

This was a retrospective, noninterventional cohort study.

Description

Study Population: Key Inclusion/Exclusion Criteria Inclusion criteria

• Diagnosis of CML (chronic phase, accelerated phase or blast crisis).
• Confirmed presence of T315I mutation prior to asciminib initiation.
• Patients enrolled in the asciminib MAP and received their first dose of asciminib between 01 November 2018 and 30 April 2022. Patients must have received at least one dose of asciminib.

• Appropriate approval was obtained for the patient chart review including:

  • Patient signed the informed consent form (ICF) or,
  • Individual ICF waiver was granted by an institutional review board/ Independent Ethics Committee (IRB)/ IEC.

Exclusion criteria

• Age less than 18 years old at the time of initiating asciminib treatment.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Asciminib

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Major molecular response (MMR) at 6 months	At 6 months
Major molecular response (MMR) by 6 months	By 6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Gender		Baseline
Race		Baseline
MMR at and by 3, 9, and 12 months		At and by 3, 9, and 12 months
Complete cytogenic response (CCyR) at and by 3, 6, 9, and 12 months		At and by 3, 6, 9, and 12 months
Complete hematologic response (CHR) at and by 3, 6, 9, and 12 months		At and by 3, 6, 9, and 12 months
Time to first CCyR at and by 3, 6, 9, and 12 months		At and by 3, 6, 9, and 12 months
Time from CML diagnosis to study index (i.e., asciminib) start date		Baseline
Country of residence		Baseline
Status of CML at baseline		Baseline
Number of patients per response type at asciminib start date	Response types were MMR, CCyR, and CHR.	Baseline
Stem-cell transplant performed status		Baseline
Medical history by system-organ-class, preferred term from CML diagnosis to asciminib start date		Baseline
Overall tyrosine kinase inhibitors (TKIs) prior to index date		Baseline
Prior TKIs between CML diagnosis and T315I mutation diagnosis		Baseline
Prior TKIs between T315I mutation diagnosis and index date		Baseline
Sequence of prior TKI treatment patterns		Baseline
Number of patients with response to prior TKIs from CML diagnosis to asciminib (index date)	Response types were MMR, CCyR, and CHR.	Baseline
Number of patients with response to prior TKIs between CML diagnosis to T315I mutation diagnosis	Response types were MMR, CCyR, and CHR.	Baseline
Number of patients with response to prior TKIs between T315I mutation diagnosis to asciminib start (index date)	Response types were MMR, CCyR, and CHR.	Baseline
Number of patients with intolerance to prior TKIs, per category	Categories were discontinuation due to adverse events, relationship to treatment, and outcome.	Baseline
Number of patients with intolerance to last TKI prior to asciminib, per category	Categories were discontinuation due to adverse events, relationship to treatment, and outcome.	Baseline
Asciminib dose		Up to 13 months
Asciminib discontinuation		Up to 13 months
Duration of exposure to asciminib		Up to 13 months

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): December 31, 2021
• Primary Completion (Actual): December 15, 2022
• Study Completion (Actual): December 15, 2022

Study Registration Dates

• First Submitted: December 15, 2023
• First Submitted That Met QC Criteria: January 8, 2024
• First Posted (Estimated): January 18, 2024

Study Record Updates

• Last Update Posted (Estimated): January 18, 2024
• Last Update Submitted That Met QC Criteria: January 8, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms by Histologic Type; Neoplasms; Disease Attributes; Bone Marrow Diseases; Hematologic Diseases; Myeloproliferative Disorders; Chronic Disease; Leukemia; Leukemia, Myeloid; Leukemia, Myelogenous, Chronic, BCR-ABL Positive
• Other Study ID Numbers: CABL001A2004