NCT04219163

Chimeric Antigen Receptor T-cells for The Treatment of AML Expressing CLL-1 Antigen

Active, enrolled Phase 1 Last updated 22 September 2025

What this trial tests

Phase 1 trial testing CLL-1.CAR T cells in Acute Myeloid Leukemia in 18 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

9 July 2020

Primary endpoint
24 March 2025

31 July 2038

Quick facts

Lead sponsor	Baylor College of Medicine
Phase	Phase 1
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	18
Start date	9 July 2020
Primary completion	24 March 2025
Estimated completion	31 July 2038
Sites	1 location across United States

Drugs / interventions tested

• CLL-1.CAR T cells — full drug profile →

Conditions studied

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

Sponsor

Baylor College of Medicine

Who can join

Under 75, any sex, with Acute Myeloid Leukemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Patients eligible for this study have a type of blood cancer Acute Myeloid Leukemia (AML) which has come back or has not gone away after treatment.

The body has different ways of fighting disease and infection, and this research study combines two different ways of fighting cancer with antibodies and T cells with the hope that they will work together. T cells (also called T lymphocytes) are special infection-fighting blood cells that can kill other cells including tumor cells. Antibodies are types of proteins that protect the body from bacterial and other infectious diseases. Both antibodies and T cells have been used to treat patients with cancers; they have shown promise, but have not been strong enough to cure most patients when used alone.

T lymphocytes can kill tumor cells but there normally are not enough of them to kill all the tumor cells. Some researchers have taken T cells from a person's blood, grown more of them in the laboratory and then given them back to the person. The antibody used in this study targets CLL-1. This antibody sticks to AML cells because of a substance (protein) on the outside of these cells called CLL-1. For this study, the antibody to CLL-1 has been changed so that instead of floating free in the blood, it is now joined to the T cells. When T-cells contain an antibody that is joined to them, they are called chimeric antigen receptor T-cells or CAR-T cells.

In the laboratory, the investigators have also found that T cells work better if proteins that stimulate T cells are also added, such as one called CD28. Adding the CD28 makes the cells grow better and last longer in the body, thus giving the cells a better chance of killing the leukemia or lymphoma cells. In this study we are going to attach the CLL-1 chimeric receptor that has CD28 added to it to the patient's T cells. We will then test how long the cells last.

These CLL-1 chimeric antigen receptor T cells with CD28 are investigational products not approved by the Food and Drug Administration.

Publications & conference data

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

1. Cancer stem cells: advances in knowledge and implications for cancer therapy.
   Chu X, Tian W, Ning J, Xiao G, et al · · 2024 · cited 311× · PMID 38965243 · DOI 10.1038/s41392-024-01851-y
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 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
4. CAR T cells redirected to cell surface GRP78 display robust anti-acute myeloid leukemia activity and do not target hematopoietic progenitor cells.
   Hebbar N, Epperly R, Vaidya A, Thanekar U, et al · · 2022 · cited 70× · PMID 35102167 · DOI 10.1038/s41467-022-28243-6
5. CD33 Expression and Gentuzumab Ozogamicin in Acute Myeloid Leukemia: Two Sides of the Same Coin.
   Molica M, Perrone S, Mazzone C, Niscola P, et al · · 2021 · cited 42× · PMID 34203180 · DOI 10.3390/cancers13133214
6. Unveiling the potential of CLL-1: a promising target for AML therapy.
   Soleimani Samarkhazan H, Zehtabcheh S, Seraji HR, Beqaj SH, et al · · 2025 · cited 37× · PMID 39940055 · DOI 10.1186/s40364-025-00738-6
7. 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
8. Challenges and Advances in Chimeric Antigen Receptor Therapy for Acute Myeloid Leukemia.
   Marvin-Peek J, Savani BN, Olalekan OO, Dholaria B. · · 2022 · cited 30× · PMID 35158765 · DOI 10.3390/cancers14030497