Skip to main content
OpenTrials
Completed

NCT Number: NCT05743465

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Leukemia Hematologic Diseases Hemic and Lymphatic Diseases Neoplasms Neoplasms by Histologic Type

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Takeda

Cambridge, Massachusetts, 02139, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Participants will be included in the study if they:

  • 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.
  • 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;
  • were aged ≥18 years on the index date;
  • 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:

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

Treatment and study plan

No intervention

Other

As this is an observational study, no intervention will be administered in this study.

Primary outcomes

  1. Number of Chronic Myeloid Leukemia (CML) Participants Categorized by Sociodemographic Variables at Diagnosis

    Time frame: Baseline (Day 1)

    Socio-demographic variables included will include categories of Age (in years), Sex (male and female), and US geographic region.

  2. Number of CML Participants With Baseline Clinical Characteristics of Disease Severity

    Time frame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1

  3. Quan-Charlson Comorbidity Index Score

    Time frame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1

    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.

  4. Number of CML Participants With Baseline Clinical Characteristics of Comorbidities

    Time frame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1

    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).

  5. Number of CML Participants With Baseline Clinical Characteristics of Concomitant Medication

    Time frame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1

    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).

  6. Number of Previous Treatments of Tyrosine Kinase Inhibitors (TKI) Drugs in Participants with CML

    Time frame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1

    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.

  7. Duration Between Last TKI Run-out Date to the Index Date for Participants with CML

    Time frame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1

    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.

  8. Number of Participants with Bone Marrow Stem Cell Transplant

    Time frame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1

  9. Clinical Characteristics Assessed by Number of Participants With Major Adverse Cardiac Events (MACE), Arterial Occlusive Events (AOEs), and Venous Thrombotic Events (VTEs)

    Time frame: Up to 6 months prior to the day of initiation of TKI drug i.e., Day 1

    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).

  10. Treatment Patterns Based on Duration of Index Treatment

    Time frame: Up to approximately 5 years

    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.

Secondary outcomes

  1. Number of Participants With BCR-ABL and Bone Marrow Testing

    Time frame: Up to approximately 5 years

    BCR-ABL test will include participants tested for BCR-ABL mutation, such as T315I, and participants with a diagnostic marrow test.

  2. Treatment Patterns Based on Mean Starting Daily Dose and Average Daily Dose in Participants with CML

    Time frame: Up to approximately 5 years

    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.

  3. Number of CML Participants With Disease Severity as per Medstat Disease Staging Clinical Criteria Version 5.21

    Time frame: Up to approximately 5 years

    The disease severity will include categories of low, moderate, and high severity.

  4. Treatment Patterns Based on Number of Participants With CML on Concomitant Medication

    Time frame: Up to approximately 5 years

    The concomitant medications will include antithrombotic agents (anticoagulants, antiplatelet), antihypertensive, and antidiabetic drugs.

  5. Number of Participants With CML With Treatment-Free Gap of the Index Treatment

    Time frame: Up to approximately 5 years

  6. Disease Progression

    Time frame: Up to approximately 5 years

    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.

  7. Percentage of CML Participants With Complications

    Time frame: Up to approximately 5 years

  8. Overall Survival (OS)

    Time frame: Up to approximately 5 years

    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.

  9. Progression Free Survival (PFS)

    Time frame: Up to approximately 5 years

    PFS is defined as the time in days from the index date to the first observed disease progression.

  10. Number of Participants with Atleast one Adverse Event, Major Adverse Cardiac Event (MACE), Arterial Occlusive Events (AOEs) and Venous Thrombotic Events (VTEs)

    Time frame: Up to approximately 5 years

    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.

Sponsors and collaborators

Lead sponsor

Takeda

Industry

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2022
Study completion
2022
First posted
Feb 24, 2023
Registry last updated
Mar 2, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.