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CART123 Cells With or Without Ruxolitinib in Relapsed/Refractory Acute Myeloid Leukemia
Sponsor: Stephan Grupp MD PhD
Summary
This study is designed to evaluate the safety and effectiveness of CART123 cells either alone or when combined with ruxolitinib in pediatric and young adult subjects with relapsed or refractory AML. Subjects will be enrolled into one of two treatment cohorts: subjects who will receive CART123 alone (Cohort A) or subjects who will receive CART123 in combination with ruxolitinib (Cohort B).
Official title: Phase 1 Trial of Autologous CD123-Directed CAR T-Cells (CART123) as Monotherapy or in Combination With Ruxolitinib in Relapsed/Refractory Acute Myeloid Leukemia
Key Details
Gender
All
Age Range
0 Years - 29 Years
Study Type
INTERVENTIONAL
Enrollment
30
Start Date
2026-04-01
Completion Date
2030-04-30
Last Updated
2026-03-11
Healthy Volunteers
No
Conditions
Interventions
Anti-CD123 LV redirected T cells (CART123)
CART123 cells: lentivirally transduced T cells expressing anti-CD123 chimeric antigen receptors expressing tandem TCRζ and 4-1BB (TCRζ /4-1BB) costimulatory domains.
Ruxolitinib (JAKAVI®)
Ruxolitinib: an orally administered janus-activated kinase (JAK) inhibitor that selectively inhibits JAK1 and JAK2.
Locations (1)
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States