NCT03766126

Lentivirally Redirected CD123 Autologous T Cells in AML

Active, enrolled Phase 1 Last updated 26 June 2025

What this trial tests

Phase 1 trial testing CART123 cells; cyclophosphamide; fludarabine in Acute Myeloid Leukemia, in Relapse in 22 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

6 December 2018

Primary endpoint
3 December 2033

3 December 2033

Quick facts

Lead sponsor	University of Pennsylvania
Phase	Phase 1
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	22
Start date	6 December 2018
Primary completion	3 December 2033
Estimated completion	3 December 2033
Sites	1 location across United States

Drugs / interventions tested

• CART123 cells; cyclophosphamide; fludarabine — full drug profile →

Conditions studied

• Acute Myeloid Leukemia, in Relapse — all drugs for Acute Myeloid Leukemia, in Relapse →
• Acute Myeloid Leukemia, Adult — all drugs for Acute Myeloid Leukemia, Adult →
• Acute Myeloid Leukemia, Refractory — all drugs for Acute Myeloid Leukemia, Refractory →

Sponsor

University of Pennsylvania

Who can join

18 and older, any sex, with Acute Myeloid Leukemia, in Relapse or Acute Myeloid Leukemia, Adult. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Phase 1 open-label study to estimate the safety, manufacturing feasibility, and efficacy of intravenously administered, lentivirally transduced T cells expressing anti-CD123 chimeric antigen receptors expressing tandem TCRζ and 4-1BB (TCRζ /4-1BB) costimulatory domains in Acute Myeloid Leukemia (AML) subjects.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Cancer Stem Cells-Origins and Biomarkers: Perspectives for Targeted Personalized Therapies.
   Walcher L, Kistenmacher AK, Suo H, Kitte R, et al · · 2020 · cited 688× · PMID 32849491 · DOI 10.3389/fimmu.2020.01280
2. Autologous CD33-CAR-T cells for treatment of relapsed/refractory acute myelogenous leukemia.
   Tambaro FP, Singh H, Jones E, Rytting M, et al · · 2021 · cited 159× · PMID 33833386 · DOI 10.1038/s41375-021-01232-2
3. CAR T Cells for Acute Myeloid Leukemia: State of the Art and Future Directions.
   Mardiana S, Gill S. · · 2020 · cited 155× · PMID 32435621 · DOI 10.3389/fonc.2020.00697
4. CD123 as a Therapeutic Target in the Treatment of Hematological Malignancies.
   Testa U, Pelosi E, Castelli G. · · 2019 · cited 114× · PMID 31547472 · DOI 10.3390/cancers11091358
5. Safe and effective off-the-shelf immunotherapy based on CAR.CD123-NK cells for the treatment of acute myeloid leukaemia.
   Caruso S, De Angelis B, Del Bufalo F, Ciccone R, et al · · 2022 · cited 90× · PMID 36335396 · DOI 10.1186/s13045-022-01376-3
6. Chimeric Antigen Receptor (CAR) T Cell Therapy in Acute Myeloid Leukemia (AML).
   Hofmann S, Schubert ML, Wang L, He B, et al · · 2019 · cited 84× · PMID 30736352 · DOI 10.3390/jcm8020200
7. CAR-T cell therapy for cancer: current challenges and future directions.
   Zugasti I, Espinosa-Aroca L, Fidyt K, Mulens-Arias V, et al · · 2025 · cited 81× · PMID 40610404 · DOI 10.1038/s41392-025-02269-w
8. Immunotherapy in Acute Myeloid Leukemia: Where We Stand.
   Isidori A, Cerchione C, Daver N, DiNardo C, et al · · 2021 · cited 76× · PMID 34041025 · DOI 10.3389/fonc.2021.656218