Chronic Myeloid LeukemiaABL-2022-03NCT06692803
Real-World Treatment Patterns and Outcomes Among Patients With Chronic Myeloid Leukemia in Earlier Lines of Therapy
An observational study of Chronic Myeloid Leukemia, run by Novartis Pharmaceuticals at 1 site in United States.
Trial summary
This was a retrospective, non-interventional, observational cohort study using Optum's de-identified Clinformatics® Data Mart Database. Adult patients newly diagnosed with chronic myeloid leukemia (CML) treated with tyrosine kinase inhibitors (TKIs) were identified using the Optum database and classified into the… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Myeloid Leukemia
- How many people
- 2,043 participants
- Where
- 1 siteUnited States
- Study dates
- 23 May 2023to 30 Nov 2023
- 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
- Adult patients who initiated their first treatment with imatinib, dasatinib, nilotinib, or bosutinib (conditional on Food and Drug Administration (FDA) approval dates) with 6 months continuous health plan enrollment prior to the first prescription fill date.
- Patients with 2 or more diagnoses for CML (adult as of the first diagnosis for CML).
- Patients had index date on or after first CML diagnosis.
- Patients had no diagnoses for CML remission/relapse prior to index date.
- Patients had no gastrointestinal stomach tumor (GIST) or chronic myelomonocytic leukemia (CMML) at any time.
- Patients had no medical claims associated with a clinical trial during the baseline period.
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- Patients had no hematopoietic stem cell transplantation (HSCT) during the baseline period.
- Patients had no CML-related chemotherapy treatments for accelerated phase (AP)/blast crisis (BC) during the baseline period.
- First treatment cohort:
- Patients started first treatment for CML with imatinib, dasatinib, nilotinib, or bosutinib in 2012 or later.
- Patients had no HSCT during the first treatment baseline period.
- Patients had no CML-related chemotherapy treatment for AP/BC during the first treatment baseline period.
- Second treatment cohort:
- Patients started second treatment for CML with imatinib, dasatinib, nilotinib, or bosutinib in 2012 or later.
- Patients had no HSCT from the first treatment baseline period up to second treatment initiation.
- Patients had no CML-related chemotherapy treatment for AP/BC from the first treatment baseline period up to second treatment initiation.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Real-World Treatment Patterns and Outcomes Among Patients With Chronic Myeloid Leukemia in Earlier Lines of Therapy (ABL-2022-03)
This was a retrospective, non-interventional, observational cohort study using Optum's de-identified Clinformatics® Data Mart Database. Adult patients newly diagnosed with chronic myeloid leukemia (CML) treated with tyrosine kinase inhibitors (TKIs) were identified using the Optum database and classified into the following cohorts:
- First treatment cohort: Patients newly diagnosed with CML who received first treatment with a TKI.
- Second treatment cohort: Patients from first treatment cohort with a subsequent line of therapy (i.e., second treatment) with a TKI.
The observation period spanned from the start of data availability (i.e., 01 January 2007) to the earliest of end of data (i.e., 30 June 2022), end of continuous health plan enrollment, or death (if available). The index date was defined as the first treatment initiation for the first treatment TKI cohort and as the second treatment initiation for the second treatment TKI cohort. The baseline period consisted of the 6 months prior to the index date. The follow-up period started on the index date and ended at the earliest of end of observation period or hematopoietic stem cell transplantation (HSCT).
- Conditions
- Chronic Myeloid Leukemia
- 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 Treatment Cohort | Patients newly diagnosed with CML who received first treatment with imatinib, dasatinib, nilotinib, or bosutinib. | |
| Second Treatment Cohort | Patients from first treatment cohort with a subsequent line of therapy (i.e., second treatment) with imatinib, dasatinib, nilotinib, bosutinib, or ponatinib. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment Patterns
Tyrosine kinase inhibitor (TKI) treatment management patterns in CML patients on first line or second line TKI were assessed.
Measured: Up to approximately 10 years
Secondary outcomes (17)
Proportion of Days Covered (PDC)
PDC was defined as the number of days of medication covered divided by the number of calendar days during the study period. The study period spanned from index date to the next line of therapy initiation (switch), hematopoietic stem cell transplantation (HSCT), initiation of a CML-related chemotherapy for accelerated phase (AP)/blast crisis (BC) (day prior to HSCT/CML chemotherapy) or last supply day if treatment gap ≥ 90 days, whichever occurred first. The index date was defined as the first treatment initiation for the first treatment cohort and as the second treatment initiation for the second treatment cohort.
Measured: Up to approximately 10 years
Number of Patients per PDC Category
PDC was defined as the number of days of medication covered divided by the number of calendar days during the study period. The study period spanned from index date to the next line of therapy initiation (switch), HSCT, initiation of a CML-related chemotherapy for AP/BC (day prior to HSCT/CML chemotherapy) or last supply day if treatment gap ≥ 90 days, whichever occurred first. The index date was defined as the first treatment initiation for the first treatment cohort and as the second treatment initiation for the second treatment cohort.
PDC categories included:
- PDC (%) ≤ 50%
- PDC (%) ≤ 70%
- PDC (%) ≥ 80%
- PDC (%) > 90%
Measured: Up to approximately 10 years
Time to Treatment Discontinuation
Measured: Up to approximately 10 years
Time to Treatment Switch
Measured: Up to approximately 10 years
Time to First Treatment Interruption
Measured: Up to approximately 10 years
Time to First Dose Reduction
Measured: Up to approximately 10 years
Rate of Treatment Discontinuation
Measured: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
Rate of Treatment Switching
Measured: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
Rate of Treatment Interruption
Measured: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
Rate of Dose Reduction
Measured: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
Number of Patients With Non-optimal Treatment (NOPT)
Three categories of NOPT were defined:
- Base Case: NOPT was defined as 1) treatment switch ≤ 6 months post-index, 2) temporary treatment interruption/dose reduction ≤6 months post-index followed by treatment switch ≤12 months post-index, or 3) PDC ≤50%.
- Sensitivity 1: NOPT was defined as 1) treatment switch ≤ 6 months post-index, 2) temporary treatment interruption/dose reduction ≤6 months post-index followed by treatment switch ≤12 months post-index, or 3) PDC ≤70%.
- Sensitivity 2: NOPT was defined as treatment switch ≤ 6 months post-index.
Index date was defined as the first treatment initiation for the first treatment cohort and as the second treatment initiation for the second treatment cohort.
Measured: Up to approximately 10 years
Number of Patients With NOPT by First Generation TKI
Three categories of NOPT were defined:
- Base Case: NOPT was defined as 1) treatment switch ≤ 6 months post-index, 2) temporary treatment interruption/dose reduction ≤6 months post-index followed by treatment switch ≤12 months post-index, or 3) PDC ≤50%.
- Sensitivity 1: NOPT was defined as 1) treatment switch ≤ 6 months post-index, 2) temporary treatment interruption/dose reduction ≤6 months post-index followed by treatment switch ≤12 months post-index, or 3) PDC ≤70%.
- Sensitivity 2: NOPT was defined as treatment switch ≤ 6 months post-index.
Index date was defined as the first treatment initiation for the first treatment cohort and as the second treatment initiation for the second treatment cohort.
Measured: Up to approximately 10 years
Number of Patients With NOPT by Second Generation TKI
Three categories of NOPT were defined:
- Base Case: NOPT was defined as 1) treatment switch ≤ 6 months post-index, 2) temporary treatment interruption/dose reduction ≤6 months post-index followed by treatment switch ≤12 months post-index, or 3) PDC ≤50%.
- Sensitivity 1: NOPT was defined as 1) treatment switch ≤ 6 months post-index, 2) temporary treatment interruption/dose reduction ≤6 months post-index followed by treatment switch ≤12 months post-index, or 3) PDC ≤70%.
- Sensitivity 2: NOPT was defined as treatment switch ≤ 6 months post-index.
Index date was defined as the first treatment initiation for the first treatment cohort and as the second treatment initiation for the second treatment cohort.
Measured: Up to approximately 10 years
All-cause Healthcare Resource Utilization (HRU) per Patient per Year in NOPT Patients Compared to Reference Subgroup
The reference subgroup was defined based on the following treatment adherence and persistence criteria denoting potential indicators of TKI treatment success based on observations from the clinical practice: (1) high treatment adherence (defined as PDC >90%) with no observed treatment discontinuation anytime post-index; or (2) high treatment adherence (defined as PDC >90%) with treatment discontinuation occurring >12 months post-index.
All-cause HRU included:
- Inpatient admission
- Inpatient days
- Emergency department visits
- Outpatient visits
Measured: 2 years
CML-related HRU per Patient per Year in NOPT Patients Compared to Reference Subgroup
The reference subgroup was defined based on the following treatment adherence and persistence criteria denoting potential indicators of TKI treatment success based on observations from the clinical practice: (1) high treatment adherence (defined as PDC >90%) with no observed treatment discontinuation anytime post-index; or (2) high treatment adherence (defined as PDC >90%) with treatment discontinuation occurring >12 months post-index.
All-cause HRU included:
- Inpatient admission
- Inpatient days
- Emergency department visits
- Outpatient visits
Measured: 2 years
All-cause Direct Healthcare Costs per Patient per Year in NOPT Patients Compared to Reference Subgroup
The reference subgroup was defined based on the following treatment adherence and persistence criteria denoting potential indicators of TKI treatment success based on observations from the clinical practice: (1) high treatment adherence (defined as PDC >90%) with no observed treatment discontinuation anytime post-index; or (2) high treatment adherence (defined as PDC >90%) with treatment discontinuation occurring >12 months post-index.
All-cause direct healthcare costs included:
- Total medical costs
- Inpatient costs
- Outpatient costs
- Emergency department costs
Measured: 2 years
CML-related Direct Healthcare Costs per Patient per Year in NOPT Patients Compared to Reference Subgroup
The reference subgroup was defined based on the following treatment adherence and persistence criteria denoting potential indicators of TKI treatment success based on observations from the clinical practice: (1) high treatment adherence (defined as PDC >90%) with no observed treatment discontinuation anytime post-index; or (2) high treatment adherence (defined as PDC >90%) with treatment discontinuation occurring >12 months post-index.
All-cause direct healthcare costs included:
- Total medical costs
- Inpatient costs
- Outpatient costs
- Emergency department costs
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2024
- Study start23 May 2023
- Primary completion30 Nov 2023
- Study completion30 Nov 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Myeloid LeukemiaStudies 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
- 2,043 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
- NCT06692803
- Sponsor's protocol ID
- CABL001A0US04
- Acronym
- ABL-2022-03
- Status last verified
- Nov 2024
- First submitted
- 15 Nov 2024
- Last updated
- 18 Nov 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real-World Treatment Patterns and Outcomes Among Patients With Chronic Myeloid Leukemia in Earlier Lines of Therapy (ABL-2022-03)
- ClinicalTrials.gov ID
- NCT06692803
- Acronym / sponsor ID
- ABL-2022-03 / CABL001A0US04
- Conditions
- Chronic Myeloid Leukemia
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 2,043 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 23 May 2023 (actual)
- Primary completion
- 30 Nov 2023 (actual)
- Study completion
- 30 Nov 2023 (actual)
- First posted
- 18 Nov 2024 (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.
This was a retrospective, non-interventional, observational cohort study using Optum's de-identified Clinformatics® Data Mart Database. Adult patients newly diagnosed with chronic myeloid leukemia (CML) treated with tyrosine kinase inhibitors (TKIs) were identified using the Optum database and classified into the following cohorts:
- First treatment cohort: Patients newly diagnosed with CML who received first treatment with a TKI.
- Second treatment cohort: Patients from first treatment cohort with a subsequent line of therapy (i.e., second treatment) with a TKI.
The observation period spanned from the start of data availability (i.e., 01 January 2007) to the earliest of end of data (i.e., 30 June 2022), end of continuous health plan enrollment, or death (if available). The index date was defined as the first treatment initiation for the first treatment TKI cohort and as the second treatment initiation for the second treatment TKI cohort. The baseline period consisted of the 6 months prior to the index date. The follow-up period started on the index date and ended at the earliest of end of observation period or hematopoietic stem cell transplantation (HSCT).
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| First Treatment Cohort | – | –Patients newly diagnosed with CML who received first treatment with imatinib, dasatinib, nilotinib, or bosutinib. |
| Second Treatment Cohort | – | –Patients from first treatment cohort with a subsequent line of therapy (i.e., second treatment) with imatinib, dasatinib, nilotinib, bosutinib, or ponatinib. |
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 (16 listed)
- Adult patients who initiated their first treatment with imatinib, dasatinib, nilotinib, or bosutinib (conditional on Food and Drug Administration (FDA) approval dates) with 6 months continuous health plan enrollment prior to the first prescription fill date.
- Patients with 2 or more diagnoses for CML (adult as of the first diagnosis for CML).
- Patients had index date on or after first CML diagnosis.
- Patients had no diagnoses for CML remission/relapse prior to index date.
- Patients had no gastrointestinal stomach tumor (GIST) or chronic myelomonocytic leukemia (CMML) at any time.
Key exclusion criteria (0 listed)
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
Treatment Patterns
Time frame: Up to approximately 10 years
Tyrosine kinase inhibitor (TKI) treatment management patterns in CML patients on first line or second line TKI were assessed.
17 secondary outcomes
Proportion of Days Covered (PDC)
Time frame: Up to approximately 10 years
Number of Patients per PDC Category
Time frame: Up to approximately 10 years
Time to Treatment Discontinuation
Time frame: Up to approximately 10 years
Time to Treatment Switch
Time frame: Up to approximately 10 years
Time to First Treatment Interruption
Time frame: Up to approximately 10 years
Time to First Dose Reduction
Time frame: Up to approximately 10 years
Rate of Treatment Discontinuation
Time frame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
Rate of Treatment Switching
Time frame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
Rate of Treatment Interruption
Time frame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
Rate of Dose Reduction
Time frame: Months 1, 3, 6, 9, 12, 18, and years 2, 3, 4
Number of Patients With Non-optimal Treatment (NOPT)
Time frame: Up to approximately 10 years
Number of Patients With NOPT by First Generation TKI
Time frame: Up to approximately 10 years
Number of Patients With NOPT by Second Generation TKI
Time frame: Up to approximately 10 years
All-cause Healthcare Resource Utilization (HRU) per Patient per Year in NOPT Patients Compared to Reference Subgroup
Time frame: 2 years
CML-related HRU per Patient per Year in NOPT Patients Compared to Reference Subgroup
Time frame: 2 years
All-cause Direct Healthcare Costs per Patient per Year in NOPT Patients Compared to Reference Subgroup
Time frame: 2 years
CML-related Direct Healthcare Costs per Patient per Year in NOPT Patients Compared to Reference Subgroup
Time frame: 2 years
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 18 Nov 2024.
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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 Chronic Myeloid Leukemia, 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 16 inclusion and 0 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 18 Nov 2024. 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?
Treatment Patterns (time frame: Up to approximately 10 years). 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 18 Nov 2024.