Chronic Myeloid LeukemiaNCT05476562
Study Conducted Among Patients With CML
An observational study of Chronic Myeloid Leukemia, run by Novartis Pharmaceuticals at 1 site in United States.
Trial summary
Retrospective, non-interventional observational cohort study conducted among patients with CML. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Chronic Myeloid Leukemia
- What is being tested?
- Tyrosine Kinase Inhibitors (TKIs)
- How many people
- 3,234 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Mar 2021to 15 Oct 2021
- 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 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The study was conducted among patients with CML.
Who may be able to join
- Patients were selected for the analysis of later lines of therapy in commercial claims (i.e., were previously treated with TKIs, who are relapsed/refractory to/intolerant of TKIs) if they met the following criteria:
- Had at least one diagnosis for CML, with first CML diagnosis observed in claims on or after May 10, 2001, the date of FDA approval for imatinib
- Were at least 18 years of age as of the first CML diagnosis
- Started a first line therapy for CML with imatinib, dasatinib, nilotinib, or bosutinib (conditional on FDA-approval dates)
- Initiated first line therapy within a maximum of 1 month prior to the first diagnosis for CML or a maximum 3 months following the first diagnosis for CML
- Had continuous health plan enrollment (pharmacy and medical benefits) from the washout period to at least 12 months following the first CML diagnosis
Who cannot join
- Patients had a diagnosis for CML remission or relapse anytime prior to first line therapy
- Patients had a medical claim associated with a clinical trial during the washout period up to the end of the observation period
- Patients had an HSCT during the washout period up to the first line therapy initiation
- Patients had chemotherapy treatment (except hydroxyurea) during the washout period up to the first line therapy initiation
Trial locations and contacts
1 site in 1 country.
United States1 site
Novartis Investigative Site
East Hanover, New Jersey, 07936-1080
About this study
Official title: Real-World Treatment Patterns, Healthcare Resource Utilization and Associated Costs Among Patients With Chronic Myeloid Leukemia in Later Lines of Therapy
Retrospective, non-interventional observational cohort study conducted among patients with CML.
Read the detailed description
A retrospective, non-interventional cohort study was used to address the study objectives. A cohort of adult patients with CML who were treated with TKIs were identified using the IBM® MarketScan® Commercial and Medicare Supplemental databases (commercial claims; the MarketScan database) to have a better understanding of real-world treatment patterns, HRU and healthcare costs among patients with CML treated with later lines of therapy (i.e., third line or later).
For Phase I, the IBM® MarketScan® Commercial Claims and Encounters and Medicare Supplemental Databases were used (commercial claims). The commercial claims covered the period from 01/01/2001 to 06/30/2019.
The study consisted of the following periods:
- The baseline period was defined as the 6-month period before the first line therapy initiation for CML.
- The observation period was defined as the period of at least 12 months from the first CML diagnosis to the end of data availability or end of health plan coverage, whichever occurs first; the observation period varied by patient.
- Conditions
- Chronic Myeloid Leukemia
- Being tested
- Tyrosine Kinase Inhibitors (TKIs)
- Keywords
- Chronic myeloid leukemia,, tyrosine kinase inhibitors,, treatment patterns,, HRU,, 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.
Tyrosine Kinase Inhibitors (TKIs)Other
The study included patients with CML who were previously treated with TKIs, who are relapsed/refractory to/intolerant of TKIs on third or later lines of therapy in the US.
| Study group | Role | What they receive |
|---|---|---|
| First-line Therapy | A cohort of adult patients with CML who were treated with TKIs were identified using the IBM® MarketScan® Commercial and Medicare Supplemental databases (commercial claims; the MarketScan database) Receives: Other: Tyrosine Kinase Inhibitors (TKIs) | |
| Second-line Therapy | A cohort of adult patients with CML who were treated with TKIs were identified using the IBM® MarketScan® Commercial and Medicare Supplemental databases (commercial claims; the MarketScan database) Receives: Other: Tyrosine Kinase Inhibitors (TKIs) | |
| Third-line Therapy | A cohort of adult patients with CML who were treated with TKIs were identified using the IBM® MarketScan® Commercial and Medicare Supplemental databases (commercial claims; the MarketScan database) Receives: Other: Tyrosine Kinase Inhibitors (TKIs) | |
| Fourth-line Therapy | A cohort of adult patients with CML who were treated with TKIs were identified using the IBM® MarketScan® Commercial and Medicare Supplemental databases (commercial claims; the MarketScan database) Receives: Other: Tyrosine Kinase Inhibitors (TKIs) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients across all lines of therapy
Number of patients across all lines of therapy were reported to evaluate treatment sequences among the patients.
Measured: throughout the study, approximately 20 years
Number of patients with the use of pre-treatment hydroxyurea
Number of patients with the use of pre-treatment hydroxyurea were reported to evaluate treatment patterns in patients.
Measured: throughout the study, approximately 20 years
Number of patients Treatment received and initial dose at each line
Number of patients Treatment received and initial dose at each line were reported to evaluate treatment patterns in patients.
Measured: throughout the study, approximately 20 years
Number of patients as per the calendar year of line of therapy initiation
Number of patients as per the calendar year of line of therapy initiation were reported to evaluate treatment patterns in patients.
Measured: throughout the study, approximately 20 years
Duration of the line of therapy
Duration of the line of therapy was reported to evaluate treatment patterns in patients.
Measured: throughout the study, approximately 20 years
Treatment-free period among those with an observed subsequent line of therapy
Treatment-free period among those with an observed subsequent line of therapy was reported to evaluate treatment patterns in patients.
Measured: throughout the study, approximately 20 years
Number of patients who discontinued treatment
Number of patients who discontinued treatment were reported to evaluate treatment patterns in patients.
Measured: throughout the study, approximately 20 years
Number of patients: All cause Health Resource Utilization among patients with three lines of therapy or more
Number of patients: All cause Health Resource Utilization were reported to estimate HRU among patients with three lines of therapy or more.
Measured: throughout the study, approximately 20 years
Healthcare costs among patients with three lines of therapy or more
Healthcare costs among patients with three lines of therapy or more were reported.
Measured: throughout the study, approximately 20 years
Secondary outcomes (3)
Prevalence of second and third or later lines of therapy
Prevalence was defined as the number of cases alive per year of patients with CML who were currently or previously treated on later lines of therapy (third line or later), by calendar year from 2006 to 2018
Measured: Calendar year 2006 to 2018, approximately 12 years
Healthcare costs among patients with earlier lines of therapy
Healthcare costs among patients with earlier lines of therapy were reported.
Measured: throughout the study, approximately 20 years
Number of patients: All cause Health Resource Utilization among patients with earlier lines of therapy
Number of patients: All cause Health Resource Utilization among patients with earlier lines of therapy were reported.
Measured: throughout the study, approximately 20 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jul 2022
- Study start8 Mar 2021
- Primary completion15 Oct 2021
- Study completion15 Oct 2021
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
- 3,234 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
- NCT05476562
- Sponsor's protocol ID
- CABL001AUS07
- Status last verified
- Nov 2022
- First submitted
- 25 Jul 2022
- Last updated
- 8 Nov 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real-World Treatment Patterns, Healthcare Resource Utilization and Associated Costs Among Patients With Chronic Myeloid Leukemia in Later Lines of Therapy
- ClinicalTrials.gov ID
- NCT05476562
- Acronym / sponsor ID
- CABL001AUS07
- Conditions
- Chronic Myeloid Leukemia
- Keywords
- Chronic myeloid leukemia,, tyrosine kinase inhibitors,, treatment patterns,, HRU,, costs
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 3,234 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 8 Mar 2021 (actual)
- Primary completion
- 15 Oct 2021 (actual)
- Study completion
- 15 Oct 2021 (actual)
- First posted
- 27 Jul 2022 (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.
Retrospective, non-interventional observational cohort study conducted among patients with CML.
Detailed description
A retrospective, non-interventional cohort study was used to address the study objectives. A cohort of adult patients with CML who were treated with TKIs were identified using the IBM® MarketScan® Commercial and Medicare Supplemental databases (commercial claims; the MarketScan database) to have a better understanding of real-world treatment patterns, HRU and healthcare costs among patients with CML treated with later lines of therapy (i.e., third line or later).
For Phase I, the IBM® MarketScan® Commercial Claims and Encounters and Medicare Supplemental Databases were used (commercial claims). The commercial claims covered the period from 01/01/2001 to 06/30/2019.
The study consisted of the following periods:
- The baseline period was defined as the 6-month period before the first line therapy initiation for CML.
- The observation period was defined as the period of at least 12 months from the first CML diagnosis to the end of data availability or end of health plan coverage, whichever occurs first; the observation period varied by patient.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| First-line Therapy | – | Other: Tyrosine Kinase Inhibitors (TKIs)A cohort of adult patients with CML who were treated with TKIs were identified using the IBM® MarketScan® Commercial and Medicare Supplemental databases (commercial claims; the MarketScan database) |
| Second-line Therapy | – | Other: Tyrosine Kinase Inhibitors (TKIs)A cohort of adult patients with CML who were treated with TKIs were identified using the IBM® MarketScan® Commercial and Medicare Supplemental databases (commercial claims; the MarketScan database) |
| Third-line Therapy | – | Other: Tyrosine Kinase Inhibitors (TKIs)A cohort of adult patients with CML who were treated with TKIs were identified using the IBM® MarketScan® Commercial and Medicare Supplemental databases (commercial claims; the MarketScan database) |
| Fourth-line Therapy | – | Other: Tyrosine Kinase Inhibitors (TKIs)A cohort of adult patients with CML who were treated with TKIs were identified using the IBM® MarketScan® Commercial and Medicare Supplemental databases (commercial claims; the MarketScan database) |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The study was conducted among patients with CML.
Key inclusion criteria (6 listed)
- Patients were selected for the analysis of later lines of therapy in commercial claims (i.e., were previously treated with TKIs, who are relapsed/refractory to/intolerant of TKIs) if they met the following criteria:
- Had at least one diagnosis for CML, with first CML diagnosis observed in claims on or after May 10, 2001, the date of FDA approval for imatinib
- Were at least 18 years of age as of the first CML diagnosis
- Started a first line therapy for CML with imatinib, dasatinib, nilotinib, or bosutinib (conditional on FDA-approval dates)
- Initiated first line therapy within a maximum of 1 month prior to the first diagnosis for CML or a maximum 3 months following the first diagnosis for CML
Key exclusion criteria (4 listed)
- Patients had a diagnosis for CML remission or relapse anytime prior to first line therapy
- Patients had a medical claim associated with a clinical trial during the washout period up to the end of the observation period
- Patients had an HSCT during the washout period up to the first line therapy initiation
- Patients had chemotherapy treatment (except hydroxyurea) during the washout period up to the first line therapy initiation
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Tyrosine Kinase Inhibitors (TKIs)The study included patients with CML who were previously treated with TKIs, who are relapsed/refractory to/intolerant of TKIs on third or later lines of therapy in the US. | Other | First-line Therapy, Fourth-line Therapy, Second-line Therapy, Third-line Therapy |
Primary outcome
Number of patients across all lines of therapy
Time frame: throughout the study, approximately 20 years
Number of patients across all lines of therapy were reported to evaluate treatment sequences among the patients.
Number of patients with the use of pre-treatment hydroxyurea
Time frame: throughout the study, approximately 20 years
Number of patients with the use of pre-treatment hydroxyurea were reported to evaluate treatment patterns in patients.
Number of patients Treatment received and initial dose at each line
Time frame: throughout the study, approximately 20 years
Number of patients Treatment received and initial dose at each line were reported to evaluate treatment patterns in patients.
Number of patients as per the calendar year of line of therapy initiation
Time frame: throughout the study, approximately 20 years
Number of patients as per the calendar year of line of therapy initiation were reported to evaluate treatment patterns in patients.
Duration of the line of therapy
Time frame: throughout the study, approximately 20 years
Duration of the line of therapy was reported to evaluate treatment patterns in patients.
Treatment-free period among those with an observed subsequent line of therapy
Time frame: throughout the study, approximately 20 years
Treatment-free period among those with an observed subsequent line of therapy was reported to evaluate treatment patterns in patients.
Number of patients who discontinued treatment
Time frame: throughout the study, approximately 20 years
Number of patients who discontinued treatment were reported to evaluate treatment patterns in patients.
Number of patients: All cause Health Resource Utilization among patients with three lines of therapy or more
Time frame: throughout the study, approximately 20 years
Number of patients: All cause Health Resource Utilization were reported to estimate HRU among patients with three lines of therapy or more.
Healthcare costs among patients with three lines of therapy or more
Time frame: throughout the study, approximately 20 years
Healthcare costs among patients with three lines of therapy or more were reported.
3 secondary outcomes
Prevalence of second and third or later lines of therapy
Time frame: Calendar year 2006 to 2018, approximately 12 years
Healthcare costs among patients with earlier lines of therapy
Time frame: throughout the study, approximately 20 years
Number of patients: All cause Health Resource Utilization among patients with earlier lines of therapy
Time frame: throughout the study, approximately 20 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 8 Nov 2022.
- Results for CABL001AUS07 from the Novartis Clinical Trials WebsiteLink provided by the sponsor.
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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 6 inclusion and 4 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 8 Nov 2022. 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 across all lines of therapy (time frame: throughout the study, approximately 20 years). The study lists 9 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 8 Nov 2022.