Leukemia, Chronic MyeloidNCT07151820
A Real-world Study About the Impact of Adverse Events (AEs) on Treatment Adjustments, Healthcare Resource Use, and Costs Among Chronic Myeloid Leukemia Patients
An observational study of Leukemia, Chronic Myeloid, run by Novartis Pharmaceuticals at 1 site in United States.
Trial summary
The aim of this study was to assess the frequency of adverse events (AEs) experienced by chronic myeloid leukemia (CML) patients treated with tyrosine kinase inhibitors (TKIs) and evaluate their impact on treatment adjustments as well as the burden of AEs on healthcare resource use (HRU) and costs. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia, Chronic Myeloid
- How many people
- 2,546 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Apr 2024to 22 Oct 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: This was a retrospective, noninterventional cohort study.
Who may be able to join
- Patients who met all of the following criteria were considered for inclusion:
- Had at least two diagnoses for CML- International Classification of Disease, 10th revision, Clinical Modification (ICD-10-CM) code: C92.1x
- Aged 18 years or older at the time of first CML diagnosis
- Received a 1G or 2G TKI after the first CML diagnosis
- Had continuous health plan enrollment (pharmacy and medical benefits) for at least 6 months before the index date (baseline) and 6 months after the index date
Who cannot join
- Patients who met any of the following criteria were excluded:
- Patients had 2 or more diagnoses of gastrointestinal stromal tumor ([GIST], ICD-10: C49.Ax) or chronic myelomonocytic leukemia ([CMML], ICD-10: C93.1x) at any time
- Patients had a hematopoietic stem cell transplantation (HSCT) during the baseline period
- Patients had CML-related chemotherapy treatment for accelerated phase (AP)/blast crisis (BC) during the baseline period
- Patients had medical claims associated with a clinical trial during the baseline period up to the end of the observation period
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Association of Adverse Events in the Real World With Treatment Adjustments, and Burden of AEs in Patients With Chronic Myeloid Leukemia
The aim of this study was to assess the frequency of adverse events (AEs) experienced by chronic myeloid leukemia (CML) patients treated with tyrosine kinase inhibitors (TKIs) and evaluate their impact on treatment adjustments as well as the burden of AEs on healthcare resource use (HRU) and costs.
This study used real-world administrative claims data from the PharMetrics Plus database. AEs of interest were selected based on information obtained from the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines, Chronic Myeloid Leukemia Version 1. 2024 and clinical inputs. Given the nature of claims data, only AEs of interest that required medical attention (i.e., resulted in a claim record) were captured in this study.
- Conditions
- Leukemia, Chronic Myeloid
- Keywords
- Tyrosine kinase inhibitor, Adverse events, Treatment adjustment, Healthcare resource use, Healthcare costs
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| First Generation (1G) TKI Cohort | Adult CML patients who received imatinib, a 1G TKI. | |
| Second Generation (2G) TKI Cohort | Adult CML patients who received the following 2G TKIs: dasatinib, nilotinib, or bosutinib. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients With AEs of Interest During the Study Period Among Those Without the Specific Event During the Baseline Period
The Study Period spanned from treatment initiation until treatment discontinuation, one day before treatment switch to another CML treatment, end of data availability, end of health plan coverage, or death, whichever came first.
Measured: Up to approximately 7 years and 3 months
Number of Patients With AEs of Interest Among All Patients During the Study Period
The Study Period spanned from treatment initiation until treatment discontinuation, one day before treatment switch to another CML treatment, end of data availability, end of health plan coverage, or death, whichever came first.
Measured: Up to approximately 7 years and 3 months
Secondary outcomes (15)
Number of Patients With Treatment Adjustments During the Observation Period
The Observation Period spanned from treatment initiation to the end of data availability, end of health plan coverage, or death, whichever occurred first.
Treatment adjustments included:
- Treatment discontinuation
- Treatment interruption
- Switching treatment
- Dose reduction
Measured: Up to approximately 7 years and 3 months
Duration of First Treatment Interruption
Measured: Up to approximately 7 years and 3 months
Time From TKI Initiation to Treatment Adjustment
Treatment adjustments included:
- Treatment discontinuation
- Treatment interruption
- Switching treatment
- Dose reduction
Measured: Up to approximately 7 years and 3 months
Number of Patients With AEs of Interest Within 30 Days Before Treatment Adjustment
Treatment adjustments included:
- Treatment discontinuation
- Treatment interruption
- Dose reduction
Measured: 6 months
Number of AEs of Interest Within 30 Days Before Treatment Adjustment
Treatment adjustments included:
- Treatment discontinuation
- Treatment interruption
- Dose reduction
Measured: 6 months
Number of Patients by AE of Interest Experienced Within 30 Days Before Treatment Adjustment
Treatment adjustments included:
- Treatment discontinuation
- Treatment interruption
- Dose reduction
Measured: 6 months
Time Between the Last AE of Interest and Treatment Adjustment
Treatment adjustments included:
- Treatment discontinuation
- Treatment interruption
- Dose reduction
Measured: 6 months
Number of Patients by Last AE of Interest Experienced Before Treatment Adjustment
Treatment adjustments included:
- Treatment discontinuation
- Treatment interruption
- Dose reduction
Measured: 6 months
Number of All-cause Healthcare Visits Per-Patient-Per-Year (PPPY)
Healthcare visits included:
- Inpatient admissions
- Emergency department visits
- Outpatient visits
- Other visits
Measured: 6 months
Number of Patients With One or More All-cause Healthcare Visits
Healthcare visits included:
- Inpatient admissions
- Emergency department visits
- Outpatient admissions
- Other visits
Measured: 6 months
Number of All-cause Inpatient Days
Measured: 6 months
Number of All-cause Inpatient Days Among Hospitalized Patients
Measured: 6 months
All-cause Healthcare Costs PPPY
Healthcare costs included:
- Total costs
- Medical costs
- Inpatient costs
- Emergency department costs
- Outpatient costs
- Other costs
- Pharmacy costs
Measured: 6 months
Annual Mean Number of Healthcare Visits Associated With AEs of Interest
Healthcare visits included:
- Inpatient admissions
- Emergency department visits
- Outpatient admissions
- Other visits
Measured: 6 months
Annual Mean Costs of Healthcare Visits Associated With AEs of Interest
Healthcare costs included:
- Total costs
- Medical costs
- Inpatient costs
- Emergency department costs
- Outpatient costs
- Other costs
- Pharmacy costs
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Sep 2025
- Study start8 Apr 2024
- Primary completion22 Oct 2024
- Study completion22 Oct 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leukemia, Chronic MyeloidStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 2,546 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07151820
- Sponsor's protocol ID
- CABL001A0US08
- Status last verified
- Aug 2025
- First submitted
- 25 Aug 2025
- Last updated
- 3 Sep 2025
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Association of Adverse Events in the Real World With Treatment Adjustments, and Burden of AEs in Patients With Chronic Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT07151820
- Acronym / sponsor ID
- CABL001A0US08
- Conditions
- Leukemia, Chronic Myeloid
- Keywords
- Tyrosine kinase inhibitor, Adverse events, Treatment adjustment, Healthcare resource use, Healthcare costs
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 2,546 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 8 Apr 2024 (actual)
- Primary completion
- 22 Oct 2024 (actual)
- Study completion
- 22 Oct 2024 (actual)
- First posted
- 3 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this study was to assess the frequency of adverse events (AEs) experienced by chronic myeloid leukemia (CML) patients treated with tyrosine kinase inhibitors (TKIs) and evaluate their impact on treatment adjustments as well as the burden of AEs on healthcare resource use (HRU) and costs.
This study used real-world administrative claims data from the PharMetrics Plus database. AEs of interest were selected based on information obtained from the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines, Chronic Myeloid Leukemia Version 1. 2024 and clinical inputs. Given the nature of claims data, only AEs of interest that required medical attention (i.e., resulted in a claim record) were captured in this study.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| First Generation (1G) TKI Cohort | – | –Adult CML patients who received imatinib, a 1G TKI. |
| Second Generation (2G) TKI Cohort | – | –Adult CML patients who received the following 2G TKIs: dasatinib, nilotinib, or bosutinib. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- This was a retrospective, noninterventional cohort study.
Key inclusion criteria (5 listed)
- Had at least two diagnoses for CML- International Classification of Disease, 10th revision, Clinical Modification (ICD-10-CM) code: C92.1x
- Aged 18 years or older at the time of first CML diagnosis
- Received a 1G or 2G TKI after the first CML diagnosis
- Had continuous health plan enrollment (pharmacy and medical benefits) for at least 6 months before the index date (baseline) and 6 months after the index date
Key exclusion criteria (5 listed)
- Patients had 2 or more diagnoses of gastrointestinal stromal tumor ([GIST], ICD-10: C49.Ax) or chronic myelomonocytic leukemia ([CMML], ICD-10: C93.1x) at any time
- Patients had a hematopoietic stem cell transplantation (HSCT) during the baseline period
- Patients had CML-related chemotherapy treatment for accelerated phase (AP)/blast crisis (BC) during the baseline period
- Patients had medical claims associated with a clinical trial during the baseline period up to the end of the observation period
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Number of Patients With AEs of Interest During the Study Period Among Those Without the Specific Event During the Baseline Period
Time frame: Up to approximately 7 years and 3 months
The Study Period spanned from treatment initiation until treatment discontinuation, one day before treatment switch to another CML treatment, end of data availability, end of health plan coverage, or death, whichever came first.
Number of Patients With AEs of Interest Among All Patients During the Study Period
Time frame: Up to approximately 7 years and 3 months
The Study Period spanned from treatment initiation until treatment discontinuation, one day before treatment switch to another CML treatment, end of data availability, end of health plan coverage, or death, whichever came first.
15 secondary outcomes
Number of Patients With Treatment Adjustments During the Observation Period
Time frame: Up to approximately 7 years and 3 months
Duration of First Treatment Interruption
Time frame: Up to approximately 7 years and 3 months
Time From TKI Initiation to Treatment Adjustment
Time frame: Up to approximately 7 years and 3 months
Number of Patients With AEs of Interest Within 30 Days Before Treatment Adjustment
Time frame: 6 months
Number of AEs of Interest Within 30 Days Before Treatment Adjustment
Time frame: 6 months
Number of Patients by AE of Interest Experienced Within 30 Days Before Treatment Adjustment
Time frame: 6 months
Time Between the Last AE of Interest and Treatment Adjustment
Time frame: 6 months
Number of Patients by Last AE of Interest Experienced Before Treatment Adjustment
Time frame: 6 months
Number of All-cause Healthcare Visits Per-Patient-Per-Year (PPPY)
Time frame: 6 months
Number of Patients With One or More All-cause Healthcare Visits
Time frame: 6 months
Number of All-cause Inpatient Days
Time frame: 6 months
Number of All-cause Inpatient Days Among Hospitalized Patients
Time frame: 6 months
All-cause Healthcare Costs PPPY
Time frame: 6 months
Annual Mean Number of Healthcare Visits Associated With AEs of Interest
Time frame: 6 months
Annual Mean Costs of Healthcare Visits Associated With AEs of Interest
Time frame: 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Leukemia, Chronic Myeloid, run by Novartis Pharmaceuticals at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Patients With AEs of Interest During the Study Period Among Those Without the Specific Event During the Baseline Period (time frame: Up to approximately 7 years and 3 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2025.