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RecruitingPhase 1

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

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Procedures mentioned: Infusions

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "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."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests a new treatment for adults with acute myeloid leukemia (AML), a blood cancer unlikely to be cured by current treatments. It combines changed blood-forming stem cells from a healthy donor with CAR T cells, immune cells made to attack leukemia. The stem cells are changed to protect healthy blood-forming cells from the CAR T cells.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Age 18 or older.
  • Have AML that is unlikely to be cured with current treatments and has not fully cleared or has returned after a donor stem cell transplant. Small amounts of cancer found by special tests after transplant may qualify.
  • If AML returned after a prior transplant, have stopped medicines that lower immune activity throughout the body for at least 1 month before joining.
  • Have a suitable stem cell donor.
  • Meet the study’s kidney and liver test limits.
  • Have a heart pumping strength of at least 40% and a lung gas-transfer test result above 45% of the expected value.
  • Be fully active or able to walk and do light work, even if hard physical activity is limited.
  • Give written consent to join. If able to have children, agree to use acceptable birth control.

Who can't join

  • Are pregnant or breastfeeding.
  • Have active hepatitis B, hepatitis C, or HIV infection.
  • Currently use steroids or other medicines that lower immune activity throughout the body.
  • Have an uncontrolled medical problem that would prevent taking part.
  • Have signs or symptoms that suggest cancer affects the brain or spinal cord.
  • Have a known allergy to human serum albumin, DMSO, or Dextran 40, ingredients in the study treatment.
  • Have severe heart-related limits on activity, called New York Heart Association class III or IV.
  • Have an irregular heartbeat that is clearly present or not stable with treatment within 2 weeks of the screening or enrollment visit.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: 1. Male or female 18 years of age or older 2. Subjects with AML unlikely to be cured with currently available therapies 1. 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: 2. 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: 3. Subjects with relapsed disease after prior transplant must be off systemic immunosuppression for at least 1 month at the time of enrollment. 3. Subjects must have a suitable stem cell donor. 4. Satisfactory organ function 1. Creatinine clearance \> 40 ml/min 2. ALT/AST must be ≤ 5x upper limit of normal unless related to disease and \< 20 x upper limit of normal if related to disease 3. Direct bilirubin \< 2.0 mg/dl, unless subject has Gilbert's syndrome (≤ 3.0 mg/dL) 5. Left ventricular ejection fraction ≥ 40% as confirmed by echocardiogram or MUGA 6. DLCO \> 45% predicted 7. ECOG performance status 0-1 8. Written informed consent is given 9. Subjects of reproductive potential must agree to use acceptable birth control methods Exclusion Criteria: 1. Pregnant or lactating (nursing) women 2. Active hepatitis B or hepatitis C or HIV infection 3. Concurrent use of systemic steroids or immunosuppressant medications 4. Any uncontrolled active medical disorder that would preclude participation as outlined 5. Subjects with signs or symptoms indicative of CNS involvement. 6. Known history of allergy or hypersensitivity to study product excipients (human serum albumin, DMSO, and Dextran 40) 7. Class III/IV cardiovascular disability according to New York Heart Association Classification 8. Subjects with clinically apparent arrhythmia, or arrhythmias that are not stable on medical management, within 2 weeks of the screening/enrollment visit.

Study sites (1)

PA