Chronic Myelogenous Leukemia, BCR-ABL1 PositiveCAPNCT06945146
Genomics Study in CML Patients With Ponatinib Treatment
An observational study of Chronic Myelogenous Leukemia, BCR-ABL1 Positive, run by Dong-Wook Kim at 1 site in South Korea.
Trial summary
This study will evaluate whether responsiveness and adverse events (AEs) to second-line or later ponatinib treatment are associated with genetic variations as measured by real-time quantitative polymerase chain reaction (qRT-PCR) and next-generation sequencing (NGS) in patients with CML of any stage who failed prior… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- What is being tested?
- Ponatinib
- How many people
- 65 participants
- Where
- 1 siteSouth Korea
- Study dates
- 1 Sep 2020to 25 Sep 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: The study will involve 100 patients with BCR-ABL1-positive CML who failed to prior targeted therapy other than ponatinib. Patients receiving ponatinib 45 mg once daily as a second-line or later therapy will be enrolled.
Who may be able to join
- Patients who are willing to and capable of providing informed consent
- Adults with BCR-ABL1-positive CML
- Males and females aged 18 years and above
- Adequate organ function
- Subjects who were resistant or intolerant to prior targeted therapy other than ponatinib and with an indication for ponatinib treatment according to the acceptance criteria by the Ministry of Food and Drug Safety (MFDS)
- Women of childbearing potential (WOCBP) should have a negative serum or urine pregnancy test (with a sensitivity of at least 25 IU/L or equivalent to HCG) within 24 hours before initiating ponatinib treatment
- Female subjects who are not breastfeeding
Who cannot join
- Patients who were previously treated with ponatinib
- Patients aged below 18 years of age
- Diagnosis of severe comorbidity at baseline
- Any other cancers within 3 years
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The Comprehensive Assessment of BCR-ABL1 Gene Expression and Genetic Variations by qRT-PCR and NGS Assays in Chronic Myeloid Leukemia Patients Who Are Treated With Ponatinib (CAP Study)
This study will evaluate whether responsiveness and adverse events (AEs) to second-line or later ponatinib treatment are associated with genetic variations as measured by real-time quantitative polymerase chain reaction (qRT-PCR) and next-generation sequencing (NGS) in patients with CML of any stage who failed prior multiple targeted therapies except ponatinib.
Read the detailed description
Ponatinib treatment will be initiated per usual treatment procedure at 45 mg once daily p.o., which will be gradually decreased to 30 mg and 15 mg according to the predefined criteria based on responsiveness to treatment and AEs in the course of the treatment. A total of 100 subjects will be enrolled within 24 months after the first subject enrollment, and ponatinib treatment will continue for 24 months after the initial dosing of ponatinib in each enrolled subject. Routine ponatinib treatment will continue at the investigator's discretion until disease progression, the occurrence of unacceptable toxicity, subject's withdrawal of consent, or occurrence of any reason for discontinuation specified in the protocol.
All molecular analysis samples will be collected, transferred, and managed by the Catholic Leukemia Research Institute, and NGS will be performed.
<Eligibility>
- Adults with BCR-ABL1-positive CML
- Subjects who were resistant or intolerant to prior targeted therapy other than ponatinib and with an indication for ponatinib treatment according to the acceptance criteria by the Ministry of Food and Drug Safety (MFDS)
- Women of childbearing potential (WOCBP) should have a negative serum or urine pregnancy test (with a sensitivity of at least 25 IU/L or equivalent to HCG) within 24 hours before initiating ponatinib treatment
- Written informed consent to ponatinib treatment
<Outcome Measures>
- Primary endpoint
- 24 months dynamics of BCR-ABL1 gene expression by qRT-PCR
- Secondary endpoints
- Type and frequency of novel genetic variations (mutations, gene expressions, CNV, INDEL, etc.)
- Cobll1/GCA/novel gene network identification: functional tests using Western blot/Knock-down assay
- Safety endpoints : To explore the dynamics of adverse events according to dose changes up to 24 months
- Frequency and severity of skin rash, fever, hypertension, pancreatitis and vascular events as common adverse events
- Frequency and severity of rare adverse events
- Treatment intolerance is defined as recurrence of a Grade ≥3 hematologic AE, or a Grade ≥2 non-hematologic AE requiring permanent discontinuation of ponatinib per protocol despite dose reduction
- Being tested
- Ponatinib
- Keywords
- ponatinib, tyrosine kinase inhibitor, resistance, intolerance, genetic variation, next generation sequencing
- 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.
PonatinibDrug
Also called Iclusig
Ponatinib treatment will be initiated per usual treatment procedure at 45 mg once daily p.o., which will be gradually decreased to 30 mg and 15 mg according to the predefined criteria based on responsiveness to treatment and AEs in the course of the treatment.
| Study group | Role | What they receive |
|---|---|---|
| CML patients failed to TKIs except Ponatinib | The study will involve 100 patients with BCR-ABL1-positive CML who failed to prior targeted therapy other than ponatinib. Patients receiving ponatinib 45 mg once daily as a second-line or later therapy will be enrolled. Receives: Drug: Ponatinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dynamics of BCR-ABL1 gene expression by qRT-PCR
To explore the dynamics of BCR-ABL1 kinetics by Ponatinib
Measured: 24 months
Secondary outcomes (2)
Type and frequency of novel genetic variations (mutations, gene expressions, CNV, INDEL, etc.)
To explore the dynamics of various genetic variations in Ponatinib failed patients
Measured: 24 months
Cobll1/GCA/novel gene network identification: functional tests using Western blot/Knock-down assay
To identify novel genetic networks in Ponatinib failed patients
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2025
- Study start1 Sep 2020
- Primary completion31 Aug 2024
- Study completion25 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Myelogenous Leukemia, BCR-ABL1 PositiveStudies of the same condition, open and under way.
- 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
- Prospective
- Enrolment
- 65 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dong-Wook Kim
- Sponsor type
- University, hospital or other
- Collaborators
- Ulsan National Institute of Science and Technology, Takeda
- Responsible party
- Sponsor-investigator, Dong-Wook Kim, Eulji University Hospital
- Investigators
- Dong-Wook Kim, MD, PhD, Principal investigator, Eulji University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06945146
- Sponsor's protocol ID
- KC20MISI0507
- Acronym
- CAP
- Status last verified
- Dec 2024
- First submitted
- 18 Apr 2025
- Last updated
- 25 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 25 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Comprehensive Assessment of BCR-ABL1 Gene Expression and Genetic Variations by qRT-PCR and NGS Assays in Chronic Myeloid Leukemia Patients Who Are Treated With Ponatinib (CAP Study)
- ClinicalTrials.gov ID
- NCT06945146
- Acronym / sponsor ID
- CAP / KC20MISI0507
- Keywords
- ponatinib, tyrosine kinase inhibitor, resistance, intolerance, genetic variation, next generation sequencing
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 65 participants (actual)
- Lead sponsor
- Dong-Wook KimUniversity, hospital or other
- Collaborators
- Ulsan National Institute of Science and Technology, Takeda
- Study start
- 1 Sep 2020 (actual)
- Primary completion
- 31 Aug 2024 (actual)
- Study completion
- 25 Sep 2024 (actual)
- First posted
- 25 Apr 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.
This study will evaluate whether responsiveness and adverse events (AEs) to second-line or later ponatinib treatment are associated with genetic variations as measured by real-time quantitative polymerase chain reaction (qRT-PCR) and next-generation sequencing (NGS) in patients with CML of any stage who failed prior multiple targeted therapies except ponatinib.
Detailed description
Ponatinib treatment will be initiated per usual treatment procedure at 45 mg once daily p.o., which will be gradually decreased to 30 mg and 15 mg according to the predefined criteria based on responsiveness to treatment and AEs in the course of the treatment. A total of 100 subjects will be enrolled within 24 months after the first subject enrollment, and ponatinib treatment will continue for 24 months after the initial dosing of ponatinib in each enrolled subject. Routine ponatinib treatment will continue at the investigator's discretion until disease progression, the occurrence of unacceptable toxicity, subject's withdrawal of consent, or occurrence of any reason for discontinuation specified in the protocol.
All molecular analysis samples will be collected, transferred, and managed by the Catholic Leukemia Research Institute, and NGS will be performed.
<Eligibility>
- Adults with BCR-ABL1-positive CML
- Subjects who were resistant or intolerant to prior targeted therapy other than ponatinib and with an indication for ponatinib treatment according to the acceptance criteria by the Ministry of Food and Drug Safety (MFDS)
- Women of childbearing potential (WOCBP) should have a negative serum or urine pregnancy test (with a sensitivity of at least 25 IU/L or equivalent to HCG) within 24 hours before initiating ponatinib treatment
- Written informed consent to ponatinib treatment
<Outcome Measures>
- Primary endpoint
- 24 months dynamics of BCR-ABL1 gene expression by qRT-PCR
- Secondary endpoints
- Type and frequency of novel genetic variations (mutations, gene expressions, CNV, INDEL, etc.)
- Cobll1/GCA/novel gene network identification: functional tests using Western blot/Knock-down assay
- Safety endpoints : To explore the dynamics of adverse events according to dose changes up to 24 months
- Frequency and severity of skin rash, fever, hypertension, pancreatitis and vascular events as common adverse events
- Frequency and severity of rare adverse events
- Treatment intolerance is defined as recurrence of a Grade ≥3 hematologic AE, or a Grade ≥2 non-hematologic AE requiring permanent discontinuation of ponatinib per protocol despite dose reduction
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CML patients failed to TKIs except Ponatinib | – | Drug: PonatinibThe study will involve 100 patients with BCR-ABL1-positive CML who failed to prior targeted therapy other than ponatinib. Patients receiving ponatinib 45 mg once daily as a second-line or later therapy will be enrolled. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The study will involve 100 patients with BCR-ABL1-positive CML who failed to prior targeted therapy other than ponatinib. Patients receiving ponatinib 45 mg once daily as a second-line or later therapy will be enrolled.
Key inclusion criteria (7 listed)
- Patients who are willing to and capable of providing informed consent
- Adults with BCR-ABL1-positive CML
- Subjects who were resistant or intolerant to prior targeted therapy other than ponatinib and with an indication for ponatinib treatment according to the acceptance criteria by the Ministry of Food and Drug Safety (MFDS)
- Women of childbearing potential (WOCBP) should have a negative serum or urine pregnancy test (with a sensitivity of at least 25 IU/L or equivalent to HCG) within 24 hours before initiating ponatinib treatment
- Female subjects who are not breastfeeding
Key exclusion criteria (4 listed)
- Patients who were previously treated with ponatinib
- Patients aged below 18 years of age
- Diagnosis of severe comorbidity at baseline
- Any other cancers within 3 years
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 |
|---|---|---|
| PonatinibAlso: IclusigPonatinib treatment will be initiated per usual treatment procedure at 45 mg once daily p.o., which will be gradually decreased to 30 mg and 15 mg according to the predefined criteria based on responsiveness to treatment and AEs in the course of the treatment. | Drug | CML patients failed to TKIs except Ponatinib |
Primary outcome
Dynamics of BCR-ABL1 gene expression by qRT-PCR
Time frame: 24 months
To explore the dynamics of BCR-ABL1 kinetics by Ponatinib
2 secondary outcomes
Type and frequency of novel genetic variations (mutations, gene expressions, CNV, INDEL, etc.)
Time frame: 24 months
Cobll1/GCA/novel gene network identification: functional tests using Western blot/Knock-down assay
Time frame: 24 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 25 Apr 2025.
- Individual participant dataSharing planned: Undecided
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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.
Dong-Wook Kim, MD, PhD principal investigator
Eulji University
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 Myelogenous Leukemia, BCR-ABL1 Positive, run by Dong-Wook Kim at 1 site in South Korea.
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 7 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 25 Apr 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 South Korea. 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?
Dynamics of BCR-ABL1 gene expression by qRT-PCR (time frame: 24 months). 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 25 Apr 2025.