Clinical trial NCT06211153: 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)
Official title: 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)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Chronic Myeloid Leukemia With T315I Mutation
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2024-01-18
Enrollment: 31
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This was a retrospective, noninterventional cohort study.

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


Eligibility criteria: 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.

Primary outcomes:
measure: Major molecular response (MMR) at 6 months
timeFrame: At 6 months
measure: Major molecular response (MMR) by 6 months
timeFrame: By 6 months
Secondary outcomes:
measure: MMR at and by 3, 9, and 12 months
timeFrame: At and by 3, 9, and 12 months
measure: Complete cytogenic response (CCyR) at and by 3, 6, 9, and 12 months
timeFrame: At and by 3, 6, 9, and 12 months
measure: Complete hematologic response (CHR) at and by 3, 6, 9, and 12 months
timeFrame: At and by 3, 6, 9, and 12 months
measure: Time to first CCyR at and by 3, 6, 9, and 12 months
timeFrame: At and by 3, 6, 9, and 12 months
measure: Gender
timeFrame: Baseline
measure: Race
timeFrame: Baseline
measure: Time from CML diagnosis to study index (i.e., asciminib) start date
timeFrame: Baseline
measure: Country of residence
timeFrame: Baseline
measure: Status of CML at baseline
timeFrame: Baseline
measure: Number of patients per response type at asciminib start date
timeFrame: Baseline
measure: Stem-cell transplant performed status
timeFrame: Baseline
measure: Medical history by system-organ-class, preferred term from CML diagnosis to asciminib start date
timeFrame: Baseline
measure: Overall tyrosine kinase inhibitors (TKIs) prior to index date
timeFrame: Baseline
measure: Prior TKIs between CML diagnosis and T315I mutation diagnosis
timeFrame: Baseline
measure: Prior TKIs between T315I mutation diagnosis and index date
timeFrame: Baseline
measure: Sequence of prior TKI treatment patterns
timeFrame: Baseline
measure: Number of patients with response to prior TKIs from CML diagnosis to asciminib (index date)
timeFrame: Baseline
measure: Number of patients with response to prior TKIs between CML diagnosis to T315I mutation diagnosis
timeFrame: Baseline
measure: Number of patients with response to prior TKIs between T315I mutation diagnosis to asciminib start (index date)
timeFrame: Baseline
measure: Number of patients with intolerance to prior TKIs, per category
timeFrame: Baseline
measure: Number of patients with intolerance to last TKI prior to asciminib, per category
timeFrame: Baseline
measure: Asciminib dose
timeFrame: Up to 13 months
measure: Asciminib discontinuation
timeFrame: Up to 13 months
measure: Duration of exposure to asciminib
timeFrame: Up to 13 months
Other outcomes:
Locations:
city: Basel
country: Switzerland
facility: Novartis
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: