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Asciminib Adult formulation

Phase 1

Acute Lymphoblastic Leukemia | Small molecule | Oncology |Novartis AG|Last Updated: Jul 20, 2026

Target and mechanism

Molecular targetABL1, BCR
Target classInhibitor
ModalitySmall molecule

Also known as Asciminib Adult formulation group

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment50

FDA Designations

No designations recorded

Clinical trial landscape

Asciminib Adult formulation · 1 trial · 3 indications

Phase 1 1
NCT07387926Safety 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)Acute Lymphoblastic Leukemia
RECRUITING50 Analytics
PHASE1RECRUITING
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)
Acute Lymphoblastic LeukemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Part 1 Dose Escalation: Incidence of Dose Limiting Toxicities (DLTs) occurring during cycle 1 (debulking induction)
During Cycle 1 (Cycle 1 = 28 days)

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.

Part 1 Dose Escalation: Incidence and severity of adverse events (AEs) during cycle 1 (debulking induction)
During Cycle 1 (Cycle = 28 days)

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.

Part 2 Dose Expansion: Percentage of complete response/remission (CR) evaluable participants who achieve CR treated at recommended phase 2 dose (RP2D) (debulking induction)
End of Cycle 1 (Cycle 1 = 28 days)

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.

Secondary Endpoints

Complete remission (CR) rate
End of Cycle 2 and Cycle 3 (Cycle 2 & 3 = 42 days)
Overall response rate (ORR)
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
At and by the end of Cycle 1 (Cycle 1 = 28 days), Cycle 2, and Cycle 3 (Cycle 2 & 3 = 42 days)
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Single ArmEXPERIMENTALAsciminib 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.

Interventions

NameTypeDescription
Asciminib Adult formulationDRUGoral, administered daily (twice daily for participants with known T315I mutation); Cycles 1, 2, 3
Asciminib Pediatric formulationDRUGoral, administered daily (twice daily for participants with known T315I mutation); Cycles 1, 2, 3
DexamethasoneDRUGFixed doses, oral (preferred) or intravenous (IV) twice daily; Cycle 1, Days 1 - 14; (Cycle 1 = 28 days)
VincristineDRUGFixed doses, IV, weekly; Cycle 1
BlinatumomabDRUGDosing based on bone marrow disease burden and weight. Continuous IV infusion; Cycles 2, 3
Methotrexate (intrathecal)DRUGIntrathecal
Cytarabine (intrathecal)DRUGIntrathecal
Hydrocortisone (intrathecal)DRUGIntrathecal
Prednisolone (intrathecal)DRUGIntrathecal
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Eligibility Criteria

Age Range1 Year to 30 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * 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 eith...

Countries:Israel
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Recent Changes (Last 90 Days)

LOWJul 20, 2026NCT07387926Status: NOT_YET_RECRUITING → RECRUITING
LOWJul 20, 2026NCT07387926Status: NOT_YET_RECRUITING → RECRUITING

Frequently asked questions about Asciminib Adult formulation

What is Asciminib Adult formulation used for?

Asciminib Adult formulation is an investigational small molecule being studied for the treatment of Acute Lymphoblastic Leukemia (ALL), specifically in pediatric and young adult patients with relapse/refractory Philadelphia positive (Ph+) or ABL-class Ph-like ALL. It is currently in Phase 1 clinical development.

What does Asciminib Adult formulation target?

Asciminib Adult formulation is a kinase inhibitor, belonging to the -nib class of drugs. It is being evaluated for its safety and efficacy in patients with Philadelphia positive or ABL-class Ph-like acute lymphoblastic leukemia, targeting the kinase pathways involved in these diseases.

Who makes Asciminib Adult formulation?

Asciminib Adult formulation is being developed by Novartis AG, a multinational pharmaceutical company listed on the stock exchange under the ticker symbol NVS. The company is conducting clinical trials to evaluate the drug's safety and efficacy in acute lymphoblastic leukemia.

What phase is Asciminib Adult formulation in?

Asciminib Adult formulation is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory authorities and is still undergoing clinical trials to assess its safety and efficacy in patients.

What clinical trials is Asciminib Adult formulation in?

Asciminib Adult formulation is being studied in a Phase 1 clinical trial with the identifier NCT07387926. This trial is recruiting 50 participants and is evaluating the safety and efficacy of the drug in pediatric and young adult patients with relapse/refractory Philadelphia positive or ABL-class Ph-like acute lymphoblastic leukemia. The trial is being conducted in Israel.

Is Asciminib Adult formulation the same as Asciminib Adult formulation group?

Yes, Asciminib Adult formulation is also known as Asciminib Adult formulation group. These names refer to the same investigational drug being developed by Novartis AG for the treatment of acute lymphoblastic leukemia.