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Chronic Phase Chronic Myelogenous LeukemiaPhase 2NCT04838041

Protocol Number: HJKC3-0003. Treatment Free Remission After Asciminib Based Therapy in Chronic Phase Chronic Myeloid Leukemia (CP-CML) Patients Who Relapsed After a Prior Attempt at TKI Discontinuation

A Phase 2 study testing Asciminib 40 MG, Asciminib 40 MG Twice Daily and Asciminib 80 MG daily in people with Chronic Phase Chronic Myelogenous Leukemia, run by Medical College of Wisconsin at 5 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

This is a single arm phase II study that will enroll a minimum of 47 subjects with a maximum of 51. All patients will have a confirmed diagnosis of chronic phase chronic myeloid Leukemia and must have previously attempted to discontinue Tyrosine Kinase inhibitors (TKI). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Asciminib 40 MG, Asciminib 40 MG Twice Daily and Asciminib 80 MG daily
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
51 participants
Where
5 sitesUnited States
Study dates
11 Nov 2021to Jul 2029
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.
  • Diagnosed with chronic myelogenous leukemia (CML) in chronic phase without BCR::ABL1 ^T315I 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.
  • Must have a documented history of attempting only one prior TKI discontinuation under the guidance of a treating physician. TKI includes dasatinib, imatinib or nilotinib.
  • Must have met all the following criteria prior to first attempt to discontinue their TKI:
  • Stable molecular response (MR4; < 0.01% IS) for > 2 years (with allowance for a two-week variance), as documented on at least four tests, performed at least three months apart (e.g., If a patient has had >4 PCR tests performed during the two years leading up to their initial TKI discontinuation, any value between 0.01 and 0.05% IS is considered a stable result, however, at least four tests must be < 0.01% IS. If any results are >0.05% IS, tests must have been repeated within one month and be less than 0.01% IS and stable.
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  • Treatment with one of the following FDA approved TKIs; imatinib, dasatinib, nilotinib at any dose for a minimum of approximately three years (allowance of a four-week variance) prior to discontinuing TKIs.
  • Has been on any number of TKIs, but has not been resistant to any TKI (changes made for intolerance are allowed).
  • Must have relapsed (defined as loss of major molecular response (MMR), RQ-PCR for BCR::ABL1 >0.1% IS after first attempted TKI discontinuation.
  • After first failed TFR attempt, must have a minimum duration of one year of retreatment with TKI, and must plan to remain on that TKI or switch to asciminib for a minimum of 12 months during the consolidation treatment phase.
  • Current TKI must be the same as the TKI being taken prior to the initial TFR attempt (e.g., if patient is on imatinib prior to first TFR attempt, they should be on imatinib at time of enrollment on this study).
  • Eastern Cooperative Oncology Group (ECOG) performance status 0-3.
  • Must have a RQ-PCR for BCR::ABL1 < 0.0032% IS (MR4.5) reported by the trial designated central lab at the time of study enrollment.
  • Lipase ≤ 1.5 x upper limit of normal (ULN). For lipase > ULN - ≤ 1.5 x ULN, value should be considered not clinically significant and not associated with risk factors for acute pancreatitis.
  • 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)
  • 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

  • History of accelerated or blast phase CML.
  • A 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 platelet (PLT) count < 100 × 109/L or an absolute neutrophil count (ANC) of < 1 × 109/L or hemoglobin < 8 g/dL.
  • Aspartate aminotransferase (AST) and alanine transaminase (ALT) ≥3 times the institutional upper limit of normal.
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  • Total bilirubin ≥ 1.5 times the institutional upper limit of normal (unless direct bilirubin is within normal limits).
  • Pregnant or lactating.
  • Unable to comply with lab appointment schedule and patient-reported outcome (PRO) assessments.
  • Another investigational drug within four 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.
  • Screening 12-lead electrocardiogram (ECG) showing a baseline corrected QT interval >480msec (patients with a pacemaker will still be eligible with QTc>500msec).
  • Known active hepatitis B infection.
  • Eligibility for TFR Phase:
  • Stable molecular response (MR4.5; < 0.0032% IS) documented on at least three tests (may include TFR phase screening PCR) by the trial designated lab, performed approximately three months apart while on consolidation phase.
  • TFR phase screening PCR RQ-PCR for BCR::ABL1 < 0.0032% IS (MR4.5) by the trial designated lab.
  • ECOG 0-3.
  • Completion of 12 cycles on the consolidation therapy phase.

Trial locations and contacts

5 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Medical College of Wisconsin Cancer Center Clinical Trials Office contact

414-805-8900

cccto@mcw.edu

Ehab Atallah, MD contact

414-805-4600

eatallah@mcw.edu

5 sites in 1 country

United States5 sites, 5 recruiting

  • Roswell Park Comprehensive Cancer Center

    Buffalo, New York, 14263

    James Thompson, MD, MS, 716-845-7110, james.thompson@roswellpark.org

    Recruiting

  • The Barbara Ann Karmanos Cancer Institute

    Detroit, Michigan, 48201

    Sharon Prokop, 313-576-9363, prokops@karmanos.org

    Jay Yang, MD

    Recruiting

  • Froedtert Hospital & the Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

    Ehab Atallah, MD, 414-805-4600, eatallah@mcw.edu

    Ehab Atallah, MD

    Recruiting

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

    Michael Mauro, MD, maurom@mskcc.org

    Recruiting

  • Huntsman Cancer Institute

    Salt Lake City, Utah, 84112

    Srinivas Tantravahi, MBBS, Srinivas.Tantravahi@hci.utah.edu

    Recruiting

Other trials in United States

About this study

This is a single arm phase II study that will enroll a minimum of 47 subjects with a maximum of 51. All patients will have a confirmed diagnosis of chronic phase chronic myeloid Leukemia and must have previously attempted to discontinue Tyrosine Kinase inhibitors (TKI). All patients must have restarted the same TKI they were on prior to discontinuation at the time of relapse in order to be eligible for this trial.

Read the detailed description

All eligible patients will begin asciminib plus/minus TKI on cycle 1 day 1 of the consolidation phase. They will continue therapy for a total of 12 cycles (minimum of 12 months). Each cycle will be ~28 days. At the end of 12 cycles, asciminib plus/minus TKI will be discontinued in patients who continue to satisfy the requirements for TFR attempt. The primary endpoint of this study is the 12-month 'second' TFR rate after completion of 12 cycles of asciminib based therapy. Patients will remain in the TFR phase of the study for up to three years and will have central PCR testing during the first two years. PCR testing will continue locally thereafter. Therefore, the total duration of the subject participation trial will be approximately five years (one year on consolidation phase + three years in the TFR phase + one year of long-term follow-up post TFR or early discontinuation.

Being tested
Asciminib 40 MG, Asciminib 40 MG Twice Daily, Asciminib 80 MG daily, Imatinib, Nilotinib
Keywords
chronic myelogenous leukemia, treatment-free remission, tyrosine kinase inhibitors, H. Jean Khoury Cure CML Consortium, Asciminib
Medical subject headings
Leukemia, Myeloid, Chronic-Phase, 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.

  • Asciminib 40 MGDrug

    Also called ABL001

    40 mg by mouth (PO) when used with imatinib.

  • Asciminib 40 MG Twice DailyDrug

    Also called ABL001

    40 mg twice daily when used with nilotinib.

  • Asciminib 80 MG dailyDrug

    Also called ABL001

    80 mg daily when used with dasatinib or taken alone.

  • ImatinibDrug

    Also called Gleevec

    Maximum dose of 400 mg PO once daily.

  • NilotinibDrug

    Also called TASIGNA

    Maximum dose of 300 mg twice daily.

  • DasatinibDrug

    Also called Sprycel

    Maximum dose of 100 mg PO once daily.

Study groups in NCT04838041
Study groupRoleWhat they receive
Asciminib 40 mg PO daily plus imatinib (maximum dose of 400 mg PO once daily)ExperimentalAll eligible patients will begin asciminib plus/minus TKI on cycle 1 day 1 of the consolidation phase. They will continue therapy for a total of 12 cycles (minimum of 12 months). Each cycle will be ~28 days. At the end of 12 cycles, asciminib plus/minus TKI will be discontinued in patients who continue to satisfy the requirements for TFR attempt.
Receives: Drug: Asciminib 40 MG; Drug: Imatinib
Asciminib 40 mg twice daily plus nilotinib (maximum dose of 300 mg twice daily)ExperimentalAll eligible patients will begin asciminib plus/minus TKI on cycle 1 day 1 of the consolidation phase. They will continue therapy for a total of 12 cycles (minimum of 12 months). Each cycle will be ~28 days. At the end of 12 cycles, asciminib plus/minus TKI will be discontinued in patients who continue to satisfy the requirements for TFR attempt.
Receives: Drug: Asciminib 40 MG Twice Daily; Drug: Nilotinib
Asciminib 80 mg daily plus dasatinib (maximum dose of 100 mg PO once daily)ExperimentalAll eligible patients will begin asciminib plus/minus TKI on cycle 1 day 1 of the consolidation phase. They will continue therapy for a total of 12 cycles (minimum of 12 months). Each cycle will be ~28 days. At the end of 12 cycles, asciminib plus/minus TKI will be discontinued in patients who continue to satisfy the requirements for TFR attempt.
Receives: Drug: Asciminib 80 MG daily; Drug: Dasatinib
Asciminib 80 mg PO daily taken aloneExperimentalAll eligible patients will begin asciminib plus/minus TKI on cycle 1 day 1 of the consolidation phase. They will continue therapy for a total of 12 cycles (minimum of 12 months). Each cycle will be ~28 days. At the end of 12 cycles, asciminib plus/minus TKI will be discontinued in patients who continue to satisfy the requirements for TFR attempt.
Receives: Drug: Asciminib 80 MG daily

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. One-year "second" treatment-free remission.

    This will be measured by the number of subjects who achieve one-year treatment-free remission after 12 months of asciminib based therapy. These subjects have previously failed a first treatment free remission attempt.

    Measured: 1 year after stopping treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Apr 2021
  2. Study start11 Nov 2021
  3. Primary completionJun 2028 (estimated)
  4. Study completionJul 2029 (estimated)

Resources

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
Masking
None (open label)
Enrolment
51 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04838041
Sponsor's protocol ID
PRO00040685
Status last verified
Aug 2026
First submitted
26 Mar 2021
Last updated
25 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Asciminib 40 MG, Asciminib 40 MG Twice Daily and Asciminib 80 MG daily in people with Chronic Phase Chronic Myelogenous Leukemia, run by Medical College of Wisconsin at 5 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 25 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: looking for participants now, as of the sponsor's last update on 25 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 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?

Contact Medical College of Wisconsin Cancer Center Clinical Trials Office (414-805-8900, cccto@mcw.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

One-year "second" treatment-free remission. (time frame: 1 year after stopping treatment). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04838041.

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 Aug 2026; AzTrials last refreshed its copy on 7 Nov 2026.

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