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

ConditionLeukemia, Myeloid, Acute
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorUniversity of Pennsylvania

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Male or female 18 years of age or older

Subjects with AML unlikely to be cured with currently available therapies

Subjects with relapsed disease after prior transplant must be off systemic immunosuppression for at least 1 month at the time of enrollment.

Subjects must have a suitable stem cell donor.

Disqualifiers

Pregnant or lactating (nursing) women

Active hepatitis B or hepatitis C or HIV infection

Concurrent use of systemic steroids or immunosuppressant medications

Any uncontrolled active medical disorder that would preclude participation as outlined

Trial design

Design model

Single group

Treatments tested in this trial

  • CD33KO-HSPC; CART33

    Biological/Vaccine

    CD33KO-HSPC: Stem cell transplant (also known as bone marrow transplant) is a common treatment used for patients with blood cancers, but for this transplant we will first modify the cells, in order to make the CAR T-cell treatment safer for when the patient receives them later. The modification is a type of gene editing - this means changing the DNA of the cells, so that a protein that the bone marrow stem cells usually show on their surface is not shown any more. This makes the bone marrow cells "invisible" to the CAR T-cells, and makes this therapy safer for the patient. The protein is called CD33. CART33: Chimeric Antigen Receptor T-cells (CART) are immune cells which are modified by adding a CAR molecule, which makes them much more efficient at finding and killing cancer cells. In this case, the CAR T-cells are programmed to target a protein called CD33, which is found on the surface of leukemia cells, and on healthy bone marrow cells.

Treatment groups

16 Participants
are divided into 1 treatment group
Group A: CD33KO-HSPC followed by CART33Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Manufacturing feasibility

Proportion of subjects whose Product 1 (CD33KO-HSPC) meets release criteria.

Time frame
1 month
2

Occurrence of dose-limiting toxicities related to CD33KO-HSPC

Safety of alloHSCT: occurrence of dose-limiting toxicities related to CD33KO-HSPC

Time frame
3 months
3

Occurrence of dose-limiting toxicities related to CART-33

Safety of CART-33: occurrence of dose-limiting toxicities related to CART-33

Time frame
6 months

Secondary outcomes

1

Efficacy of CD33KO-HSPC

Proportion of subjects with hematopoietic engraftment according to standard criteria

Time frame
1 month
2

Efficacy of at least 1 dose of CART-33

Proportion of subjects with residual or recurrent AML before CART-33 infusion who attain a clinical response

Time frame
6 months
3

Overall Survival (OS)

Proportion of patients who are alive at 6 months and at 12 months

Time frame
6 months, 12 months
4

Progression free survival (PFS)

Proportion of patients who remained in response at 6 and 12 months after attaining a response to the first CART-33 infusion. Median time to progression of AML from infusion of CART-33.

Time frame
6 months, 12 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

University of Pennsylvania

Lead sponsor

Source ClinicalTrials.gov
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