Chronic Myeloid Leukemia (CML)Phase 2ASC4TARGETNCT06514534
Open-label Study of Asciminib for CML-CP or CML-AP Patients With T315I Mutation Who Are Resistant, Intolerant or Ineligible to Ponatinib.
A Phase 2 study testing ABL001/Asciminib in people with Chronic Myeloid Leukemia (CML), run by Novartis Pharmaceuticals at 6 sites in France.
Trial summary
The objective of this Phase II study is to assess the potential of asciminib in managing CML-CP or CML-AP in patient carrying the T315I mutation. The presence of this mutation introduces treatment difficulties due to the limited available options. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Chronic Myeloid Leukemia (CML)
- What is being tested?
- ABL001/Asciminib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 6 sitesFrance
- Study dates
- 18 Feb 2025to 18 Feb 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 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Signed informed consent must be obtained prior to participation in the study.
- Male or female participants with a diagnosis of CML-CP or CML-AP ≥ 18 years of age.
- Patients with CML-CP or CML-AP with history of documented T315I mutation after at least one TKI and are resistant, intolerant, or ineligible to ponatinib (according to Investigator judgment)
- Not already treated with asciminib or another any allosteric TKI
- Failure (adapted from the 2020 \& 2013 ELN Guidelines) or intolerance to Ponatinib at the time of Screening.
- Ineligible to ponatinib according to Investigator (based on EU ponatinib SmPC)
- Evidence of typical BCR::ABL1 transcript or atypical transcripts at the time of Screening which are amenable to standardized or non-standardized RQ-PCR quantification.
Who cannot join
- Previous hematopoietic allogeneic stem-cell transplantation
- Cardiac or cardiac repolarization abnormality
- Severe and/or uncontrolled concurrent medical disease that in the opinion of the Investigator could cause unacceptable safety risks or compromise compliance with the protocol (e.g. uncontrolled diabetes, active or uncontrolled infection, pulmonary hypertension)
- History of clinical acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis (except if ponatinib-induced and completely resolved at time of Screening)
- History of acute or chronic liver disease (i.e., cirrhosis; liver impairment)
- Known presence of significant congenital or acquired bleeding disorder unrelated to cancer
Show all 15Show fewer
- History of other active malignancy within 3 years prior to study entry with the exception of previous or concomitant basal cell skin cancer and previous carcinoma in situ treated curatively
- Known history of Human Immunodeficiency Virus (HIV), chronic Hepatitis B Virus (HBV), or chronic Hepatitis C Virus (HCV) infection. Testing for Hepatitis B surface antigen (HBs Ag) and Hepatitis B core antibody (HBcAb / anti HBc) will be performed at Screening
- Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of study drug (e.g. ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, small bowel resection, or gastric bypass surgery)
- Treatment with medications that meet one of the following criteria and that cannot be discontinued at least one week prior to the start of treatment with study treatment:
- Moderate or strong inducers of CYP3A
- Moderate or strong inhibitors of CYP3A
- Pregnant or nursing (lactating) women
- Women of child-bearing potential
- Compound mutant T315I resistant to asciminib monotherapy (polyclonal ABL1 mutations including T315I can be enrolled) Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
6 sites in 1 country
France6 sites, 6 recruiting
- Recruiting
Novartis Investigative Site
Bordeaux, 33076
- Recruiting
Novartis Investigative Site
Lille, 59037
- Recruiting
Novartis Investigative Site
Lyon, 69373
- Recruiting
Novartis Investigative Site
Nantes, 44093
- Recruiting
Novartis Investigative Site
Paris, 75475
- Recruiting
Novartis Investigative Site
Vandœuvre-lès-Nancy, 54511
About this study
Official title: A Phase II, Multi-center, Prospective, Open-label Study of Asciminib in Patients With Chronic Myeloid Leukemia in Chronic Phase (CML-CP) or Accelerated Phase (CML-AP) With T315I Mutation Who Are Resistant, Intolerant or Ineligible to Ponatinib.
The objective of this Phase II study is to assess the potential of asciminib in managing CML-CP or CML-AP in patient carrying the T315I mutation. The presence of this mutation introduces treatment difficulties due to the limited available options. The study seeks to collect additional data on the effectiveness and safety of asciminib for these patients. By determining the drug's capacity to manage the disease and enhance patients outcomes, the study is designed to fill the unmet medical need and potentially offer a new therapeutic path for patients at a treatment deadlock.
Read the detailed description
This study is a Phase II, multi-center, single-arm prospective, open-label study that aims to evaluate the efficacy and safety of oral asciminib in patients with CML-CP or CML-AP with T315I mutation and after at least one tyrosine kinase inhibitors (TKI) and are resistant, intolerant, or ineligible for treatment with ponatinib.
Patients who have not been previously treated with asciminib would be enrolled in this study. The researchers will assess the effectiveness of asciminib in these participants, as well as evaluate its safety profile. The study will consist of two phases:
- The "core phase" which aims to answer the scientific and medical objectives.
- An "extension phase" intended to provide opportunity to the participants to continue their ongoing treatment (asciminib) up to commercialization in France or decision to not commercialize asciminib for the study population (stopping development, refusal to extend marketing authorization, refusal of reimbursement).
- Conditions
- Chronic Myeloid Leukemia (CML)
- Being tested
- ABL001/Asciminib
- Keywords
- Asciminib, ABL001, Abelson proto-oncogene (ABL1), Chronic Myelogenous Leukemia (CML), BCR::ABL1, Phase II, Tyrosine kinase inhibitor (TKI)
- 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.
ABL001/AsciminibDrug
The study treatment for this clinical trial is an investigational drug called asciminib, which is marketed under the brand name Scemblix®. Asciminib is a compound that is being evaluated for its efficacy and safety in the treatment of the target condition.
The minimum dose of asciminib to be administered in this study is 200 mg, while the maximum dose is 400 mg. The dose is planned as 200 mg twice a day (BID).
The drug will be administered orally, allowing for convenient and non-invasive administration.
| Study group | Role | What they receive |
|---|---|---|
| Asciminib (Scemblix®) | Experimental | Asciminib will be administered 200 mg twice a day orally. The minimum dose is 200 mg, and maximum dose is 400 mg. Receives: Drug: ABL001/Asciminib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of BCR::ABL1 (Breakpoint Cluster Region Gene::Abelson proto-oncogene) IS (International Scale) ≤ 1% [MR2 (Molecular Response 2)]
Evaluation of asciminib efficacy : proportion of patients with MR2 (BCR::ABL1 IS ≤1%) level of response at 12 months.
Measured: Month 12
Secondary outcomes (12)
Kinetics of response: BCR::ABL1 IS (MR2, MMR, MR4.0, MR4.5, undetectable MR4.5)
Proportion of patients achieving the response levels (MR2, MMR, MR4.0, MR4.5, undetectable MR4.5)
Measured: At 3, 6, 9, 12, 18 and 24 months
Estimate response to treatment MR2 at 12 months in participants with BCR::ABL1 IS > 1% at treatment initiation or maintenance of MR2 at 12 months in participants with MR2 at treatment initiation
Proportion of patients with MR2 level of response (BCR::ABL1 IS ≤ 1%) in participants without MR2 at treatment initiation or maintenance of MR2 at 12 months in participants with MR2 at treatment initiation.
Measured: At 12 months
Time to Major Molecular Response (MMR) (for participants not in MMR at treatment initiation)
Major Molecular Response (MMR) criteria is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized Baseline equivalent to ≤ 0.1 % BCR::ABL1 % by international scale as measured by RQ-PCR, confirmed by duplicate analysis of the same sample.
Measured: up to 24 months
Duration of MMR
MMR criteria is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized Baseline equivalent to ≤ 0.1 % BCR::ABL1 % by international scale as measured by RQ-PCR, confirmed by duplicate analysis of the same sample.
Measured: up to 24 months
Time to Molecular Response 2 (MR2) (for participants not in MR2 at treatment initiation)
MR2 criteria is defined as a ≥ 2.0 log reduction in BCR::ABL1 transcripts compared to the standardized Baseline equivalent to ≤ 1 % BCR::ABL1 % by international scale as measured by RQ-PCR, confirmed by duplicate analysis of the same sample.
Measured: up to 24 months
Duration of MR2
MR2 criteria is defined as a ≥ 2.0 log reduction in BCR::ABL1 transcripts compared to the standardized Baseline equivalent to ≤ 1 % BCR::ABL1 % by international scale as measured by RQ-PCR, confirmed by duplicate analysis of the same sample
Measured: up to 24 months
Overall survival (OS)
Overall Survival (OS) is defined as the time from the first dose of study medication to death due to any cause. If a patient is not known to have died, then OS will be censored at the latest date the patient was known to be alive (on or before the cut-off date).
Measured: up to 24 months
Progression Free Survival (PFS)
Progression Free Survival (PFS) is defined as time from the first dose of study medication to earliest occurrence of documented disease progression to AP/BC (depending on CML phase at asciminib initiation: CP or AP) or death due to any cause, whichever occurs first.
Measured: up to 24 months
Event Free Survival (EFS)
Event Free Survival is defined as time from the first dose of study medication to an event which may include:
- Treatment failure
- Confirmed loss of MR2 at any time while on study treatment,
- Discontinuation of study treatment due to AE,
- Progression to AP/BC (including progressions observed during the survival follow up period),
- Death from any cause (including deaths observed during the survival follow-up period).
Measured: up to 24 months
Failure Free Survival (FFS)
Failure Free Survival (FFS) is defined as time from the first dose of study medication to a failure event which may include:
- Treatment failure
- Confirmed loss of MR2 at any time while on study treatment,
- Discontinuation of study treatment due to AE.
Measured: up to 24 months
Adverse events (AEs) and Serious Adverse events (SAEs)
Description/severity (grade of severity) of AE (all AE, serious or not serious AE, related and not related AE), and number of patients who discontinued the treatment due to AE.
Measured: up to 24 months
Deaths and reasons for death
To evaluate the safety and tolerability profile of asciminib.
Measured: up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2024
- Study start18 Feb 2025
- Primary completion17 Feb 2029 (estimated)
- Study completion18 Feb 2029 (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 (CML)Studies 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single-arm, open-label study
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
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
- NCT06514534
- Sponsor's protocol ID
- CABL001AFR05
- Other IDs
- 2024-516049-38-00
- Acronym
- ASC4TARGET
- Status last verified
- Apr 2026
- First submitted
- 17 Jul 2024
- Last updated
- 21 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Multi-center, Prospective, Open-label Study of Asciminib in Patients With Chronic Myeloid Leukemia in Chronic Phase (CML-CP) or Accelerated Phase (CML-AP) With T315I Mutation Who Are Resistant, Intolerant or Ineligible to Ponatinib.
- ClinicalTrials.gov ID
- NCT06514534
- Acronym / sponsor ID
- ASC4TARGET / CABL001AFR05
- Conditions
- Chronic Myeloid Leukemia (CML)
- Keywords
- Asciminib, ABL001, Abelson proto-oncogene (ABL1), Chronic Myelogenous Leukemia (CML), BCR::ABL1, Phase II, Tyrosine kinase inhibitor (TKI)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 18 Feb 2025 (actual)
- Primary completion
- 17 Feb 2029 (estimated)
- Study completion
- 18 Feb 2029 (estimated)
- First posted
- 23 Jul 2024 (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.
The objective of this Phase II study is to assess the potential of asciminib in managing CML-CP or CML-AP in patient carrying the T315I mutation. The presence of this mutation introduces treatment difficulties due to the limited available options. The study seeks to collect additional data on the effectiveness and safety of asciminib for these patients. By determining the drug's capacity to manage the disease and enhance patients outcomes, the study is designed to fill the unmet medical need and potentially offer a new therapeutic path for patients at a treatment deadlock.
Detailed description
This study is a Phase II, multi-center, single-arm prospective, open-label study that aims to evaluate the efficacy and safety of oral asciminib in patients with CML-CP or CML-AP with T315I mutation and after at least one tyrosine kinase inhibitors (TKI) and are resistant, intolerant, or ineligible for treatment with ponatinib.
Patients who have not been previously treated with asciminib would be enrolled in this study. The researchers will assess the effectiveness of asciminib in these participants, as well as evaluate its safety profile. The study will consist of two phases:
- The "core phase" which aims to answer the scientific and medical objectives.
- An "extension phase" intended to provide opportunity to the participants to continue their ongoing treatment (asciminib) up to commercialization in France or decision to not commercialize asciminib for the study population (stopping development, refusal to extend marketing authorization, refusal of reimbursement).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single-arm, open-label study
| Arm | Type | Receives |
|---|---|---|
| Asciminib (Scemblix®) | Experimental | Drug: ABL001/AsciminibAsciminib will be administered 200 mg twice a day orally. The minimum dose is 200 mg, and maximum dose is 400 mg. |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Signed informed consent must be obtained prior to participation in the study.
- Male or female participants with a diagnosis of CML-CP or CML-AP ≥ 18 years of age.
- Patients with CML-CP or CML-AP with history of documented T315I mutation after at least one TKI and are resistant, intolerant, or ineligible to ponatinib (according to Investigator judgment)
- Not already treated with asciminib or another any allosteric TKI
- Failure (adapted from the 2020 \& 2013 ELN Guidelines) or intolerance to Ponatinib at the time of Screening.
Key exclusion criteria (15 listed)
- Previous hematopoietic allogeneic stem-cell transplantation
- Cardiac or cardiac repolarization abnormality
- Severe and/or uncontrolled concurrent medical disease that in the opinion of the Investigator could cause unacceptable safety risks or compromise compliance with the protocol (e.g. uncontrolled diabetes, active or uncontrolled infection, pulmonary hypertension)
- History of clinical acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis (except if ponatinib-induced and completely resolved at time of Screening)
- History of acute or chronic liver disease (i.e., cirrhosis; liver impairment)
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 |
|---|---|---|
| ABL001/AsciminibThe study treatment for this clinical trial is an investigational drug called asciminib, which is marketed under the brand name Scemblix®. Asciminib is a compound that is being evaluated for its efficacy and safety in the treatment of the target condition. The minimum dose of asciminib to be administered in this study is 200 mg, while the maximum dose is 400 mg. The dose is planned as 200 mg twice a day (BID). The drug will be administered orally, allowing for convenient and non-invasive administration. | Drug | Asciminib (Scemblix®) |
Primary outcome
Rate of BCR::ABL1 (Breakpoint Cluster Region Gene::Abelson proto-oncogene) IS (International Scale) ≤ 1% [MR2 (Molecular Response 2)]
Time frame: Month 12
Evaluation of asciminib efficacy : proportion of patients with MR2 (BCR::ABL1 IS ≤1%) level of response at 12 months.
12 secondary outcomes
Kinetics of response: BCR::ABL1 IS (MR2, MMR, MR4.0, MR4.5, undetectable MR4.5)
Time frame: At 3, 6, 9, 12, 18 and 24 months
Estimate response to treatment MR2 at 12 months in participants with BCR::ABL1 IS > 1% at treatment initiation or maintenance of MR2 at 12 months in participants with MR2 at treatment initiation
Time frame: At 12 months
Time to Major Molecular Response (MMR) (for participants not in MMR at treatment initiation)
Time frame: up to 24 months
Duration of MMR
Time frame: up to 24 months
Time to Molecular Response 2 (MR2) (for participants not in MR2 at treatment initiation)
Time frame: up to 24 months
Duration of MR2
Time frame: up to 24 months
Overall survival (OS)
Time frame: up to 24 months
Progression Free Survival (PFS)
Time frame: up to 24 months
Event Free Survival (EFS)
Time frame: up to 24 months
Failure Free Survival (FFS)
Time frame: up to 24 months
Adverse events (AEs) and Serious Adverse events (SAEs)
Time frame: up to 24 months
Deaths and reasons for death
Time frame: up to 24 months
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 21 Apr 2026.
- Individual participant dataSharing planned: Yes. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
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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.
Novartis Pharmaceuticals contact
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
6 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 ABL001/Asciminib in people with Chronic Myeloid Leukemia (CML), run by Novartis Pharmaceuticals at 6 sites in France.
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 15 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 21 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in France. 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 Novartis Pharmaceuticals (+41613241111, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of BCR::ABL1 (Breakpoint Cluster Region Gene::Abelson proto-oncogene) IS (International Scale) ≤ 1% [MR2 (Molecular Response 2)] (time frame: Month 12). 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 21 Apr 2026.