A Study to Evaluate Available Treatment Information of Ponatinib, Bosutinib, Imatinib, Dasatinib and Nilotinib in Adults With Chronic Myeloid Leukemia

Leukemia Clinical Trial

Official title:

Evaluate the Real-World Safety Outcomes and Clinical Efficacy of Ponatinib and Other Tyrosine Kinase Inhibitors Among Chronic Myeloid Leukemia Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05743465
• Other study ID #: GOR-2017-102256
• Status: Completed
• Start date: October 6, 2021
• Est. completion date: November 30, 2022

Study information

• Verified date: February 2023
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1769
• Est. completion date: November 30, 2022
• Est. primary completion date: November 30, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Leukemia
• Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Intervention

Other:
• No Intervention
As this is an observational study, no intervention will be administered in this study.

Locations

Country	Name	City	State
United States	Takeda	Cambridge	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Takeda

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Chronic Myeloid Leukemia (CML) Participants Categorized by Sociodemographic Variables at Diagnosis	Socio-demographic variables included will include categories of Age (in years), Sex (male and female), and US geographic region.	Baseline (Day 1)
Primary	Number of CML Participants With Baseline Clinical Characteristics of Disease Severity		Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
Primary	Quan-Charlson Comorbidity Index Score	The Charlson Comorbidity Index is a 19-item measure assessing comorbid conditions. The total possible score on the Charlson Comorbidity Index ranges from 0 to 37. If a condition is not present, the score for that condition is zero. The higher scores indicate greater comorbidity.	Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
Primary	Number of CML Participants With Baseline Clinical Characteristics of Comorbidities	Comorbidities will include anemia, diabetes, chronic pulmonary disease, congestive heart failure, hypertension, hypercholesterolemia, obesity, renal disease, moderate to severe liver disease, dementia, acquired immune deficiency syndrome (AIDS)/human immunodeficiency virus (HIV).	Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
Primary	Number of CML Participants With Baseline Clinical Characteristics of Concomitant Medication	Concomitant medication will include antithrombotic agents (anticoagulants, antiplatelet), antihypertensive, and antidiabetic drugs (angiotensin converting enzyme inhibitor, angiotensin receptor blocker, beta blockers and statins) and antidiabetic drugs (metformin, sulfonylurea, thiazolidinedione, insulin and other antidiabetic drugs).	Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
Primary	Number of Previous Treatments of Tyrosine Kinase Inhibitors (TKI) Drugs in Participants with CML	The number of TKI drugs used prior to the index date will be identified. The type of the 1^st and 2^nd TKI drugs will be identified.	Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
Primary	Duration Between Last TKI Run-out Date to the Index Date for Participants with CML	Time from the run-out date of the last prescription to the index date will be calculated. If the run-out date passed the index date, the gap will be counted as 0.	Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
Primary	Number of Participants with Bone Marrow Stem Cell Transplant		Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
Primary	Clinical Characteristics Assessed by Number of Participants With Major Adverse Cardiac Events (MACE), Arterial Occlusive Events (AOEs), and Venous Thrombotic Events (VTEs)	MACE will be categorized as myocardial infarction and stroke. AOE will be categorized according to cardiovascular events, cerebrovascular events and peripheral vascular arterial events will be categorized as pulmonary embolism (PE) and deep vein thrombosis (DVT).	Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1
Primary	Treatment Patterns Based on Duration of Index Treatment	Time from the first prescription of the index drug to the run-out date of the last prescription of the index drug, or 1 day before a new TKI prescription date, whichever occurred earlier.	Up to approximately 5 years
Secondary	Number of Participants With BCR-ABL and Bone Marrow Testing	BCR-ABL test will include participants tested for BCR-ABL mutation, such as T315I, and participants with a diagnostic marrow test.	Up to approximately 5 years
Secondary	Treatment Patterns Based on Mean Starting Daily Dose and Average Daily Dose in Participants with CML	The mean daily and average dose of the first prescription in the treatment line will be calculated. The starting and average daily dose will be classified as low, standard, and high for each drug cohort.	Up to approximately 5 years
Secondary	Number of CML Participants With Disease Severity as per Medstat Disease Staging Clinical Criteria Version 5.21	The disease severity will include categories of low, moderate, and high severity.	Up to approximately 5 years
Secondary	Treatment Patterns Based on Number of Participants With CML on Concomitant Medication	The concomitant medications will include antithrombotic agents (anticoagulants, antiplatelet), antihypertensive, and antidiabetic drugs.	Up to approximately 5 years
Secondary	Number of Participants With CML With Treatment-Free Gap of the Index Treatment		Up to approximately 5 years
Secondary	Disease Progression	Disease progression will be defined as having a change of disease severity from low severity in the baseline period to moderate or high severity in the current line, or from moderate severity in the baseline period to high severity in the current line OR a change in TKI type, addition of chemotherapy agents, or allogeneic stem cell transplant procedure OR mortality.	Up to approximately 5 years
Secondary	Percentage of CML Participants With Complications		Up to approximately 5 years
Secondary	Overall Survival (OS)	OS is defined as the interval between the first does of study treatment and death due to any cause, censored at the last contact date when the participant was alive.	Up to approximately 5 years
Secondary	Progression Free Survival (PFS)	PFS is defined as the time in days from the index date to the first observed disease progression.	Up to approximately 5 years
Secondary	Number of Participants with Atleast one Adverse Event, Major Adverse Cardiac Event (MACE), Arterial Occlusive Events (AOEs) and Venous Thrombotic Events (VTEs)	AE is any untoward medical occurrence in participant administered medicinal investigational drug. Untoward medical occurrence does not necessarily have to have causal relationship with the treatment. MACE will be categorized as myocardial infarction and stroke. AOE will be categorized according to cardiovascular events, cerebrovascular events and peripheral vascular arterial events will be categorized as PE and DVT.	Up to approximately 5 years

External Links

•   To obtain more information on the study, click here/on this link