A Phase 2 interventional study of Asciminib single agent in Chronic Myelogenous Leukemia and Leukemia, Myelogenous, Chronic, Philadelphia Chromosome Positive, sponsored by Novartis Pharmaceuticals. Recruiting at 39 sites in 17 countries. Open to participants aged 1 Year to 18 Years. Per ClinicalTrials.gov, last updated 2026-11-10.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 2, Interventional, and Treatment
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.
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:
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.
894 studies on the registry are indexed under Leukemia, Myelogenous, Chronic, BCR-ABL Positive; 109 are open to participants now.
This study's planned enrollment of 50 is above the median of 44 across 681 interventional studies indexed under Leukemia, Myelogenous, Chronic, BCR-ABL Positive.
Browse Leukemia, Myelogenous, Chronic, BCR-ABL Positive studies →Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Participants eligible for inclusion in this study must meet all of the following criteria:
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:
Intolerance to TKI is defined as:
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: ≥ 50% for participants ≥ 16 years of age, and Lansky ≥ 50 for participants \< 16 years of age at the time of screening.
Key Exclusion Criteria:
Other inclusion/exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
Drug: Asciminib single agent
Asciminib (labelled as ABL001) administered as 40 mg tablet (adult formulation) or as 1 mg film-coated granules mini-tablets (pediatric formulation)
Also known as: ABL001
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.
Time frame: 48 weeks
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.
Time frame: 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.
Time frame: 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
Time frame: 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.
Time frame: 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.
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)
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.
Time frame: 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.
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)
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.
Time frame: 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).
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
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.
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
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.
Time frame: 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.
Time frame: 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.
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
Cmax is the maximum (peak) observed plasma drug concentration after single dose administration.
Time frame: 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.
Time frame: 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.
Time frame: Week 2, Day 1: pre-dose (0 hr), Week 24: pre-dose (0 hr), Week 48: Pre-dose (0 hr)
Plan to share: 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.
Data sharing details ↗ · www.clinicalstudydatarequest.com
No publications or documents are linked to this record.
Started Apr 2026 (actual). Primary completion is estimated for Feb 2033, 81 months after the start.
Of 25 industry-sponsored phase 2 interventional studies under Leukemia, Myelogenous, Chronic, BCR-ABL Positive, half reached primary completion within 44 months of starting (the median), counting those still running; a quarter within 33 and three quarters within 69. This study's planned 81 months is above that.
Counting every sponsor, half of 126 studies reached it within 73 months; a quarter within 43 and three quarters within 124.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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