AzTrials
AzTrials

Leukemia, Myeloid, AcutePhase 1CART33NCT05945849

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

A Phase 1 study testing CD33KO-HSPC; CART33 in people with Leukemia, Myeloid, Acute, run by University of Pennsylvania at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Leukemia, Myeloid, Acute
What is being tested?
CD33KO-HSPC; CART33
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
1 siteUnited States
Study dates
23 Feb 2024to 23 Feb 2044
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male or female 18 years of age or older
  • Subjects with AML unlikely to be cured with currently available therapies
  • AML that has not achieved a complete remission or morphologic leukemia free state by ELN criteria; partial remission or refractory disease (including primary refractory) are eligible; OR:
  • AML relapsed following allogeneic stem cell transplantation (including MDS evolved to AML post-allogeneic stem cell transplantation). Note: morphologic relapse is not required; persistent/recurrent disease-associated molecular, phenotypic or cytogenetic abnormalities (measurable residual disease, MRD) at any time after allogeneic HCT is eligible; OR:
  • 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.
Show all 15Show fewer
  • Satisfactory organ function
  • Creatinine clearance > 40 ml/min
  • ALT/AST must be ≤ 5x upper limit of normal unless related to disease and < 20 x upper limit of normal if related to disease
  • Direct bilirubin < 2.0 mg/dl, unless subject has Gilbert's syndrome (≤ 3.0 mg/dL)
  • Left ventricular ejection fraction ≥ 40% as confirmed by echocardiogram or MUGA
  • DLCO > 45% predicted
  • ECOG performance status 0-1
  • Written informed consent is given
  • Subjects of reproductive potential must agree to use acceptable birth control methods

Who cannot join

  • 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
  • Subjects with signs or symptoms indicative of CNS involvement.
  • Known history of allergy or hypersensitivity to study product excipients (human serum albumin, DMSO, and Dextran 40)
  • Class III/IV cardiovascular disability according to New York Heart Association Classification
  • Subjects with clinically apparent arrhythmia, or arrhythmias that are not stable on medical management, within 2 weeks of the screening/enrollment visit.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Abramson Cancer Center Clinical Trials Service contact

215-349-8245

PMCancerResearch@pennmedicine.upenn.edu

United States1 site, 1 recruiting

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

    Abramson Cancer Center Clinical Trials Service, 215-349-8245, PMCancerResearch@pennmedicine.upenn.edu

    Recruiting

Other trials in United States

About this study

Official title: Phase 1 Study of Lentivirally Transduced T Cells Engineered to Contain Anti-CD33 Linked to TCRζ And 4-1BB Signaling Domains In Combination With CD33KO-HSPC In Subjects With Refractory Or Relapsed Acute Myeloid Leukemia

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.

Being tested
CD33KO-HSPC; CART33
Medical subject headings
Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CD33KO-HSPC; CART33Biological

    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.

Study groups in NCT05945849
Study groupRoleWhat they receive
CD33KO-HSPC followed by CART33ExperimentalAll subjects will receive CD33KO-HSPC, followed by 1-3 CART-33 infusions
Receives: Biological: CD33KO-HSPC; CART33

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Manufacturing feasibility

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

    Measured: 1 month

  2. Occurrence of dose-limiting toxicities related to CD33KO-HSPC

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

    Measured: 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

    Measured: 6 months

Secondary outcomes (5)

  1. Efficacy of CD33KO-HSPC

    Proportion of subjects with hematopoietic engraftment according to standard criteria

    Measured: 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

    Measured: 6 months

  3. Overall Survival (OS)

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

    Measured: 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.

    Measured: 6 months, 12 months

  5. Duration of Response (DOR)

    Median number of months in remission. Median time to relapse in patients who receive CART-33 and attain a response.

    Measured: 15 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Jul 2023
  2. Study start23 Feb 2024
  3. Primary completion23 Feb 2029 (estimated)
  4. Study completion23 Feb 2044 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
16 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05945849
Sponsor's protocol ID
IRB # 854325, UPCC # 26423
Acronym
CART33
Status last verified
Jul 2026
First submitted
6 Jul 2023
Last updated
9 Jul 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CD33KO-HSPC; CART33 in people with Leukemia, Myeloid, Acute, run by University of Pennsylvania at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 15 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 9 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Abramson Cancer Center Clinical Trials Service (215-349-8245, PMCancerResearch@pennmedicine.upenn.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Manufacturing feasibility (time frame: 1 month). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05945849.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jul 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in