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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)
Sponsor: Novartis Pharmaceuticals
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.
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
Key Details
Gender
All
Age Range
1 Year - 30 Years
Study Type
INTERVENTIONAL
Enrollment
50
Start Date
2026-03-26
Completion Date
2035-12-12
Last Updated
2026-02-04
Healthy Volunteers
No
Conditions
Interventions
Asciminib Adult formulation
oral, administered daily; Cycles 1, 2, 3
Asciminib Pediatric formulation
oral, administered daily; Cycles 1, 2, 3
Dexamethasone
Fixed doses, oral (preferred) or intravenous (IV) twice daily; Cycle 1, Days 1 - 14; (Cycle 1 = 28 days)
Vincristine
Fixed doses, IV, weekly; Cycle 1
Blinatumomab
Dosing based on bone marrow disease burden and weight. Continuous IV infusion; Cycles 2, 3
Methotrexate (intrathecal)
Intrathecal
Cytarabine (intrathecal)
Intrathecal
Hydrocortisone (intrathecal)
Intrathecal
Prednisolone (intrathecal)
Intrathecal