Chronic Myeloid LeukaemiaNCT05893836
Real-World Disease Management and Outcomes in Chronic Myeloid Leukaemia
An observational study of Chronic Myeloid Leukaemia, run by Novartis Pharmaceuticals at 1 site in United Kingdom.
Trial summary
Chronic myeloid leukaemia (CML) diagnosis is based on the demonstration of a BCR-ABL fusion transcript expressed by the Philadelphia (Ph) chromosome by RQ-PCR and/or the demonstration of t(9;22)(q34;q11) by conventional karyotyping or interphase FISH. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Myeloid Leukaemia
- How many people
- 555 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 27 Apr 2021to 30 Jun 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 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 (18+ years) patients newly diagnosed with CML in chronic phase (ICD-O-3: 9875/3) by HMDS between 1st September, 2004 to 31st August, 2019 whilst resident in the HMRN region and treated within the Network.
Who cannot join
- None specified.
Trial locations and contacts
1 site in 1 country.
About this study
Chronic myeloid leukaemia (CML) diagnosis is based on the demonstration of a BCR-ABL fusion transcript expressed by the Philadelphia (Ph) chromosome by RQ-PCR and/or the demonstration of t(9;22)(q34;q11) by conventional karyotyping or interphase FISH. As per standard practice, response to therapy is monitored using either molecular or cytogenetic tests or both; specifically, patients are monitored by quantitative PCR on peripheral blood, supplemented by bone marrow karyotyping if it was clinically indicated. ABL kinase mutational analysis is carried out when the transcript ratio has increased over two sequential samples or on clinical demand. Testing for T315I mutation is also performed for patients who fail to respond to first line TKI and all patients who acquire TKI resistance over the course of their treatment.
Data collection is initiated six months after date of diagnosis; research nurses working to agreed operating procedures and data standards visit each of the 14 hospitals in the region and abstract a core clinical dataset from the patients' medical records. The information collected includes demographic details, baseline blood count data and first line treatment. All details are abstracted onto structured forms and entered onto the web-based system, which integrates Haematological Malignancy Research Network (HMRN) and Haematological Malignancy Diagnostic Service (HMDS) data. An important feature of data acquisition is the emphasis on primary source information; data from radiology reports, blood tests, clinical examination, and clinician summaries are recorded, enabling embedded algorithms in the database system to automatically generate stage and prognostic scores. Further data abstraction from the medical records has been undertaken to capture information on subsequent treatment lines. Information on date and cause of death were obtained from the National Health Service (NHS) Central Register.
- Conditions
- Chronic Myeloid Leukaemia
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients who received tyrosine kinase inhibitors (TKIs), by treatment llne
Measured: Up to 18 years
Year treatment started, by TKI
Measured: Up to 18 years
Secondary outcomes (8)
Number of patients with response to TKIs
Disease response was defined as either a major molecular response (MMR, ≤ 0.1% BCR-ABL1) or as an MR2, which is a molecular response (MR, ≤ 1.0% BCR-ABL1), or complete cytogenetic remission (CCyR).
Measured: Up to 12 months
Time to response to TKIs
Measured: Up to 12 months
Reason for switching TKI
Measured: Up to 12 months
Number of patients tested for T315I mutation by treatment line
Measured: Up to 12 months
Overall survival (OS)
OS was defined as the time (in years) from initiation of treatment (i.e., the index date) to death (any cause).
Measured: Up to 10 years
Progression-free survival
PFS was defined from the initiation of treatment (i.e., the index date) to the earliest documentation of disease progression to accelerated phase/blast crisis (AP/BC) or date of death from any cause.
Measured: Up to 10 years
Time to treatment discontinuation
Measured: Up to 10 years
Relative survival by treatment line
Relative survival (RS) was estimated to examine the CML-specific mortality rate. The Stata program strel (v1.2.7) was used to estimate RS and corresponding 95% Confidence Intervals (95%CI); with age and sex-specific background mortality rates being obtained from national life tables.
Measured: Up to 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jun 2023
- Study start27 Apr 2021
- Primary completion30 Jun 2021
- Study completion30 Jun 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Myeloid LeukaemiaStudies 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
- 555 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
- NCT05893836
- Sponsor's protocol ID
- CABL001AGB01
- Status last verified
- Jun 2023
- First submitted
- 17 May 2023
- Last updated
- 8 Jun 2023
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real-World Disease Management and Outcomes in Chronic Myeloid Leukaemia
- ClinicalTrials.gov ID
- NCT05893836
- Acronym / sponsor ID
- CABL001AGB01
- Conditions
- Chronic Myeloid Leukaemia
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 555 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 27 Apr 2021 (actual)
- Primary completion
- 30 Jun 2021 (actual)
- Study completion
- 30 Jun 2021 (actual)
- First posted
- 8 Jun 2023 (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.
Chronic myeloid leukaemia (CML) diagnosis is based on the demonstration of a BCR-ABL fusion transcript expressed by the Philadelphia (Ph) chromosome by RQ-PCR and/or the demonstration of t(9;22)(q34;q11) by conventional karyotyping or interphase FISH. As per standard practice, response to therapy is monitored using either molecular or cytogenetic tests or both; specifically, patients are monitored by quantitative PCR on peripheral blood, supplemented by bone marrow karyotyping if it was clinically indicated. ABL kinase mutational analysis is carried out when the transcript ratio has increased over two sequential samples or on clinical demand. Testing for T315I mutation is also performed for patients who fail to respond to first line TKI and all patients who acquire TKI resistance over the course of their treatment.
Data collection is initiated six months after date of diagnosis; research nurses working to agreed operating procedures and data standards visit each of the 14 hospitals in the region and abstract a core clinical dataset from the patients' medical records. The information collected includes demographic details, baseline blood count data and first line treatment. All details are abstracted onto structured forms and entered onto the web-based system, which integrates Haematological Malignancy Research Network (HMRN) and Haematological Malignancy Diagnostic Service (HMDS) data. An important feature of data acquisition is the emphasis on primary source information; data from radiology reports, blood tests, clinical examination, and clinician summaries are recorded, enabling embedded algorithms in the database system to automatically generate stage and prognostic scores. Further data abstraction from the medical records has been undertaken to capture information on subsequent treatment lines. Information on date and cause of death were obtained from the National Health Service (NHS) Central Register.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
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 (1 listed)
- Adult (18+ years) patients newly diagnosed with CML in chronic phase (ICD-O-3: 9875/3) by HMDS between 1st September, 2004 to 31st August, 2019 whilst resident in the HMRN region and treated within the Network.
Key exclusion criteria (1 listed)
- None specified.
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 who received tyrosine kinase inhibitors (TKIs), by treatment llne
Time frame: Up to 18 years
Year treatment started, by TKI
Time frame: Up to 18 years
8 secondary outcomes
Number of patients with response to TKIs
Time frame: Up to 12 months
Time to response to TKIs
Time frame: Up to 12 months
Reason for switching TKI
Time frame: Up to 12 months
Number of patients tested for T315I mutation by treatment line
Time frame: Up to 12 months
Overall survival (OS)
Time frame: Up to 10 years
Progression-free survival
Time frame: Up to 10 years
Time to treatment discontinuation
Time frame: Up to 10 years
Relative survival by treatment line
Time frame: Up to 10 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 Jun 2023.
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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 Leukaemia, run by Novartis Pharmaceuticals at 1 site in United Kingdom.
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 1 inclusion and 1 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 Jun 2023. 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 Kingdom. 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 who received tyrosine kinase inhibitors (TKIs), by treatment llne (time frame: Up to 18 years). 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 8 Jun 2023.