Acute Lymphoblastic LeukemiaPhase 1/2NCT07387926
Safety and Efficacy of Asciminib in Pediatrics and Young Adults With Relapse/Refractory (r/r) Philadelphia Positive (Ph+) or ABL-class Ph-like Acute Lymphoblastic Leukemia (ALL)
A Phase 1/2 study testing Asciminib Adult formulation, Asciminib Pediatric formulation and Dexamethasone in people with Acute Lymphoblastic Leukemia and Leukemia, Lymphoblastic, Acute, Philadelphia-Positive, run by Novartis Pharmaceuticals at 4 sites in 4 countries.
Trial summary
Multi-center, open-label, single arm study of asciminib in participants aged ≥1 year to ≤30 years old with r/r Ph+ or ABL-class Ph-like ALL. This study will have 2 parts: Part 1 dose escalation and Part 2 dose expansion. Read more about this study
- Who is it for?
- Ages 1 year to 30 years
- Conditions the study is for
- Acute Lymphoblastic Leukemia, Leukemia, Lymphoblastic, Acute, Philadelphia-Positive, Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia
- What is being tested?
- Asciminib Adult formulation, Asciminib Pediatric formulation and Dexamethasone
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 4 sitesin 4 countries
- Study dates
- 5 Jul 2026to 30 May 2036
- Phase
- Phase 1/2
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 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Evidence of Ph+ ALL or ABL1 or ABL2 fusion Ph-like ALL, inclusive of participants with ABL1 T315I mutation
- Participants with CNS1, CNS2, CNS3a, or CNS3b at screening
- Active B-Cell ALL at screening defined by MFC or IG/TCR PCR of ALL blasts >0.01% in participants with either:
- Primary refractory disease (>0.01% ALL blasts present at the end of consolidation) OR
- Relapsed ALL with evidence of involvement of BM with ALL (MFC or IG/TCR PCR >0.01%) after at least one line of therapy
- Documented history of CD19 expressing B-cell ALL (in peripheral blood or bone marrow by flow cytometry).
Show all 12Show fewer
- For participants who received anti-CD19 targeted therapy (e.g CD19 CAR T cells or blinatumomab), CD19 expressing B-cell ALL must be documented after anti-CD19 therapy completion prior to cycle 1 day 1
- Adequate hepatic and renal function (local laboratory analysis) as defined:
- ALT ≤ 5x upper limit of normal (ULN) for age
- Total bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x ULN) for age, except for participants with Gilbert's syndrome who may only be included if total bilirubin ≤ 3.0 x ULN or direct bilirubin ≤ 1.5 x ULN
- Estimated glomerular filtration rate (eGFR) using the Cockcroft-Gault formula in participants ≥ 18 years, OR radioisotope GFR ≥50 mL/min/1.73 m^2, OR creatinine based on age and sex for participants < 18 years old
- Adequate cardiac function defined as shortening fraction ≥27% by echocardiogram (ECHO) OR left ventricular ejection fraction of ≥50% by ECHO
Who cannot join
- Participants with >3 relapses of ALL
- Extramedullary disease (non-CNS and/or isolated CNS disease)
- Participants with CNS3c (Clinical signs of CNS leukemia (such as facial nerve palsy, brain/eye involvement or hypothalamic syndrome))
- Cardiac or cardiac repolarization abnormality, including but not limited to clinically significant cardiac arrhythmias, long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome or other clinically significant heart disease (e.g., congestive heart failure, etc.)
- 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.
- Other protocol defined inclusion/exclusion criteria may apply.
Trial locations and contacts
4 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
4 sites in 4 countries
Australia1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Sydney, New South Wales, 2031
Israel1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Ramat Gan, 5265601
South Korea1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Seoul, 03080
About this study
Official title: Open-label, Phase I/II Study to Evaluate Safety and Efficacy of Asciminib With Chemotherapy Followed by Asciminib Plus Blinatumomab in Pediatric, Adolescent, and Young Adults With Relapsed or Refractory BCR::ABL1-positive (Philadelphia Positive, Ph+) or BCR::ABL1-like (Ph-like) ALL
Multi-center, open-label, single arm study of asciminib in participants aged ≥1 year to ≤30 years old with r/r Ph+ or ABL-class Ph-like ALL. This study will have 2 parts: Part 1 dose escalation and Part 2 dose expansion. Part 1 dose escalation will enroll participants aged ≥1 year to ≤30 years to determine the recommended phase 2 dose (RP2D) of asciminib when administered with low intensity chemotherapy. Part 2 dose expansion will enroll participants aged ≥1 year to ≤30 years to evaluate safety, tolerability, and efficacy of asciminib at the RP2D with the treatment regimen.
Read the detailed description
This is a single arm phase I/II multicenter study to assess the safety and efficacy of asciminib at the RP2D in combination with low intensity chemotherapy (debulking induction) followed by asciminib plus blinatumomab (consolidation) in pediatric and young adult participants with r/r Ph+ ALL (inclusive of participants with T315I mutation).
The aim of the study design is to explore a novel treatment regimen which is expected to be more tolerable than the high intensity chemotherapy backbone-based regimens.
This study will consist of a 2-part design:
Part 1 dose escalation using a Bayesian Optimal Interval (BOIN) statistical design, and after determination of RP2D Part 2 dose expansion.
Participants will only enroll in either Part 1 or Part 2, and not both.
Both Part 1 and Part 2 (dose escalation and dose expansion) will have the following phases:
- Core Study Treatment Phase
- Survival Follow up Phase
Participants with known T315I mutation will not participate in Part 1 or Part 2. They will be part of a separate cohort.
The core study treatment phase will consist of 3 cycles of therapy: cycle 1 asciminib with low intensity chemotherapy (debulking induction), followed by cycle 2 (blinatumomab-block 1 with asciminib) and cycle 3 (blinatumomab-block 2 with asciminib).
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Lymphoblastic, Acute, Philadelphia-Positive, Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia
- Being tested
- Asciminib Adult formulation, Asciminib Pediatric formulation, Dexamethasone, Vincristine, Blinatumomab
- Keywords
- Asciminib, ABL001, Ph+ chromosome acute lymphoblastic leukemia, ABL1/ABL2 fusion Ph-like ALL, BCR::ABL1, blinatumomab, dexamethasone, vincristine, chemotherapy
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Asciminib Adult formulationDrug
Also called ABL001
oral, administered daily (twice daily for participants with known T315I mutation); Cycles 1, 2, 3
Asciminib Pediatric formulationDrug
Also called ABL001
oral, administered daily (twice daily for participants with known T315I mutation); Cycles 1, 2, 3
DexamethasoneDrug
Fixed doses, oral (preferred) or intravenous (IV) twice daily; Cycle 1, Days 1 - 14; (Cycle 1 = 28 days)
VincristineDrug
Fixed doses, IV, weekly; Cycle 1
BlinatumomabDrug
Dosing based on bone marrow disease burden and weight. Continuous IV infusion; Cycles 2, 3
Methotrexate (intrathecal)Drug
Intrathecal
Cytarabine (intrathecal)Drug
Intrathecal
Hydrocortisone (intrathecal)Drug
Intrathecal
Prednisolone (intrathecal)Drug
Intrathecal
| Study group | Role | What they receive |
|---|---|---|
| Single Arm | Experimental | Asciminib Adult formulation group: escalating doses evaluated
Asciminib Pediatric formulation group: dose is based on body weight; dose level being evaluated will be converted into a mg/kg daily dose.
For known T315I mutation:
Fixed asciminib dose twice daily or the pediatric formulation dose equivalent twice daily. Receives: Drug: Asciminib Adult formulation; Drug: Asciminib Pediatric formulation; Drug: Dexamethasone; Drug: Vincristine; Drug: Blinatumomab; Drug: Methotrexate (intrathecal); Drug: Cytarabine (intrathecal); Drug: Hydrocortisone (intrathecal); Drug: Prednisolone (intrathecal) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part 1 Dose Escalation: Incidence of Dose Limiting Toxicities (DLTs) occurring during cycle 1 (debulking induction)
A dose-limiting toxicity (DLT) is defined as an adverse event which starts between Day 1 and Day 28, is suspected by the investigator to be related to asciminib, and meets one of the several criteria.
Measured: During Cycle 1 (Cycle 1 = 28 days)
Part 1 Dose Escalation: Incidence and severity of adverse events (AEs) during cycle 1 (debulking induction)
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment.
Measured: During Cycle 1 (Cycle = 28 days)
Part 2 Dose Expansion: Percentage of complete response/remission (CR) evaluable participants who achieve CR treated at recommended phase 2 dose (RP2D) (debulking induction)
Participants with Complete Response/Remission (CR) will achieve the following:
No circulating blasts or extramedullary disease; No lymphadenopathy, splenomegaly, skin/gum infiltration, testicular mass/CNS involvement; Marrow <5% blasts (M1) OR < 1% blasts by flow cytometry. If a discrepancy occurs between disease detection methods, the result of the flow cytometry assay will be used to determine CR status; Peripheral blood count recovery; ANC > 1000μL and platelets > 100,000μL.
CR evaluable: Participants will be considered CR evaluable for Part 2 if they have relapsed/refractory Ph+ ALL defined as either >1% bone marrow (BM) blasts by MFC or immunoglobulin/T-cell receptor (IG/TCR) PCR, OR > 5% BM blasts by morphologic evaluation at enrollment and are treated at RP2D.
Measured: End of Cycle 1 (Cycle 1 = 28 days)
Secondary outcomes (12)
Complete remission (CR) rate
Percentage of participants who had a CR
Measured: End of Cycle 2 and Cycle 3 (Cycle 2 & 3 = 42 days)
Overall response rate (ORR)
Percentage of participants who had CR and/or complete remission with incomplete blood count recovery (CRi)
Measured: At and by the end of Cycle 1 (Cycle 1 = 28 days), Cycle 2, and Cycle 3 (Cycle 2 & 3 = 42 days)
Next generation sequencing (NGS) minimal residual disease (MRD) negative rate
Percentage of participants who have NGS MRD negative CR at and by the end of cycle 1 (debulking induction), cycle 2 and cycle 3 (consolidation).
Measured: At and by the end of Cycle 1 (Cycle 1 = 28 days), Cycle 2, and Cycle 3 (Cycle 2 & 3 = 42 days)
Multiparametric flow cytometry (MFC) MRD negative CR rate
Percentage of participants who have MFC MRD negative CR at and by the end of cycle 1 (debulking induction), cycle 2 and cycle 3 (consolidation).
Measured: At and by the end of Cycle 1 (Cycle 1 = 28 days), Cycle 2, and Cycle 3 (Cycle 2 & 3 = 42 days)
Overall MRD negative response (MRD negative CR and/or CRi rate) by NGS
Overall MRD negative response is the percentage of participants who have NGS MRD negative complete response (CR) and complete response incomplete blood count recovery (CRi).
Measured: at and by the end of cycle 1 (debulking induction) (Cycle 1 = 28 days), cycle 2, and cycle 3 (consolidation) (each cycle = 42 days)
Overall MRD negative response (MRD negative CR and/or CRi rate) by MFC
Overall MRD negative response is the percentage of participants who have MFC MRD negative complete response (CR) and complete response incomplete blood count recovery (CRi).
Measured: at and by the end of cycle 1 (Cycle 1 = 28 days) (debulking induction), cycle 2, cycle 3 (consolidation) (each cycle = 42 days)
Disease Free Survival (DFS)
The period from when a participant is in remission until they have relapse or death due to any reason.
Measured: End of Cycle 3 (Cycle 3 = 42 days) in Part 2 of the study and at the end of study (EOS = approx. 3 years)
Overall survival (OS)
The duration of time from treatment initiation until the participant's death due to any reason.
Measured: End of Cycle 3 (Cycle 3 = 42 days) in Part 2 of the study and at the end of study (EOS = approx. 3 years)
Pharmacokinetic (PK) parameter of asciminib at steady state: AUClast
Area Under the Curve to the Last Measurable Concentration-represents the total drug exposure (area under the plasma concentration-time curve) from the time of administration until the last measurable concentration
Measured: Cycle 1, Day 8 (Cycle 1 = 28 days)
PK parameter of asciminib at steady state: Cmax
Maximum concentration-the peak (highest) plasma drug concentration observed after administration
Measured: Cycle 1, Day 8 (Cycle 1 = 28 days)
PK parameter of asciminib at steady state: Tmax
The time it takes to reach the Cmax after drug administration
Measured: Cycle 1, Day 8 (Cycle 1 = 28 days)
PK parameter of asciminib at steady state: Ctrough
Trough concentration- lowest drug concentration observed, typically right before the next dose is administered (steady-state condition)
Measured: Cycle 1, Day 8 (Cycle 1 = 28 days); Cycle 2, Day 22 (Cycle 2 = 42 days); Cycle 3, Day 22 (Cycle 3 = 42 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Feb 2026
- Study start5 Jul 2026
- Primary completion19 Sep 2033 (estimated)
- Study completion30 May 2036 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Lymphoblastic 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- 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
- NCT07387926
- Sponsor's protocol ID
- CABL001L12101
- Other IDs
- 2025-522019-40, 2025
- Status last verified
- Oct 2026
- First submitted
- 13 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.