NCT05945849: CART33

CD33KO-HSPC Infusion Followed by CART-33 Infusion(s) for Refractory/Relapsed AML

Recruiting now Phase 1 Last updated 29 June 2025

What this trial tests

Phase 1 trial testing CD33KO-HSPC; CART33 in Leukemia, Myeloid, Acute in 16 participants. Currently enrolling.

Timeline

23 February 2024

Primary endpoint
23 February 2029

23 February 2044

Quick facts

Lead sponsor	University of Pennsylvania
Phase	Phase 1
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	16
Start date	23 February 2024
Primary completion	23 February 2029
Estimated completion	23 February 2044
Sites	1 location across United States

Drugs / interventions tested

• CD33KO-HSPC; CART33 — full drug profile →

Conditions studied

• Leukemia, Myeloid, Acute — all drugs for Leukemia, Myeloid, Acute →

Sponsor

University of Pennsylvania

Who can join

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

Sponsor's own description

The purpose of this study is to provide a new type of treatment for AML. This treatment combines a new type of stem cell transplant along with treatment using chimeric antigen receptor (CAR) T cells that have been engineered to recognize and attack your AML cells.

The first treatment is a modified stem cell transplant, using blood-forming stem cells donated from a healthy donor. From the same donor, we will also make CAR T-cells, which are leukemia fighting cells, which will be given to the patient via an infusion into the vein after the transplanted stem cells have started to grow healthy blood cells. The modification of the stem cell transplant means that the healthy bone marrow cells will be "invisible" to the CAR T-cells that are trying to kill the leukemia cells.

Publications & conference data

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

1. 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
2. Recent Advances in Immune-Based Therapies for Acute Myeloid Leukemia.
   Restelli C, Ruella M, Paruzzo L, Tarella C, et al · · 2024 · cited 36× · PMID 38904305 · DOI 10.1158/2643-3230.bcd-23-0202
3. TP53 -Mutated Myeloid Neoplasms: 2024 Update on Diagnosis, Risk-Stratification, and Management.
   Shah MV, Arber DA, Hiwase DK. · · 2025 · cited 15× · PMID 40066944 · DOI 10.1002/ajh.27655
4. A structural, genetic and clinical comparison of CAR-T cells and CAR-NK cells: companions or competitors?
   Andrea AE, Chiron A, Sarrabayrouse G, Bessoles S, et al · · 2024 · cited 8× · PMID 39430751 · DOI 10.3389/fimmu.2024.1459818
5. Recent advances in CAR-T cell therapy for acute myeloid leukaemia.
   Gao C, Li X, Xu Y, Zhang T, et al · · 2024 · cited 8× · PMID 38712978 · DOI 10.1111/jcmm.18369
6. The Immune Resistance Signature of Acute Myeloid Leukemia and Current Immunotherapy Strategies.
   Chandra DJ, Alber B, Saultz JN. · · 2024 · cited 7× · PMID 39123343 · DOI 10.3390/cancers16152615
7. Recent advances of CAR-T cells in acute myeloid leukemia.
   Deng H, Wang Q, Tong X, Cui Z, et al · · 2025 · cited 5× · PMID 40144774 · DOI 10.1177/20406207251326802
8. CAR-T and CAR-NK cell therapies in AML: breaking barriers and charting the future.
   Wu H, Shafiei FS, Taghinejad Z, Maleknia M, et al · · 2025 · cited 4× · PMID 41131610 · DOI 10.1186/s12967-025-07151-5