Chronic Myeloid Leukemia, Chronic PhasePhase 2ALERTCMLNCT05143840
Asciminib as Initial Therapy for Patients With Chronic Myeloid Leukemia in Chronic Phase
A Phase 2 study testing Single Agent Asciminib, Low TKI and Elective Free Treatment in people with Chronic Myeloid Leukemia, Chronic Phase and Adult CML, run by University of Alabama at Birmingham at 8 sites in United States.
Trial summary
This study is a multicenter Phase 2, non-randomized, open-label single-group frontline study administering asciminib in patients with newly diagnosed Chronic Myeloid Leukemia-Chronic Phase (CML-CP). The aim of this study is to evaluate the efficacy and safety of asciminib in newly diagnosed CML-CP. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Myeloid Leukemia, Chronic Phase, Adult CML, Leukemia, Myeloid
- What is being tested?
- Single Agent Asciminib, Low TKI and Elective Free Treatment
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 8 sitesUnited States
- Study dates
- 22 Apr 2022to Feb 2032
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
Who may be able to join
- Age ≥18 years old
- Willing and able to give informed consent
- Newly diagnosed with CML in chronic phase within 6 months from confirmed diagnosis via bone marrow biopsy/aspirate and have either the b3a2 (e14a2) or b2a2 (e13a2) variants that give rise to the p210 BCR::ABL1 protein. Subtype classification whether b3a2 (e14a2) or b2a2 (e13a2) is not required for study eligibility.
- Minimal prior CML therapy with a TKI for less than or equal to 30 days. Treatment with hydroxyurea, busulfan, anagrelide or other non-specific chemotherapy agents is allowed with no time restrictions within the eligible time from diagnosis.
- ECOG performance status 0-2 (appendix 1)
- Adequate organ function:
Show all 22Show fewer
- AST and ALT < 3 times the institutional upper limit of normal (ULN)
- eGFR ≥ 30 mL/min as calculated using the 2021 chronic kidney disease epidemiology (CKD-EPI) creatinine equation (https://www.kidney.org/professionals/kdoqi/gfr_calculator)
- Total bilirubin < 1.5 times the institutional ULN or < 3.0 x the institutional ULN with Gilbert Syndrome (unless direct bilirubin is within normal limits)
- Adequately controlled blood pressure, defined as systolic blood pressure of <140 mmHq and diastolic of <90 mmHg, at the time of enrollment.
- Lipase ≤ 1.5 x ULN. For lipase > ULN - ≤ 1.5 x ULN, value should be considered not clinically significant and not associated with risk factors for acute pancreatitis.
- Creatine phosphokinase < 2.5 x ULN
- Female patients must meet one of the following:
- Postmenopausal for at least one year before the screening visit,
- Surgically sterile
- If they are of childbearing potential, agree to practice two effective methods of contraception from the time of signing of the informed consent form through 90 days after the last dose of study drug,
- Must also adhere to the guidelines of any treatment-specific pregnancy prevention program, if applicable
- Agree to practice true abstinence when this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence [e.g., calendar, ovulation, symptothermal, postovulation methods] and withdrawal are not acceptable contraception methods.)
- Male patients, even if surgically sterilized (i.e., status post vasectomy), must agree to one of the following:
- Practice effective barrier contraception during the entire study treatment period and through 90 days after the last study drug dose
- Must also adhere to the guidelines of any treatment-specific pregnancy prevention program, if applicable
- Agree to practice true abstinence when this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence [e.g., calendar, ovulation, symptothermal, postovulation methods] and withdrawal are not acceptable methods of contraception.)
Who cannot join
- Patients with accelerated or blast phase CML (refer to appendix 4)
- Active second malignancy requiring active treatment
- History of recent (within 12 months) acute pancreatitis or chronic pancreatitis
- Subjects who have previously received treatment with asciminib.
- Subjects with PLT count < 50,000 mm3 or ANC of < 500 mm3 or Hemoglobin < 8 g/dL
- Cardiac or cardiac repolarization abnormality, including any of the following:
Show all 19Show fewer
- History within 6 months prior to starting study treatment of myocardial infarction (MI), angina pectoris, coronary artery bypass graft (CABG)
- Clinically significant cardiac arrhythmias (e.g., ventricular tachycardia), complete left bundle branch block, high-grade AV block (e.g., bifascicular block, Mobitz type II and third degree AV block)
- QTcF at screening greater than or equal to 450 msec (male patients), greater than or equal to 460 msec (female patients) unless patient has a pacemaker
- Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome, or any of the following:
- Risk factors for Torsades de Pointes (TdP) including uncorrected hypokalemia or hypomagnesemia, history of cardiac failure, or history of clinically significant/symptomatic bradycardia ii. Concomitant medication(s) with a "Known risk of Torsades de Pointes" per wwwcrediblemeds.org/ that cannot be discontinued or replace 7 days prior to starting study drug by safe alternative medication.
- Inability to determine the QTcF interval
- Pregnant or lactating
- Taking a strong inhibitors or inducers of CYP3A4 or CYP3A4 substrates with narrow therapeutic index (refer to appendix 6) at time of enrollment
- Unable to comply with lab appointment schedule and PRO assessments
- Another investigational drug within 4 weeks of enrollment
- Any serious medical or psychiatric illness that could, in the investigator's opinion, interfere with the completion of treatment according to this protocol
- Patient has undergone a prior allogeneic stem cell transplant
- Known clinical history of active HBV infection
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Winship Cancer Institute Emory University
Atlanta, Georgia, 30322
Georgia Cancer Center at Augusta University
Augusta, Georgia, 30912
University of Alabama at Birmingham
Birmingham, Alabama, 35294
Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14263
Karmanos Cancer Institute
Detroit, Michigan, 48201
Froedtert Hospital & the Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Huntsman Cancer Institute
Salt Lake City, Utah, 84112
About this study
Official title: Asciminib as Initial Therapy With Addition of Lower Dose Tyrosine Kinase Inhibitors for Patients With Chronic Myeloid Leukemia Who do Not Achieve Optimal Response or a Deep Molecular Remission (ALERT CML)
This study is a multicenter Phase 2, non-randomized, open-label single-group frontline study administering asciminib in patients with newly diagnosed Chronic Myeloid Leukemia-Chronic Phase (CML-CP). The aim of this study is to evaluate the efficacy and safety of asciminib in newly diagnosed CML-CP. Patients will receive asciminib 80 mg orally once daily during the single asciminib phase. Response is determined by PCR (polymerase chain reaction) blood test during the study. Patients who have not achieved a response after 24 months (but no later than 36 months) of single agent asciminib will be offered the addition of a low dose tyrosine kinase inhibitor (low-TKI) namely dasatinib, imatinib, or nilotinib at the investigator's discretion. The following doses of the TKIs will be used:
- Dasatinib 50 mg daily
- Imatinib 300 mg daily
- Nilotinib 300 mg daily
Patients will discontinue study treatment if they experience disease progression, or unacceptable toxicity.
Read the detailed description
Asciminib is a potent allosteric inhibitor of BCR-ABL1 oncogene that confers resistance to tyrosine kinase inhibitors (TKIs). Asciminib has potential to combine with TKIs to prevent the emergence of BCR-ABL1 mutations, increasing the depth of molecular response in CML-CP patients. Anticipated enrollment is 50 subjects across sites.
Primary Objective:
To estimate the proportion of patients with previously untreated CML-CP who attain BCR::ABL1 <0.01% (MR4.0) IS by RQ-PCR with single agent asciminib therapy.
Secondary Objectives:
- To estimate the proportion of patients achieving molecular response at specific time points
- To estimate the time to molecular response
- To evaluate the duration of hematologic and molecular response to asciminib
- To define the time to progression and overall survival for patients with CML in early CP treated with asciminib
- To evaluate the safety profile of asciminib in patients with CML-CP
- To evaluate the development of ABL mutations for patients with CML in early CP treated with asciminib
- To analyze differences in response rates and in prognosis within different risk groups and patient characteristics
- To evaluate patient-reported outcomes in patients with CML receiving asciminib
- To investigate treatment-free remission after at least 2 years of sustained deep molecular remission for patients receiving single agent asciminib or combination (asciminib + low TKI)
Exploratory objectives:
- To evaluate the safety and efficacy of concomitant use of low TKI with asciminib in patients who have not achieved MR4.5.
- To evaluate the rate of successful treatment discontinuation for patients using the combination of asciminib and low TKI
- To evaluate the safety and efficacy of concomitant use of lowTKI with asciminib in patients who experience treatment failure at any time with single agent asciminib
- To evaluate the safety and efficacy of concomitant use of lowTKI with asciminib in patients who have not achieved an optimal response after 12 months of single agent asciminib
- Evaluate the role of Digital droplet PCR (ddPCR) in predicting TFR
- Evaluating the correlation between the gene expression signature of patients and the chances of achieving MMR and DMR
- Evaluate whether B, NK and T cells DNA mutation and RNA expression are relevant and whether they can predict response in patients with CML using single cell analysis.
Subjects must meet all inclusion criteria and none of the exclusion criteria of the study. No enrollment waivers will be granted. After successful screening, subjects will be enrolled and treatment will start within 7 days of enrollment. Eligible subjects will begin asciminib on cycle 1 day 1 of the trial. After 2 years (but no later than 3 years), subjects will be offered the addition of taking nilotinib, dasatinib, or imatinib with asciminib if a molecular response is not met (PCR blood test).
Duration of each participant is expected to take approximately 5 years on treatment and up to a total of 8 years if attempting treatment free remission.
Regimen Description
Asciminib 80 mg Oral Once a day 4 weeks (28 days) Nilotinib 300 mg* Oral Once a day 4 weeks (28 days) Dasatinib 50 mg* Oral Once a day 4 weeks (28 days) Imatinib 300 mg* Oral Once a day 4 weeks (28 days)
*Nilotinib, dasatinib, or imatinib will be taken if indicated.
Dose levels and dose modifications of the study drugs will be made per protocol.
- Conditions
- Chronic Myeloid Leukemia, Chronic Phase, Adult CML, Leukemia, Myeloid, Leukemia,Myeloid, Chronic
- Being tested
- Single Agent Asciminib, Low TKI, Elective Free Treatment
- Keywords
- Chronic Myeloid Leukemia, Adult CML, tyrosine kinase inhibitors, H. Jean Khoury Cure CML Consortium, HJKC3-0004
- Medical subject headings
- Leukemia, Myeloid, Chronic-Phase, Leukemia, Myeloid, 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.
Single Agent AsciminibDrug
Also called ABL001
taken orally once a day starting cycle 1 day 1 for up to 24 months during the single agent asciminib phase. Asciminib is a potent allosteric inhibitor of BCR::ABL1.
Low TKICombination product
Low dose tyrosine kinase inhibitor (lowTKI) (dasatinib 50 mg daily or imatinib 300 mg daily or nilotinib 300 mg daily) at investigators discretion, may be added to asciminib in the following situations:
- Patients who have treatment failure at any time based on ELN criteria (Appendix 7)
- Patients who have a warning response after 12 months of single agent asciminib based on ELN criteria (Appendix 7)
- Patients who have not achieved MR4.5 after 24 months, but no later than 36 months, of single agent asciminib.
Elective Free TreatmentOther
Once central eligibility has been obtained the patient should discontinue asciminib and if applicable lowTKI within 14 days.
| Study group | Role | What they receive |
|---|---|---|
| Single Agent Asciminib Arm | Experimental | Asciminib 80mg Asciminib will be taken orally once a day starting cycle 1 day 1 for up to 24 months during the single agent asciminib phase.
Patients will receive asciminib orally 80mg orally once a day starting cycle 1 day 1 for up to 24 months during the single agent asciminib phase. Receives: Drug: Single Agent Asciminib |
| Adding Low TKI | Experimental | TKI should begin within 28-days of obtaining central eligibility confirmation. This phase II trial will use single agent asciminib 80 mg PO daily during the single agent asciminib phase. All eligible subjects will begin asciminib on cycle 1 day 1 of this trial.
Low dose tyrosine kinase inhibitor (lowTKI) (dasatinib 50 mg daily or imatinib 300 mg daily or nilotinib 300 mg daily) at investigators discretion, may be added to asciminib in the following situations:
* Patients who have treatment failure at any time based on ELN criteria (Appendix 7)
* Patients who have a warning response after 12 months of single agent asciminib based on ELN criteria (Appendix 7)
* Patients who have not achieved MR4.5 after 24 months, but no later than 36 months, of single agent asciminib. Receives: Combination Product: Low TKI |
| Elective treatment free remission arm: | Experimental | .Elective treatment free remission arm: Once central eligibility has been obtained the patient should discontinue asciminib and if applicable lowTKI within 14 days. Receives: Other: Elective Free Treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Outcome Measure 1: Deep Molecular Response
The proportion of patients achieving BCR::ABL1 <0.01% IS (MR4.0) within 12 months of treatment with single agent asciminib
Measured: 24 months
Digital Droplet PCR
Digital droplet PCR (ddPCR). Our Consortium work in the LAST study showed that ddPCR is more sensitive than conventional PCR and is very important in predicting TFR in detecting BCR::ABL1 in cases without detectable disease by conventional PCR29. Thus, samples taken for BCR::ABL1 monitoring in this trial will also be tested by ddPCR using the Bio-Rad platform. This will allow for a head to head comparison of these two methods.
Measured: 2 years
Secondary outcomes (9)
The rates of BCR::ABL1 IS ≤10%, ≤1% (MR2), ≤0.1% (MMR), ≤0.01% (MR4), and ≤0.0032% MR4.5
The rates of BCR::ABL1 IS ≤10%, ≤1% (MR2), ≤0.1% (MMR), ≤0.01% (MR4), and ≤0.0032% MR4.5 by 3, 6, 12, 18, and 24 months of therapy
Measured: 2 years
Time to complete hematological response.
Time to complete hematological response (CHR), MR1, MR2, MMR, MR4.0, MR4.5
Measured: 2 years
Duration of MR1, MR2, MMR, MR4.0, MR4.5, and undetectable BCR::ABL1.
Duration of MR1, MR2, MMR, MR4.0, MR4.5, and undetectable BCR::ABL1.
Measured: 2 years
FFS, TFS, EFS, and OS
- Failure-free (FFS), transformation-free (TFS), event-free (EFS), and overall (OS) survival.
Measured: 2 years
Adverse Events
The rates of adverse events related to asciminib
Measured: 2 years
ABL1 sequencing at the time of primary resistance
ABL1 sequencing at the time of primary resistance and test the diagnostic sample for the specific ABL mutation found at resistance (if any).
Measured: 2 years
Clonal evolution and Sokal risk score
Evaluate response and clinical outcomes by presence of clonal evolution, or Sokal risk score at diagnosis.
Measured: 2 years
Patient Reported Outcomes
Patient-reported outcomes at various timepoints per schedule of events.
Measured: 2 years
Rate of MMR loss
Evaluate the rate of MMR loss after asciminib discontinuation for an attempt at treatment free remission at 6 and 24 months from stopping therapy.
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 posted3 Dec 2021
- Study start22 Apr 2022
- Primary completionFeb 2032 (estimated)
- Study completionFeb 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Myeloid Leukemia, Chronic PhaseStudies of the same condition, open and under way.
- More trials run by University of Alabama at BirminghamThe 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- Single group frontline asciminib
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Alabama at Birmingham
- Sponsor type
- University, hospital or other
- Collaborators
- H. Jean Khoury Cure CML Consortium
- Responsible party
- Principal investigator, Omer Jamy, University of Alabama at Birmingham
- Investigators
- Jorge S Cortes, MD, Principal investigator, University of Alabama at Birmingham
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05143840
- Sponsor's protocol ID
- IRB-300016026 (HJKC3-0004)
- Other IDs
- CABL001AUS06T
- Acronym
- ALERTCML
- Status last verified
- Jul 2026
- First submitted
- 16 Nov 2021
- Last updated
- 22 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Asciminib as Initial Therapy With Addition of Lower Dose Tyrosine Kinase Inhibitors for Patients With Chronic Myeloid Leukemia Who do Not Achieve Optimal Response or a Deep Molecular Remission (ALERT CML)
- ClinicalTrials.gov ID
- NCT05143840
- Acronym / sponsor ID
- ALERTCML / IRB-300016026 (HJKC3-0004)
- Conditions
- Chronic Myeloid Leukemia, Chronic Phase, Adult CML, Leukemia, Myeloid, Leukemia,Myeloid, Chronic
- Keywords
- Chronic Myeloid Leukemia, Adult CML, tyrosine kinase inhibitors, H. Jean Khoury Cure CML Consortium, HJKC3-0004
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- University of Alabama at BirminghamUniversity, hospital or other
- Collaborators
- H. Jean Khoury Cure CML Consortium
- Study start
- 22 Apr 2022 (actual)
- Primary completion
- Feb 2032 (estimated)
- Study completion
- Feb 2032 (estimated)
- First posted
- 3 Dec 2021 (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 is a multicenter Phase 2, non-randomized, open-label single-group frontline study administering asciminib in patients with newly diagnosed Chronic Myeloid Leukemia-Chronic Phase (CML-CP). The aim of this study is to evaluate the efficacy and safety of asciminib in newly diagnosed CML-CP. Patients will receive asciminib 80 mg orally once daily during the single asciminib phase. Response is determined by PCR (polymerase chain reaction) blood test during the study. Patients who have not achieved a response after 24 months (but no later than 36 months) of single agent asciminib will be offered the addition of a low dose tyrosine kinase inhibitor (low-TKI) namely dasatinib, imatinib, or nilotinib at the investigator's discretion. The following doses of the TKIs will be used:
- Dasatinib 50 mg daily
- Imatinib 300 mg daily
- Nilotinib 300 mg daily
Patients will discontinue study treatment if they experience disease progression, or unacceptable toxicity.
Detailed description
Asciminib is a potent allosteric inhibitor of BCR-ABL1 oncogene that confers resistance to tyrosine kinase inhibitors (TKIs). Asciminib has potential to combine with TKIs to prevent the emergence of BCR-ABL1 mutations, increasing the depth of molecular response in CML-CP patients. Anticipated enrollment is 50 subjects across sites.
Primary Objective:
To estimate the proportion of patients with previously untreated CML-CP who attain BCR::ABL1 <0.01% (MR4.0) IS by RQ-PCR with single agent asciminib therapy.
Secondary Objectives:
- To estimate the proportion of patients achieving molecular response at specific time points
- To estimate the time to molecular response
- To evaluate the duration of hematologic and molecular response to asciminib
- To define the time to progression and overall survival for patients with CML in early CP treated with asciminib
- To evaluate the safety profile of asciminib in patients with CML-CP
- To evaluate the development of ABL mutations for patients with CML in early CP treated with asciminib
- To analyze differences in response rates and in prognosis within different risk groups and patient characteristics
- To evaluate patient-reported outcomes in patients with CML receiving asciminib
- To investigate treatment-free remission after at least 2 years of sustained deep molecular remission for patients receiving single agent asciminib or combination (asciminib + low TKI)
Exploratory objectives:
- To evaluate the safety and efficacy of concomitant use of low TKI with asciminib in patients who have not achieved MR4.5.
- To evaluate the rate of successful treatment discontinuation for patients using the combination of asciminib and low TKI
- To evaluate the safety and efficacy of concomitant use of lowTKI with asciminib in patients who experience treatment failure at any time with single agent asciminib
- To evaluate the safety and efficacy of concomitant use of lowTKI with asciminib in patients who have not achieved an optimal response after 12 months of single agent asciminib
- Evaluate the role of Digital droplet PCR (ddPCR) in predicting TFR
- Evaluating the correlation between the gene expression signature of patients and the chances of achieving MMR and DMR
- Evaluate whether B, NK and T cells DNA mutation and RNA expression are relevant and whether they can predict response in patients with CML using single cell analysis.
Subjects must meet all inclusion criteria and none of the exclusion criteria of the study. No enrollment waivers will be granted. After successful screening, subjects will be enrolled and treatment will start within 7 days of enrollment. Eligible subjects will begin asciminib on cycle 1 day 1 of the trial. After 2 years (but no later than 3 years), subjects will be offered the addition of taking nilotinib, dasatinib, or imatinib with asciminib if a molecular response is not met (PCR blood test).
Duration of each participant is expected to take approximately 5 years on treatment and up to a total of 8 years if attempting treatment free remission.
Regimen Description
Asciminib 80 mg Oral Once a day 4 weeks (28 days) Nilotinib 300 mg* Oral Once a day 4 weeks (28 days) Dasatinib 50 mg* Oral Once a day 4 weeks (28 days) Imatinib 300 mg* Oral Once a day 4 weeks (28 days)
*Nilotinib, dasatinib, or imatinib will be taken if indicated.
Dose levels and dose modifications of the study drugs will be made per protocol.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Single group frontline asciminib
| Arm | Type | Receives |
|---|---|---|
| Single Agent Asciminib Arm | Experimental | Drug: Single Agent AsciminibAsciminib 80mg Asciminib will be taken orally once a day starting cycle 1 day 1 for up to 24 months during the single agent asciminib phase. Patients will receive asciminib orally 80mg orally once a day starting cycle 1 day 1 for up to 24 months during the single agent asciminib phase. |
| Adding Low TKI | Experimental | Combination Product: Low TKITKI should begin within 28-days of obtaining central eligibility confirmation. This phase II trial will use single agent asciminib 80 mg PO daily during the single agent asciminib phase. All eligible subjects will begin asciminib on cycle 1 day 1 of this trial. Low dose tyrosine kinase inhibitor (lowTKI) (dasatinib 50 mg daily or imatinib 300 mg daily or nilotinib 300 mg daily) at investigators discretion, may be added to asciminib in the following situations: * Patients who have treatment failure at any time based on ELN criteria (Appendix 7) * Patients who have a warning response after 12 months of single agent asciminib based on ELN criteria (Appendix 7) * Patients who have not achieved MR4.5 after 24 months, but no later than 36 months, of single agent asciminib. |
| Elective treatment free remission arm: | Experimental | Other: Elective Free Treatment.Elective treatment free remission arm: Once central eligibility has been obtained the patient should discontinue asciminib and if applicable lowTKI within 14 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (22 listed)
- Age ≥18 years old
- Willing and able to give informed consent
- Newly diagnosed with CML in chronic phase within 6 months from confirmed diagnosis via bone marrow biopsy/aspirate and have either the b3a2 (e14a2) or b2a2 (e13a2) variants that give rise to the p210 BCR::ABL1 protein. Subtype classification whether b3a2 (e14a2) or b2a2 (e13a2) is not required for study eligibility.
- Minimal prior CML therapy with a TKI for less than or equal to 30 days. Treatment with hydroxyurea, busulfan, anagrelide or other non-specific chemotherapy agents is allowed with no time restrictions within the eligible time from diagnosis.
- ECOG performance status 0-2 (appendix 1)
Key exclusion criteria (19 listed)
- Patients with accelerated or blast phase CML (refer to appendix 4)
- Active second malignancy requiring active treatment
- History of recent (within 12 months) acute pancreatitis or chronic pancreatitis
- Subjects who have previously received treatment with asciminib.
- Subjects with PLT count < 50,000 mm3 or ANC of < 500 mm3 or Hemoglobin < 8 g/dL
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 |
|---|---|---|
| Single Agent AsciminibAlso: ABL001taken orally once a day starting cycle 1 day 1 for up to 24 months during the single agent asciminib phase. Asciminib is a potent allosteric inhibitor of BCR::ABL1. | Drug | Single Agent Asciminib Arm |
| Low TKILow dose tyrosine kinase inhibitor (lowTKI) (dasatinib 50 mg daily or imatinib 300 mg daily or nilotinib 300 mg daily) at investigators discretion, may be added to asciminib in the following situations: * Patients who have treatment failure at any time based on ELN criteria (Appendix 7) * Patients who have a warning response after 12 months of single agent asciminib based on ELN criteria (Appendix 7) * Patients who have not achieved MR4.5 after 24 months, but no later than 36 months, of single agent asciminib. | Combination product | Adding Low TKI |
| Elective Free TreatmentOnce central eligibility has been obtained the patient should discontinue asciminib and if applicable lowTKI within 14 days. | Other | Elective treatment free remission arm: |
Primary outcome
Primary Outcome Measure 1: Deep Molecular Response
Time frame: 24 months
The proportion of patients achieving BCR::ABL1 <0.01% IS (MR4.0) within 12 months of treatment with single agent asciminib
Digital Droplet PCR
Time frame: 2 years
Digital droplet PCR (ddPCR). Our Consortium work in the LAST study showed that ddPCR is more sensitive than conventional PCR and is very important in predicting TFR in detecting BCR::ABL1 in cases without detectable disease by conventional PCR29. Thus, samples taken for BCR::ABL1 monitoring in this trial will also be tested by ddPCR using the Bio-Rad platform. This will allow for a head to head comparison of these two methods.
9 secondary outcomes
The rates of BCR::ABL1 IS ≤10%, ≤1% (MR2), ≤0.1% (MMR), ≤0.01% (MR4), and ≤0.0032% MR4.5
Time frame: 2 years
Time to complete hematological response.
Time frame: 2 years
Duration of MR1, MR2, MMR, MR4.0, MR4.5, and undetectable BCR::ABL1.
Time frame: 2 years
FFS, TFS, EFS, and OS
Time frame: 2 years
Adverse Events
Time frame: 2 years
ABL1 sequencing at the time of primary resistance
Time frame: 2 years
Clonal evolution and Sokal risk score
Time frame: 2 years
Patient Reported Outcomes
Time frame: 2 years
Rate of MMR loss
Time frame: 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2026.
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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.
Jorge S Cortes, MD principal investigator
University of Alabama at Birmingham
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Single Agent Asciminib, Low TKI and Elective Free Treatment in people with Chronic Myeloid Leukemia, Chronic Phase and Adult CML, run by University of Alabama at Birmingham at 8 sites 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 22 inclusion and 19 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: under way, not looking for participants, as of the sponsor's last update on 22 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Primary Outcome Measure 1: Deep Molecular Response (time frame: 24 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 22 Jul 2026.