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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
2026-07-05
Completion Date
2036-05-30
Last Updated
2026-09-11
Healthy Volunteers
No
Conditions
Interventions
Asciminib Adult formulation
oral, administered daily (twice daily for participants with known T315I mutation); Cycles 1, 2, 3
Asciminib Pediatric formulation
oral, administered daily (twice daily for participants with known T315I mutation); 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
Locations (3)
Novartis Investigative Site
Sydney, New South Wales, Australia
Novartis Investigative Site
Ramat Gan, Israel
Novartis Investigative Site
Taipei, Taiwan