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Patients will receive CD123-targeted CAR-T cells in the dose-climbing trial. Each dose group has 3 patients and the the maximum dose can be extended.

From the minimum dose, If no DLT emerges in the group, then the next group uses the subsequent higher dose. If DLT emerges in a single subject in any dose level, 3 more subjects will be enrolled to the same dose level.

Third-generation anti-CD123 CAR-T cells

Yu Hu, M.D., Ph.D

Heng Mei, M.D., Ph.D

This is a single-center, open-label, single-arm study to evaluate the safety and efficacy of anti-CD123 CAR-T cells in patients with refractory/relapsed CD123+ Acute Myeloid Leukemia.

CD123 is a transmembrane subunit of the IL-3 receptor expressed on AML blasts. The investigators have conducted a third generationCD123-targeted CAR containing CD137 and CD28 costimulatory domains.This study aims to evaluate the safety and efficacy of anti-CD123 CAR-T cells in patients with relapsed/refractory CD123+ Acute Myeloid Leukemia.

This study was a single-center, open-label, single-arm, non-randomized,3+3 dose escalation clinical trial.Each dose group has 3 patients.If no DLT emerges in the group, then the next group uses the subsequent higher dose. If DLT emerges in a single subject in any dose level, 3 more subjects will be enrolled to the same dose level. The maximum dose could be extended.

Safety and Efficacy of Anti-CD123 CAR-T Therapy in Patients With Refractory/ Relapsed CD123+ Acute Myeloid Leukemia.

Single-center, Open-label, Single-arm Clinical Study of Efficacy and Safety of Anti-CD123 CAR-T Therapy in Patients With Refractory/Relapsed CD123+ Acute Myeloid Leukemia.

Therapy-related adverse events will be recorded and assessed according to the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE, Version 4.0).

ORR will be assessed from the first CAR-T cell infusion to death or last follow-up (censored).

OS will be assessed from the first CAR-T cell infusion to death or last follow-up (censored).

DOR will be assessed from the first CAR-T cell infusion to death or last follow-up (censored).

PFS will be assessed from the first CAR-T cell infusion to death or last follow-up (censored).

In vivo (bone marrow and peripheral blood) rate and quantity of anti-CD123 CAR-T cells were determined by means of flow cytometry.

In vivo (bone marrow and peripheral blood) quantity of anti-CD123 CAR copies were determined by means of qPCR.

Wuhan Bio-Raid Biotechnology Co., Ltd.

Wuhan Union Hospital, China

Principal Investigator

Dendritic Cell Tumor

Blastic Plasmacytoid Dendritic Cell

12 mcg/kg/day of tagraxofusp for 5 days, for at least 4 cycles of therapy; each cycle is 21 days.

Patients will receive the study drug until disease progression or in case of toxicity.

Tagraxofusp is provided as an intravenous (IV) injectable and administered as a 15-minute IV infusion. Cycle 1 will include a 2-day dosing period for the first 3 enrolled patients (Days 1-2), a 2-day plus an optional 3rd day for patients 4-6 (Days 1-2 + Day 3 if patient meets criteria for continued dosing) and a 3-day dosing period for patients 7 and beyond (Days 1-3). Cycle 2 and beyond will include a 5-day dosing period for all patients (Days 1-5). In all cycles and schedules, dose delays up to Day 10 of each cycle will be allowed for resolution of toxicities.

tagraxofusp

Paola Fazi

Enrico Crea

Alessandro Rambaldi

Antonio Curti

Chiara Cattaneo

Roberto Massimo Lemoli

Rosa Greco

Nicola Fracchiolla

Maria Paola Martelli

Monica Bocchia

Non-randomized, open-label, multicenter phase II Study for the treatment of

* 25 R/R BPDCN-IF (CD123/CD4/CD56 positive) AML patients and
* 25 patients presenting R/R AML CD123+, but negative for either, or both, CD4 and CD56.

Patients will be treated with 12 mcg/kg/day of tagraxofusp for 5 days, for at least 4 cicles.

Tagraxofusp in Patients With CD123+ or With BPDCN-IPh-like Acute Myeloid Leukemia

Tagraxofusp in Patients With CD123+ or With Blastic Plasmacytoid Dendritic Cell Neoplasm Immunophenotype-like Acute Myeloid Leukemia

Evaluate the activity of tagraxofusp, in terms of ORR (PR + CR + CRi), in patients with CD123+ or BlasticPlasmacytoid Dendritic Cell Neoplasm-like phenotype (BPDCN-IF) Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML).

Safety analysis according to CTCAE criteria

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Safety and Efficacy of Anti-CD123 CAR-T Therapy in Patients With Refractory/ Relapsed CD123+ Acute Myeloid Leukemia.	NCT04014881	CD123+ Acute My...	Third-generatio...	18 Years - 70 Years	Wuhan Union Hospital, China
Tagraxofusp in Patients With CD123+ or With BPDCN-IPh-like Acute Myeloid Leukemia	NCT04342962	Acute Myeloid L...	tagraxofusp	18 Years -	Gruppo Italiano Malattie EMatologiche dell'Adulto
Tagraxofusp in Patients With CD123+ or With BPDCN-IPh-like Acute Myeloid Leukemia	NCT04342962	Acute Myeloid L...	tagraxofusp	18 Years -	Gruppo Italiano Malattie EMatologiche dell'Adulto

Spots Global Cancer Trial Database for cd123+ acute myeloid leukemia