Safety and Efficacy of CD123-targeted CAR-NK for Relapsed/Refractory Acute Myeloid Leukemia or Bl… (NCT06006403) | Clinical Trial Compass
CompletedPhase 1/2
Safety and Efficacy of CD123-targeted CAR-NK for Relapsed/Refractory Acute Myeloid Leukemia or Blastic Plasmacytoid Dendritic Cell Neoplasm
China5 participantsStarted 2024-03-28
Plain-language summary
This study is a single-arm, open-label, dose-escalating + dose-expansion clinical study, aiming to evaluate the safety and efficacy of targeting CD123 CAR-NK cell preparations in Relapsed/refractory acute myeloid leukemia (AML) or blastocytic plasmacytoid dendritic cell neoplasm (BPDCN). The pharmacokinetic characteristics of CAR-NK cell preparations for the treatment of patients with Relapsed/refractory acute myeloid leukemia or blastocytic plasmacytoid dendritic cell neoplasm were obtained and the recommended dose.
Who can participate
Age range
18 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Gender is not limited, age 18-75 years old (including the threshold value);
. The expression of CD123 in tumor cells was detected by flow cytometry.
. Patients with relapsed/refractory AML or BPDCN diagnosed with CD123 positive: 1) AML: a. Recurrent: After complete response (CR), the recurrence of leukemia cells in peripheral blood or bone marrow original cells ≥5% (except for other reasons such as bone marrow regeneration after consolidation chemotherapy) or the occurrence of extramedullary leukemia cell infiltration; b. Refractory: refers to those who have failed to receive 2 courses of treatment with standard protocols; Patients recurrence within 12 months after CR with consolidation and intensive treatment; Recurrence after 12 months but failed to respond to conventional chemotherapy; 2 or more relapses; Extramedullary leukemia persists;
. Cardiac function: echocardiography indicated cardiac ejection fraction ≥50%, and no obvious abnormality was found in electrocardiogram;
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial used CAR-NK cells targeting a protein called CD123 — can you tell me whether my leukemia cells actually express CD123, and whether that makes this kind of treatment potentially relevant to my situation?
2Since this was a Phase 1/2 trial, the main focus was still on figuring out safety and dosing — what do the results so far tell us about the side effects patients experienced, and how does that risk profile compare to other options I might have?
3The trial has already completed recruitment, so there are likely some results — have you seen any published or preliminary data on how well this CD123-targeted CAR-NK approach worked for patients with relapsed or refractory AML or BPDCN like mine?
4CAR-NK cell therapy is different from the more commonly known CAR-T therapy — can you explain what that difference means practically, including whether the side effect risks like cytokine release syndrome might be different?
5Given that this specific trial is closed, are there other CAR-NK or CAR-T trials targeting CD123 that are currently enrolling, or would standard salvage chemotherapy or a bone marrow transplant be a better first step to discuss for my case?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
To evaluate the safety of CAR-NK cell preparations in the treatment of Relapsed/Refractory Acute Myeloid Leukemia or Blastic Plasmacytoid Dendritic Cell Neoplasm[Safety and Tolerability]
Timeframe: 1 month
2
To evaluate the efficacy of CAR-NK cell preparations in the treatment of Relapsed/Refractory Acute Myeloid Leukemia or Blastic Plasmacytoid Dendritic Cell Neoplasm[Effectiveness]
. Total bilirubin and alkaline phosphatase ≤ 2.0×ULN (Gilbert syndrome ≤ 3.0×ULN);
. Blood oxygen saturation \> 92%.
Exclusion criteria
. Prior to screening, the following anti-tumor therapies were received: chemotherapy, targeted therapy, or other investigational drug treatment within 14 days or at least 5 half-lives (whichever is shorter), except in cases where disease progression has been confirmed after treatment;
. had a cerebrovascular accident or seizure within 6 months before signing the ICF;
. There is an active or uncontrolled infection that requires systemic treatment within 1 week prior to screening;
. suffering from any of the following heart diseases:
. New York Heart Association (NYHA) Stage III or IV congestive heart failure;
. Had myocardial infarction or coronary artery bypass grafting (CABG) within ≤6 months before enrollment;
. A history of clinically significant ventricular arrhythmia, or unexplained syncope (other than those caused by vasovagal or dehydration);