Chronic Myelogenous LeukemiaPhase 2NCT07354074
Study to Determine the Efficacy and Safety of Asciminib in Pediatric Patients With Ph+ CML-CP
A Phase 2 study testing Asciminib single agent in people with Chronic Myelogenous Leukemia and Leukemia, Myelogenous, Chronic, Philadelphia Chromosome Positive, run by Novartis Pharmaceuticals at 34 sites in 15 countries.
Trial summary
The aim of this study is to support development of asciminib in the pediatric population (1 to < 18 years) with Ph+ CML-CP. The study will evaluate the efficacy and safety of asciminib in pediatric formulation (weigh-based dose, fed state) or adult formulation (fasted) in newly diagnosed and resistant or intolerant… Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Chronic Myelogenous Leukemia, Leukemia, Myelogenous, Chronic, Philadelphia Chromosome Positive
- What is being tested?
- Asciminib single agent
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 34 sitesin 15 countries
- Study dates
- 28 Apr 2026to 23 Feb 2033
- 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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participants eligible for inclusion in this study must meet all of the following criteria:
- Signed informed consent must be obtained prior to participation in the study.
- Male or female participants 1 and < 18 years of age at study enrollment
- Diagnosis of CML-CP (Apperley et al 2025) with cytogenetic confirmation of Philadelphia positive (Ph+) chromosome
- For participants with CML-CP newly diagnosed within 3 months of screening OR 5 For participants with CML - CP with high risk of developing resistance or intolerance to previous TKI:
- Unfavourable response to TKI is defined following the Apperley et al 2025 guidelines as:
Show all 16Show fewer
- At three months after the initiation of therapy: BCR::ABL1 ratio > 10% IS (if confirmed within 1-3 months)
- At six months after the initiation of therapy: BCR::ABL1 ratio > 10% IS
- At twelve months after initiation of therapy: BCR::ABL1 ratio > 1% IS
- At any time loss of previous response
- At any time emergent resistant BCR::ABL1 mutations or high-risk ACA from prior TKI treatment as per local test results
- Intolerance to TKI is defined as:
- Non-hematologic intolerance: participants with grade 3 or 4 toxicity while on therapy (in which case the patient is eligible whether or not there was a dose reduction); or with persistent grade 2 toxicity unresponsive to optimal management including dose adjustments (unless dose reduction is not considered in the best interest of the patient if response is already suboptimal)
- Hematologic intolerance: participants with grade 3 or 4 toxicity (absolute neutrophil count [ANC] or platelets) while on therapy that is recurrent after dose reduction to the lowest doses of the TKI
- 6. Evidence of typical BCR::ABL1 transcript [e14a2 and/or e13a2] at the time of screening which are amenable to standardized RQ-PCR quantification.
- 7. Performance status: Karnofsky ≥ 50% for participants ≥ 16 years of age, and Lansky ≥ 50 for participants < 16 years of age at the time of screening.
Who cannot join
- Known second chronic phase (CP) of CML after previous progression to Accelerated Phase (AP)/Blast Phase (BP).
- Previous treatment with a hematopoietic stem-cell transplantation.
- Patient planned to undergo allogeneic hematopoietic stem cell transplantation
- Known presence of a BCR::ABL1 mutation with known resistance to study treatment in accordance with the most recent public version of international CML clinical guidelines (e.g. NCCN CML treatment guidelines v 1.2026 and Apperley et al 2025) any time prior to study entry
- Other inclusion/exclusion criteria may apply.
Trial locations and contacts
34 sites in 15 countries. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
34 sites in 15 countries
United States4 sites, 4 recruiting
- Recruiting
Cinn Children Hosp Medical Center
Cincinnati, Ohio, 45229-3039
Lori Backus, lori.backus@cchmc.org
Benjamin Mizukawa
- Recruiting
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, 08901
Beverly Poscablo, bp646@cinj.rutgers.edu
Richard DRACHTMAN
- Recruiting
Columbia University Medical Center New York Presbyterian
New York, New York, 10032
Karishma Sookraj, 212-305-6953, kss2158@cumc.columbia.edu
Nobuko Hijiya
- Recruiting
Seattle Childrens Hospital
Seattle, Washington, 98105
Angela Bleeker, 206-884-1071, Angela.Bleeker@seattlechildrens.org
Todd Cooper
France4 sites, 4 recruiting
- Recruiting
Novartis Investigative Site
Marseille, 13885
- Recruiting
Novartis Investigative Site
Paris, 75019
- Recruiting
Novartis Investigative Site
Poitiers, 86021
- Recruiting
Novartis Investigative Site
Strasbourg, 67000
Brazil3 sites, 3 recruiting
- Recruiting
Novartis Investigative Site
Barretos, São Paulo, 14784-400
- Recruiting
Novartis Investigative Site
Rio de Janeiro, Rio de Janeiro, 20211-030
- Recruiting
Novartis Investigative Site
São Paulo, São Paulo, 04829-310
Japan3 sites, 3 recruiting
- Recruiting
Novartis Investigative Site
Fukuoka, 8128582
- Recruiting
Novartis Investigative Site
Saitama, 3308777
- Recruiting
Novartis Investigative Site
Yokohama, Kanagawa, 2328555
Spain3 sites, 3 recruiting
- Recruiting
Novartis Investigative Site
Barcelona, 08035
- Recruiting
Novartis Investigative Site
Madrid, 28009
- Recruiting
Novartis Investigative Site
Madrid, 28046
Australia2 sites, 2 recruiting
- Recruiting
Novartis Investigative Site
Brisbane, Queensland, 4101
- Recruiting
Novartis Investigative Site
North Adelaide, South Australia, 5066
Canada2 sites, 2 recruiting
- Recruiting
Novartis Investigative Site
Edmonton, Alberta, T6G 1C9
- Recruiting
Novartis Investigative Site
Montreal, Quebec, H3T 1C5
China2 sites, 2 recruiting
- Recruiting
Novartis Investigative Site
Tianjin, 300020
- Recruiting
Novartis Investigative Site
Zhengzhou, Henan, 450003
Germany2 sites, 2 recruiting
- Recruiting
Novartis Investigative Site
Erlangen, 91054
- Recruiting
Novartis Investigative Site
Frankfurt am Main, Hesse, 60590
Italy2 sites, 2 recruiting
- Recruiting
Novartis Investigative Site
Padova, PD, 35128
- Recruiting
Novartis Investigative Site
Roma, RM, 00165
Poland2 sites, 2 recruiting
- Recruiting
Novartis Investigative Site
Gdansk, 80-214
- Recruiting
Novartis Investigative Site
Wroclaw, 50-556
South Korea2 sites, 2 recruiting
- Recruiting
Novartis Investigative Site
Seoul, 03080
- Recruiting
Novartis Investigative Site
Seoul, 06591
Hungary1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Debrecen, Hajdu Bihar Megye, 4032
Netherlands1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Utrecht, 3584 CS
Thailand1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Bangkok, 10700
About this study
Official title: A Phase II, Multicenter, Open-label, Single Arm Study to Evaluate the Safety and Efficacy of Asciminib in Pediatric Participants Newly Diagnosed or Previously Treated With Philadelphia Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP) With or Without Known T315I Mutation
The aim of this study is to support development of asciminib in the pediatric population (1 to < 18 years) with Ph+ CML-CP. The study will evaluate the efficacy and safety of asciminib in pediatric formulation (weigh-based dose, fed state) or adult formulation (fasted) in newly diagnosed and resistant or intolerant Ph+ CML-CP with or without T315I mutation.
Read the detailed description
This is a multi-center, open-label, single arm study of asciminib in pediatric participants aged
1 to <18 years old with Ph+ CML-CP newly diagnosed and previously treated with TKI treatment, with or without T315I mutation.
The study population will consist of three cohorts of Ph+ CML-CP pediatric participants:
- Newly-diagnosed Ph+ CML-CP participants without known T315I mutation
- Ph+ CML-CP participants resistant or intolerant to previous TKI without known T315I mutation
- Ph+ CML-CP participants with known T315I mutation irrespective of prior TKI treatment
There is no fixed duration of study treatment for the participants. The study will end 5 years (240 weeks) after the last enrolled participants received their first dose of treatment in the study. The objective is to have enough follow up for safety, including growth and development and efficacy.
- Conditions
- Chronic Myelogenous Leukemia, Leukemia, Myelogenous, Chronic, Philadelphia Chromosome Positive
- Being tested
- Asciminib single agent
- Keywords
- Asciminib, ABL001, Pediatric participants, Philadelphia chromosome positive chronic myeloid leukemia in chronic phase, Ph+ CML-CP, tyrosine kinase inhibitor, TKI, Molecular Response, MR, CML, Chronic phase, T3151, Ph+
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Bronchiolitis Obliterans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Asciminib single agentDrug
Also called ABL001
Asciminib (labelled as ABL001) administered as 40 mg tablet (adult formulation) or as 1 mg film-coated granules mini-tablets (pediatric formulation)
| Study group | Role | What they receive |
|---|---|---|
| Single arm study | Experimental | This study will enroll pediatric patients (≥ 1 and < 18 years of age) with newly diagnosed or previously treated with Philadelphia positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP) with or without known T315I mutation Receives: Drug: Asciminib single agent |
What is being measured
The primary outcome is the main question the study is designed to answer.
MMR at Week 48
MMR is defined as BCR::ABL1 IS ≤0.1%. MMR is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1 % BCR::ABL1/ABL % by international scale as measured by RQ-PCR.
Measured: 48 weeks
Secondary outcomes (17)
MMR at Week 96
MMR is defined as BCR::ABL1 IS ≤0.1%. MMR is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1 % BCR::ABL1/ABL % by international scale as measured by RQ-PCR.
Measured: 96 weeks
MMR at and by scheduled timepoints
MMR is defined as BCR::ABL1 IS ≤0.1%. MMR is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1 % BCR::ABL1/ABL % by international scale as measured by RQ-PCR.
Measured: Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96
Hematologic response at and by scheduled timepoints
Complete hematologic response (CHR) is defined as all of the following present for ≥ 4 weeks (which means present at least at 2 visits 4-week apart, with no intermediate visit showing no CHR):
- White blood cell(s) (WBC) count < 10 x 10^9/L
- Platelet count < 450 x 10^9/L
- Basophils < 5%
- No blasts and promyelocytes in peripheral blood
- Myelocytes + metamyelocytes < 5% in peripheral blood
- No evidence of extramedullary disease, including spleen and liver
Measured: Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96
Cytogenetic response at and by scheduled timepoints
Cytogenetic response is defined as follows:
- Complete (CCyR) - 0% Ph+ metaphases
- Partial (PCyR) - >0 to 35% Ph+ metaphases
- Major* (MCyR) - 0 to 35% Ph+ metaphases
- Minor (mCyR) - >35 to 65% Ph+ metaphases
- Minimal - >65 to 95% Ph+ metaphases
- None - >95 to 100% Ph+ metaphases Bone marrow aspirate for cytogenetic analyses will be performed at screening, and upon unfavorable response to treatment at the physician's discretion.
Measured: 5 years
Time to response
Time to the first response is defined as: date of the first documented occurrence of the response or time from first study drug intake to first response - date of the first study treatment intake + 1.
Measured: 240 weeks after the last participant enrolled in the study received the first dose of treatment
Duration of response (limited to the binary response endpoints)
Duration of response is defined as the time between the date of the first documented occurrence of the response and the earliest date of confirmed loss of the response, progression to accelerated phase/blast crisis (AP/BC) or CML-related death for participants in the respective Responder Set.
Measured: 240 weeks after the last participant enrolled in the study received the first dose of treatment
Time to treatment failure (TTF)
TTF is defined as the time from the first study drug intake to the first/earliest documented date of any of the following events: unfavorable response to treatment, confirmed loss of MMR at any time while on study treatment and discontinuation from study treatment due to any reason.
Measured: 240 weeks after the last participant enrolled in the study received the first dose of treatment
Time to disease progression
Measured: 5 years
Event-free survival (EFS)
EFS is defined as the time from the first study drug intake to the earliest occurrence of the following events: unfavorable response to treatment, confirmed loss of MMR at any time while on study treatment, discontinuation from the study treatment due to an adverse event (AE) and death due to any cause.
Measured: 240 weeks after the last participant enrolled in the study received the first dose of treatment
Overall survival (OS)
OS is defined as the time from the first study drug intake to the date of death from any cause during the study (including death observed during the survival follow-up period after discontinuation of study treatment).
Measured: 240 weeks after the last participant enrolled in the study received the first dose of treatment
Growth: Height/length, weight, bone age measured by X-Ray
To assess growth and sexual maturation. An X-ray of the left hand and wrist to assess bone age will be performed at screening and every 48 weeks until end of treatment (EOT). This assessment will no longer be required for participants once bone maturity in post-puberty stage is confirmed by the last X-ray.
Measured: 240 weeks after the last participant enrolled in the study received the first dose of treatment
Sexual maturation: Height/length, weight, bone age measured by Tanner staging
To assess growth and sexual maturation. Tanner staging will be based on external genitalia, breast and pubic hair examination. Tanner staging will be assessed for all study participants at screening. Participants who do not have a baseline Tanner Stage of 5 will continue to be assessed every 24 weeks until achievement of Tanner Stage 5 on both secondary sexual characteristics or EOT, whichever comes first. Once a participant reaches stage 5, examinations do not need to be conducted anymore. For all male participants, Tanner Stage 5 will be achieved once the participant has demonstrated both stage 5 genitalia development and stage 5 pubic hair development. For all female participants, Tanner Stage 5 will be achieved once the participant has demonstrated both stage 5 breast development and stage 5 pubic hair development.
Measured: 240 weeks after the last participant enrolled in the study received the first dose of treatment
PK parameters of asciminib: AUClast
AUClast: The AUC from time zero to the last measurable concentration sampling time.
Measured: Week 2, Day 1 at 1, 3 and 6 hours post dose
PK parameters of asciminib: AUCtau
AUCtau: The AUC calculated to the end of a dosing interval (tau) at steady-state.
Measured: Week 2, Day 1: pre dose (0 hour), Week 2: Day 1 at 1, 2, 3, and 6 hours post-dose
PK parameter of asciminib: Cmax
Cmax is the maximum (peak) observed plasma drug concentration after single dose administration.
Measured: Week 2, Day 1 at 1, 3, and 6 hours post dose
PK parameter of asciminib: Tmax
Tmax is the time to reach maximum (peak) plasma drug concentration (h) after administration.
Measured: Week 2, Day 1 at 1, 3, and 6 hours post dose
PK parameter of asciminib: Ctrough
Ctrough refers to the lowest concentration a drug reaches in the bloodstream immediately before the next dose is administered.
Measured: Week 2, Day 1: pre-dose (0 hr), Week 24: pre-dose (0 hr), Week 48: Pre-dose (0 hr)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jan 2026
- Study start28 Apr 2026
- Primary completion23 Feb 2033 (estimated)
- Study completion23 Feb 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Myelogenous LeukemiaStudies 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
- Three cohorts of Ph+ CML-CP pediatric participants receive asciminib: (1) newly-diagnosed without known T315I mutation; (2) resistant or intolerant to previous TKI without known T315I mutation; and (3)with known T315I mutation irrespective of prior TKI treatment. Outcomes are evaluated separately for each cohort.
- Masking
- None (open label)
- Enrolment
- 50 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
- NCT07354074
- Sponsor's protocol ID
- CABL001I12202
- Other IDs
- 2025-522138-29, 2025
- Status last verified
- Oct 2026
- First submitted
- 15 Jan 2026
- Last updated
- 15 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- 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 expert 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 is currently available according to the process described on www.clinicalstudydatarequest.com.
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Multicenter, Open-label, Single Arm Study to Evaluate the Safety and Efficacy of Asciminib in Pediatric Participants Newly Diagnosed or Previously Treated With Philadelphia Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP) With or Without Known T315I Mutation
- ClinicalTrials.gov ID
- NCT07354074
- Acronym / sponsor ID
- CABL001I12202
- Conditions
- Chronic Myelogenous Leukemia, Leukemia, Myelogenous, Chronic, Philadelphia Chromosome Positive
- Keywords
- Asciminib, ABL001, Pediatric participants, Philadelphia chromosome positive chronic myeloid leukemia in chronic phase, Ph+ CML-CP, tyrosine kinase inhibitor, TKI, Molecular Response
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 28 Apr 2026 (actual)
- Primary completion
- 23 Feb 2033 (estimated)
- Study completion
- 23 Feb 2033 (estimated)
- First posted
- 21 Jan 2026 (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 aim of this study is to support development of asciminib in the pediatric population (1 to < 18 years) with Ph+ CML-CP. The study will evaluate the efficacy and safety of asciminib in pediatric formulation (weigh-based dose, fed state) or adult formulation (fasted) in newly diagnosed and resistant or intolerant Ph+ CML-CP with or without T315I mutation.
Detailed description
This is a multi-center, open-label, single arm study of asciminib in pediatric participants aged
1 to <18 years old with Ph+ CML-CP newly diagnosed and previously treated with TKI treatment, with or without T315I mutation.
The study population will consist of three cohorts of Ph+ CML-CP pediatric participants:
- Newly-diagnosed Ph+ CML-CP participants without known T315I mutation
- Ph+ CML-CP participants resistant or intolerant to previous TKI without known T315I mutation
- Ph+ CML-CP participants with known T315I mutation irrespective of prior TKI treatment
There is no fixed duration of study treatment for the participants. The study will end 5 years (240 weeks) after the last enrolled participants received their first dose of treatment in the study. The objective is to have enough follow up for safety, including growth and development and efficacy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Three cohorts of Ph+ CML-CP pediatric participants receive asciminib: (1) newly-diagnosed without known T315I mutation; (2) resistant or intolerant to previous TKI without known T315I mutation; and (3)with known T315I mutation irrespective of prior TKI treatment. Outcomes are evaluated separately for each cohort.
| Arm | Type | Receives |
|---|---|---|
| Single arm study | Experimental | Drug: Asciminib single agentThis study will enroll pediatric patients (≥ 1 and < 18 years of age) with newly diagnosed or previously treated with Philadelphia positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP) with or without known T315I mutation |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (16 listed)
- Participants eligible for inclusion in this study must meet all of the following criteria:
- Signed informed consent must be obtained prior to participation in the study.
- Male or female participants 1 and < 18 years of age at study enrollment
- Diagnosis of CML-CP (Apperley et al 2025) with cytogenetic confirmation of Philadelphia positive (Ph+) chromosome
- For participants with CML-CP newly diagnosed within 3 months of screening OR 5 For participants with CML - CP with high risk of developing resistance or intolerance to previous TKI:
Key exclusion criteria (5 listed)
- Known second chronic phase (CP) of CML after previous progression to Accelerated Phase (AP)/Blast Phase (BP).
- Previous treatment with a hematopoietic stem-cell transplantation.
- Patient planned to undergo allogeneic hematopoietic stem cell transplantation
- Known presence of a BCR::ABL1 mutation with known resistance to study treatment in accordance with the most recent public version of international CML clinical guidelines (e.g. NCCN CML treatment guidelines v 1.2026 and Apperley et al 2025) any time prior to study entry
- Other inclusion/exclusion criteria may apply.
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 |
|---|---|---|
| Asciminib single agentAlso: ABL001Asciminib (labelled as ABL001) administered as 40 mg tablet (adult formulation) or as 1 mg film-coated granules mini-tablets (pediatric formulation) | Drug | Single arm study |
Primary outcome
MMR at Week 48
Time frame: 48 weeks
MMR is defined as BCR::ABL1 IS ≤0.1%. MMR is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1 % BCR::ABL1/ABL % by international scale as measured by RQ-PCR.
17 secondary outcomes
MMR at Week 96
Time frame: 96 weeks
MMR at and by scheduled timepoints
Time frame: Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96
Hematologic response at and by scheduled timepoints
Time frame: Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96
Cytogenetic response at and by scheduled timepoints
Time frame: 5 years
Time to response
Time frame: 240 weeks after the last participant enrolled in the study received the first dose of treatment
Duration of response (limited to the binary response endpoints)
Time frame: 240 weeks after the last participant enrolled in the study received the first dose of treatment
Time to treatment failure (TTF)
Time frame: 240 weeks after the last participant enrolled in the study received the first dose of treatment
Time to disease progression
Time frame: 5 years
Event-free survival (EFS)
Time frame: 240 weeks after the last participant enrolled in the study received the first dose of treatment
Overall survival (OS)
Time frame: 240 weeks after the last participant enrolled in the study received the first dose of treatment
Growth: Height/length, weight, bone age measured by X-Ray
Time frame: 240 weeks after the last participant enrolled in the study received the first dose of treatment
Sexual maturation: Height/length, weight, bone age measured by Tanner staging
Time frame: 240 weeks after the last participant enrolled in the study received the first dose of treatment
PK parameters of asciminib: AUClast
Time frame: Week 2, Day 1 at 1, 3 and 6 hours post dose
PK parameters of asciminib: AUCtau
Time frame: Week 2, Day 1: pre dose (0 hour), Week 2: Day 1 at 1, 2, 3, and 6 hours post-dose
PK parameter of asciminib: Cmax
Time frame: Week 2, Day 1 at 1, 3, and 6 hours post dose
PK parameter of asciminib: Tmax
Time frame: Week 2, Day 1 at 1, 3, and 6 hours post dose
PK parameter of asciminib: Ctrough
Time frame: Week 2, Day 1: pre-dose (0 hr), Week 24: pre-dose (0 hr), Week 48: Pre-dose (0 hr)
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 15 Oct 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 expert 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 is currently available according to the 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
34 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Asciminib single agent in people with Chronic Myelogenous Leukemia and Leukemia, Myelogenous, Chronic, Philadelphia Chromosome Positive, run by Novartis Pharmaceuticals at 34 sites in 15 countries.
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 16 inclusion and 5 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 15 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 34 sites in United States, France, Brazil, Japan, Spain and 10 more countries. 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 (1-888-669-6682, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
MMR at Week 48 (time frame: 48 weeks). 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 15 Oct 2026.