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)

NCT07387926 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: CABL001L121012025-522019-40
• Study Type: interventional
• Target: 50
• Locations: 0 countries
• Sites: N/A

Brief 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. 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.

Conditions

Leukemia, Lymphoblastic, Acute, Philadelphia-Positive; Acute Lymphoblastic Leukemia; Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia

Keywords

Asciminib; ABL001; Ph+ chromosome acute lymphoblastic leukemia; ABL1/ABL2 fusion Ph-like ALL; BCR::ABL1; blinatumomab; dexamethasone; vincristine; chemotherapy

Outcome Measures

Primary Outcomes (2)

• Part 1 Dose Escalation: To determine the Recommended Phase II Dose (RP2D) of asciminib when administered with low intensity chemotherapy

  An integrated assessment of tolerability, safety profiles, dose-limiting toxicities (DLTs), treatment responses, and available pharmacokinetic (PK) data.

  During Cycle 1 (Cycle 1 = 28 days)

• Part 2 Dose Expansion: Assess the rate of complete remission (CR) at RP2D

  Percentage of participants achieving complete remission (CR)

  End of Cycle 1 (Cycle 1 = 28 days)

Secondary Outcomes (10)

• 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)

• Multiparametric flow cytometry (MFC) MRD negative CR/CRi rate

  At and by the end of Cycle 1 (Cycle 1 = 28 days), Cycle 2, and Cycle 3 (Cycle 2 & 3 = 42 days)

• Disease Free Survival (DFS)

  End of Cycle 3 (Cycle 3 = 42 days) in Part 2 of the study and at the end of study (EOS = approx. 3 years)

Study Arms (1)

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.

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)

Interventions

Asciminib Adult formulation DRUG

oral, administered daily; Cycles 1, 2, 3

Also known as: ABL001

Single Arm

Asciminib Pediatric formulation DRUG

oral, administered daily; Cycles 1, 2, 3

Also known as: ABL001

Single Arm

Dexamethasone DRUG

Fixed doses, oral (preferred) or intravenous (IV) twice daily; Cycle 1, Days 1 - 14; (Cycle 1 = 28 days)

Single Arm

Vincristine DRUG

Fixed doses, IV, weekly; Cycle 1

Single Arm

Blinatumomab DRUG

Dosing based on bone marrow disease burden and weight. Continuous IV infusion; Cycles 2, 3

Single Arm

Methotrexate (intrathecal) DRUG

Intrathecal

Single Arm

Cytarabine (intrathecal) DRUG

Intrathecal

Single Arm

Hydrocortisone (intrathecal) DRUG

Intrathecal

Single Arm

Prednisolone (intrathecal) DRUG

Intrathecal

Single Arm

Eligibility Criteria

• Age: 1 Year - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Evidence of Ph+ ALL or ABL1 or ABL2 fusion Ph-like ALL, inclusive of participants with ABL1 T315I mutations
• 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).
• a) 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Interventions

asciminib; Dexamethasone; Vincristine; blinatumomab; Methotrexate; Cytarabine; Hydrocortisone; Prednisolone

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated; Vinca Alkaloids; Secologanin Tryptamine Alkaloids; Indole Alkaloids; Alkaloids; Heterocyclic Compounds; Indoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Indolizidines; Indolizines; Aminopterin; Pterins; Pteridines; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Arabinonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Pregnenediones; Pregnenes; 11-Hydroxycorticosteroids; Hydroxycorticosteroids; Adrenal Cortex Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; 17-Hydroxycorticosteroids

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

1-888-669-6682; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+41613241111

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 13, 2026
• First Posted: February 4, 2026
• Study Start: March 26, 2026
• Primary Completion (Estimated): January 10, 2033
• Study Completion (Estimated): December 12, 2035
• Last Updated: February 4, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL