Clinical trial NCT05743465: A Study to Evaluate Available Treatment Information of Ponatinib, Bosutinib, Imatinib, Dasatinib and Nilotinib in Adults With Chronic Myeloid Leukemia
Official title: Evaluate the Real-World Safety Outcomes and Clinical Efficacy of Ponatinib and Other Tyrosine Kinase Inhibitors Among Chronic Myeloid Leukemia Patients
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Leukemia
Keywords: Drug Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2023-02-24
Enrollment: 1769
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: Participants with a diagnosis of CP-CML and who had ≥1 prescription for TKI (imatinib, dasatinib, nilotinib, bosutinib, or ponatinib) from April 1, 2013-March 31, 2017 will be included in this study.

Brief summary: The aims of this study are to learn out about treatment information (including amongst others treatment patterns, safety, development of a participant's condition) ponatinib, bosutinib, imatinib, dasatinib and nilotinib using already available data. No new data will be collected from participants as part of this study and no study medicines will be provided in this study.

Detailed description: This is a retrospective cohort analysis study in participants with chronic phase chronic myeloid leukemia (CP-CML). This study will use Humedica electronic medical record (EMR) data to evaluate the real-world treatment patterns, safety, and efficacy of ponatinib and other tyrosine kinase inhibitors (TKIs) among CP-CML participants.

The study will enroll approximately 1769 patients. Based on the TKI drug used on index date, stratified by prior TKI use, participants will be classified into the following cohorts -

* Ponatinib Cohort
* Bosutinib Cohort
* Other TKI Cohort

This is a multicenter study conducted in the United States (US). The overall duration for data collection in this trial will be approximately 5 years.

Eligibility criteria: Inclusion criteria:

Participants will be included in the study if they:

1. had ≥1 prescription for TKI (imatinib, dasatinib, nilotinib, bosutinib, or ponatinib) from April 1, 2013-March 31, 2017;

   * For participants with ponatinib use, the first ponatinib prescription date will be defined as the index date; for participants with bosutinib but not ponatinib use, the first bosutinib prescription date will be defined as the index date; and for participants without ponatinib and bosutinib use, the first imatinib, dasatinib, nilotinib prescription date will be defined as the index date.
   * Participants with \>1 type of TKI on the index date will be dropped.
2. had ≥1 medical diagnosis for CML (International Classification of Diseases, Ninth Revision, Clinical Modification \[ICD-9-CM\]: 205.1; ICD-10-CM: C92.1) any time prior to the index date or within 6 months post-index date; diagnosis codes in the primary or secondary position will be used; the first CML diagnosis date will be designated as the initial CML diagnosis date;
3. were aged ≥18 years on the index date;
4. were active in the Humedica EMR data 6 months pre- and post-index date, indicated by the first and last healthcare activity in the data;

   * Participant data will be assessed until the earliest of switch to another TKI, disenrollment, death, or study end date. The minimum required duration of follow-up can be further revised based on the average duration of first-line treatment.
   * Participants who died in 6 months will be included.

Exclusion criteria:

Participants will be excluded from the study if they:

1. had ≥1 prescription for their index TKI (imatinib, dasatinib, nilotinib, bosutinib, or ponatinib) any time prior to the index date.
2. This will allow ≥6-month wash-out period, and ensure we are capturing second line participants.

Primary outcomes:
measure: Number of Chronic Myeloid Leukemia (CML) Participants Categorized by Sociodemographic Variables at Diagnosis
timeFrame: Baseline (Day 1)
measure: Number of CML Participants With Baseline Clinical Characteristics of Disease Severity
timeFrame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
measure: Quan-Charlson Comorbidity Index Score
timeFrame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
measure: Number of CML Participants With Baseline Clinical Characteristics of Comorbidities
timeFrame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
measure: Number of CML Participants With Baseline Clinical Characteristics of Concomitant Medication
timeFrame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
measure: Number of Previous Treatments of Tyrosine Kinase Inhibitors (TKI) Drugs in Participants with CML
timeFrame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
measure: Duration Between Last TKI Run-out Date to the Index Date for Participants with CML
timeFrame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
measure: Number of Participants with Bone Marrow Stem Cell Transplant
timeFrame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
measure: Clinical Characteristics Assessed by Number of Participants With Major Adverse Cardiac Events (MACE), Arterial Occlusive Events (AOEs), and Venous Thrombotic Events (VTEs)
timeFrame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
measure: Treatment Patterns Based on Duration of Index Treatment
timeFrame: Up to approximately 5 years
Secondary outcomes:
measure: Number of Participants With BCR-ABL and Bone Marrow Testing
timeFrame: Up to approximately 5 years
measure: Treatment Patterns Based on Mean Starting Daily Dose and Average Daily Dose in Participants with CML
timeFrame: Up to approximately 5 years
measure: Number of CML Participants With Disease Severity as per Medstat Disease Staging Clinical Criteria Version 5.21
timeFrame: Up to approximately 5 years
measure: Treatment Patterns Based on Number of Participants With CML on Concomitant Medication
timeFrame: Up to approximately 5 years
measure: Number of Participants With CML With Treatment-Free Gap of the Index Treatment
timeFrame: Up to approximately 5 years
measure: Disease Progression
timeFrame: Up to approximately 5 years
measure: Percentage of CML Participants With Complications
timeFrame: Up to approximately 5 years
measure: Overall Survival (OS)
timeFrame: Up to approximately 5 years
measure: Progression Free Survival (PFS)
timeFrame: Up to approximately 5 years
measure: Number of Participants with Atleast one Adverse Event, Major Adverse Cardiac Event (MACE), Arterial Occlusive Events (AOEs) and Venous Thrombotic Events (VTEs)
timeFrame: Up to approximately 5 years
Other outcomes:
Locations:
city: Cambridge
country: United States
facility: Takeda
state: Massachusetts
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: