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New treatment option for AML, Childhood
Official title Pilot Study of Memory-like Natural Killer (ML NK) Cells After TCRαβ T Cell Depleted Haploidentical Transplant in AML
ClinicalTrials.gov ID: NCT06158828
What this study is testing
What is Rabbit Anti thymocyte globulin?
Rabbit Anti thymocyte globulin is an investigational medicine, given as an once-daily infusion into a vein, being studied as a potential treatment for aml, childhood.
Also referred to as rATG.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This trial represents a single institution phase I/II pilot study with the primary objective of establishing the safety and feasibility of generating and infusing ML NK cells after TCRαβ haplo-HCT.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Patient - Cohort 1:
- High risk acute myeloid leukemia (AML) in either:
- Complete remission (CR) defined by \< 5% marrow blasts by morphology in the context of hematological recovery (ANC ≥ 0.5× 10\^9/L, platelet count ≥...
- Morphological leukemia free state (MLFS) defined by the absence of hematological recovery and \< 5% marrow blasts by morphology
- Patients must further meet one of the below for inclusion into the study:
You likely can't join if
- Both Cohorts
- Active GvHD. If patient had prior GvHD, patient must be off immunosuppression for at least 3 months prior to starting study treatment.
- Active non-hematologic malignancy. History of other malignancy is acceptable as long as therapy has been completed and there is no current evidence...
- Currently receiving any other investigational agents at the time of transplant.
- Active CNS or extramedullary disease. History of CNS or extramedullary disease currently in remission is acceptable.
- A history of allergic reactions attributed to compounds of similar chemical or biologic composition to agents used in the study.
See the full eligibility criteria
Who can join
- Patient - Cohort 1:
- High risk acute myeloid leukemia (AML) in either:
- Complete remission (CR) defined by \< 5% marrow blasts by morphology in the context of hematological recovery (ANC ≥ 0.5× 10\^9/L, platelet count ≥ 50 × 10\^9/L).
- Morphological leukemia free state (MLFS) defined by the absence of hematological recovery and \< 5% marrow blasts by morphology
- Patients must further meet one of the below for inclusion into the study:
- De novo AML in CR1 with any of the following high-risk features:
- MRD ≥ 1% after first induction course
- MRD ≥ 0.1% after second induction course
- RPN1-MECOM
- RUNX1-MECOM
- NPM1-MLF1
- DEK-NUP214
- KAT6A-CREBBP (if ≥ 90 days at diagnosis)
- FUS-ERG
- KMT2A-AFF1
- KMT2A-AFDN
- KMT2A-ABI1
- KMT2A-MLLT1
- 11p15 rearrangement (NUP98 - any partner gene)
- 12p13.2 rearrangement (ETV6 - any partner gene)
- Deletion 12p to include 12p13.2 (loss of ETV6)
- Monosomy 5/Del(5q) to include 5q31 (loss of EGR1)
- Monosomy 7
- 10p12.3 rearrangement (MLLT10b - any partner gene)
- FLT3/ITD with allelic ratio \> 0.1%, without bZIP CEBPA or NPM1
- RAM phenotype as evidenced by flow cytometry
- Other high-risk features not explicitly stated here, after discussion/approval with protocol PI.
- De novo AML in ≥ CR2
- Therapy-related AML in CR1
- AML evolving from myelodysplastic syndrome (MDS)
- One prior hematopoietic cell transplant is allowed, provided remission criteria as defined above are met. Patient - Cohort 2:
- High risk acute myeloid leukemia (AML) defined by either of the following:
- Treatment refractory disease: AML that is not in complete remission despite prior standard or salvage therapies.
- Multiply relapsed disease: AML that has relapsed after 2 or more hematopoietic cell transplantations.
- BM disease burden: Less than 25% bone marrow blasts by morphology must be present (M2 marrow), irrespective of peripheral hematological recovery. Patient - Both Cohorts:
- Less than or equal to 40 years of age.
- Lansky (\ 60%.
- Adequate organ function as defined below:
- Total bilirubin ≤ 3 x IULN for age
- AST(SGOT)/ALT(SGPT) ≤ 5 x IULN for age
- GFR ≥ 60 mL/min/1.73m2 as estimated by (1) updated Schwartz formula for ages 1-17 years or Cockcroft-Gault formula for ages ≥ 18 years, (2) 24-hour creatinine clearance, or (3) renal scintigraphy. If GFR is abnormal for...
- Renal function may also be estimated by serum creatinine based on age/gender. A serum creatinine \< 2 x IULN for age/gender is required for inclusion on this protocol.
- Adequate cardiac function, defined by left ventricular ejection fraction (LVEF) at rest ≥50% or shortening fraction (SF) ≥27% (via echocardiogram or MUGA).
- Adequate pulmonary function, defined by:
- FEV1, FVC, and DLCO ≥50% of predicted.
- O2 saturation ≥ 92% on room air by pulse oximetry and no supplemental O2 at rest for children \< 8 years of age or those unable to perform pulmonary function testing (PFT). For children unable to perform PFT, a...
- The effects of these treatments on the developing human fetus are unknown. For this reason, women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control...
- Ability to understand and willingness to sign an IRB approved written informed consent document, or patient has a guardian who has the ability to understand and willingness to sign an IRB approved written informed...
- Available familial haploidentical donor. The HCT donor must be available and willing to undergo 2 leukapheresis procedures: (I) one mobilized collection for the HPC graft and (II) one non-mobilized leukapheresis...
- Donor and recipient must be identical at a minimum of one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, HLA-DRB1, and HLA- DQB1. A minimum of 5/10 match is required and will be considered...
What rules you out
- Both Cohorts
- Active GvHD. If patient had prior GvHD, patient must be off immunosuppression for at least 3 months prior to starting study treatment.
- Active non-hematologic malignancy. History of other malignancy is acceptable as long as therapy has been completed and there is no current evidence of disease.
- Currently receiving any other investigational agents at the time of transplant.
- Active CNS or extramedullary disease. History of CNS or extramedullary disease currently in remission is acceptable.
- A history of allergic reactions attributed to compounds of similar chemical or biologic composition to agents used in the study.
- Inability to discontinue medications that are likely to interfere with ML NK cell activity, i.e., glucocorticoids and other immunosuppressants.
- Presence of significant anti-donor HLA antibodies per institutional standards. Anti-donor HLA - Antibody Testing is defined as a positive crossmatch test of any titer (by complement dependent cytotoxicity or flow...
- Presence of a second major disorder deemed a contraindication for HCT.
- Patients with Fanconi Anemia or Down Syndrome.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection (bacterial, viral with clinical instability, or fungal), symptomatic congestive heart failure, or unstable cardiac arrhythmia.
- Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 14 days of the start of conditioning. Donor Eligibility Criteria - Both Cohorts
- The preferred donor should be an adult aged 18 years or older. However, in circumstances where no suitable adult donor is available, consideration may be given to a minor donor aged 12 years or older. This exception...
- A medical condition that poses unacceptable risk, including autoimmune disease, infection, hematologic disorder, malignancy or a pathogenic germline mutation.
- Comorbidities that preclude safe administration of granulocyte colony-stimulating factor (G-CSF), placement of a pheresis catheter and/or stem cell collection.
- Served as donor in prior haploidentical HCT.
- Significant psychosocial or logistical barriers.
- Donor must be HLA haploidentical (≥ 5/10 and ≤ 9/10 allele match at the -A, -B, -C, DRB1 and DQ loci) by high resolution typing and related to the patient.
- Donor must meet the selection criteria as defined by the Foundation for the Accreditation of Hematopoietic Cell Therapy (FACT).
- Donor must be available and willing to undergo one mobilized and one non-mobilized leukapheresis procedure.
- Donor may not be pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 7 days prior to initiation of recipient's conditioning regimen, within 7 days of donor stem cell...
- Donor must be able to understand and willing to sign an IRB-approved written informed consent document.
The study team makes the final eligibility decision.
Where it's taking place
- St Louis, Missouri, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include St Louis, Missouri, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.