Clinical Study on the Safety and Efficacy of PID23 Injection in Patients With Relapsed/Refractory Acute Leukemia

Starting soon · Early Phase 1

Conditions studied: Relapsed or Refractory Acute Leukemia, Relapsed or Refractory Acute Lymphoblastic Leukemia, Relapsed or Refractory Acute Myeloid Leukemia (AML)

In brief

Relapsed or refractory acute leukemia (R/R AL) is a life-threatening blood cancer with poor outcomes and limited treatment options. PID23 Injection is an innovative in vivo CAR-T therapy that delivers a viral vector encoding three targets (CD19, BCMA, and CD70) directly into patients, enabling their own T cells to generate functional CAR-T cells against leukemia cells. This single-center, single-arm, open-label, dose-escalation study (3 dose levels: 0.8×10⁹, 2×10⁹, and 4×10⁹ TU) plans to enroll 3-18 patients with R/R AL aged 3-75 years, ECOG 0-2, and positive for at least one target. The primary objective is to evaluate safety, tolerability, and determine the recommended dose. Secondary objectives include preliminary efficacy (remission, survival), pharmacokinetics (CAR-T expansion), pharmacodynamics (cytokine changes), and exploratory viral clearance. After a single intravenous infusion, patients are hospitalized for ≥3 weeks, followed by monthly visits for 3 months, then every 3 months for up to 2 years. Enrollment is from May 2026 to May 2027, with follow-up through May 2029. The study is conducted at Zhujiang Hospital of Southern Medical University (PI: Prof. Li Yuhua).

Key facts

Study ID: NCT07696481
Run by: Zhujiang Hospital
People needed: 18
Starts: 2026-08-01
Expected to finish: 2030-03-31
Last updated by the study team: 2026-07-10

Who can join

Age: 3 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Patient or legal guardian voluntarily signs the informed consent form (ICF), demonstrating understanding of the study purpose and procedures, and willingness to participate.
Age 3 to 75 years, inclusive, male or female.
Diagnosis of relapsed or refractory acute leukemia per guideline criteria:
Relapsed: reappearance of leukemic cells in peripheral blood or bone marrow blasts ≥5% after achieving complete remission (CR); Refractory: failure to achieve CR after 2 courses of standard induction chemotherapy; relapse within 12 months after consolidation/intensification therapy; relapse after 12 months with no response to conventional chemotherapy; 2 or more relapses; extramedullary leukemia relapse or persistence; relapse after allogeneic hematopoietic stem cell transplantation.
Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2. 5.Life expectancy ≥12 weeks. 6.Bone marrow morphology showing ≥5% primitive/immature lymphocytes (blasts). 7.Tumor cells positive for CD19, BCMA, or CD70 expression by flow cytometry. 8.Adequate major organ function, defined as:
Cardiac: left ventricular ejection fraction (LVEF) ≥40% by echocardiogram;
Renal: serum creatinine ≤2.0× upper limit of normal (ULN), or creatinine clearance ≥50 mL/min (Cockcroft-Gault formula);
Hepatic: ALT and AST ≤3.0×ULN (≤5.0×ULN if with liver involvement); total bilirubin ≤2.0×ULN (≤3.0×ULN for Gilbert's syndrome);
Pulmonary: oxygen saturation ≥92% on room air;
Hematologic: absolute neutrophil count (ANC) ≥1.0×10⁹/L, platelets ≥50×10⁹/L, hemoglobin ≥80 g/L (with bone marrow involvement, ANC ≥0.5×10⁹/L, platelets ≥20×10⁹/L permitted) - assessments allowed after transfusion or hematopoietic growth factor support.
For women of childbearing potential, negative serum pregnancy test; all participants agree to use reliable (non-rhythm) contraceptive methods from ICF signing through 1 year post-PID23 infusion.

You may not qualify if…

Prior treatment with CAR-T or other genetically modified cell therapies, unless the investigator determines that safety risks have been adequately excluded.
Received the following anti-tumor therapies prior to PID23 infusion: Chemotherapy or molecular targeted therapy within 14 days or 5 half-lives (whichever is longer) (excluding conditioning chemotherapy and intrathecal chemotherapy; intrathecal therapy must be stopped ≥1 week prior to PID23 infusion); Radiotherapy to non-hematopoietic sites within 7 days; Radiotherapy to hematopoietic sites within 14 days.
Any of the following cardiac conditions:
(1) New York Heart Association (NYHA) Class III or IV congestive heart failure; (2) Myocardial infarction or coronary artery bypass grafting (CABG) within 6 months prior to enrollment; (3) Clinically significant ventricular arrhythmia, or unexplained syncope (excluding vasovagal or dehydration-related); (3) History of severe non-ischemic cardiomyopathy. 4.Active or uncontrolled infection requiring systemic therapy within 1 week prior to screening.
Grade 2-4 acute graft-versus-host disease (GVHD) or moderate-to-severe chronic GVHD within 4 weeks prior to screening.
Cerebrovascular accident or seizure within 6 months prior to screening. 7.Deep vein or arterial thrombosis event within 6 months prior to screening. 8.Active malignancy other than acute leukemia (excluding: inactive disease with treatment completed >2 years; adequately treated cervical carcinoma in situ, basal/squamous cell skin carcinoma, localized prostate cancer post-curative surgery, ductal carcinoma in situ post-curative surgery).
Received (attenuated) live vaccine within 4 weeks prior to screening. 10.Any other condition that, in the investigator's judgment, makes the patient unsuitable for participation in this study.

Where it is running

Department of Hematology, Zhujiang Hospital, Southern Medical University — Guangzhou, Guangdong, China

Full record on ClinicalTrials.gov

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