NCT07387926

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)

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

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 1 Year – 30 Years
• Healthy volunteers: Not accepted

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.

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). A separate cohort on this study will enroll participants with r/r ABL-class Ph-like 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: 1. Part 1 dose escalation using a Bayesian Optimal Interval (BOIN) statistical design, and after determination of RP2D, 2. 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 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

Interventions

Type	Name	Description
DRUG	Asciminib Adult formulation	oral, administered daily; Cycles 1, 2, 3
DRUG	Asciminib Pediatric formulation	oral, administered daily; Cycles 1, 2, 3
DRUG	Dexamethasone	Fixed doses, oral (preferred) or intravenous (IV) twice daily; Cycle 1, Days 1 - 14; (Cycle 1 = 28 days)
DRUG	Vincristine	Fixed doses, IV, weekly; Cycle 1
DRUG	Blinatumomab	Dosing based on bone marrow disease burden and weight. Continuous IV infusion; Cycles 2, 3
DRUG	Methotrexate (intrathecal)	Intrathecal
DRUG	Cytarabine (intrathecal)	Intrathecal
DRUG	Hydrocortisone (intrathecal)	Intrathecal
DRUG	Prednisolone (intrathecal)	Intrathecal

Timeline

• Start date: 2026-03-26
• Primary completion: 2033-01-10
• Completion: 2035-12-12
• First posted: 2026-02-04
• Last updated: 2026-02-04

Regulatory

• FDA-regulated drug study