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

NCT07696481 | Not Yet Recruiting Early Phase 1

• 1 other identifier: 2026-KY-163-01
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

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).

Conditions

Relapsed or Refractory Acute Leukemia; Relapsed or Refractory Acute Lymphoblastic Leukemia; Relapsed or Refractory Acute Myeloid Leukemia (AML)

Keywords

PID23; In vivo CAR-T; Acute Leukemia; Relapsed; Refractory; AML; B-ALL; Acute Myeloid Leukemia; Acute lymphoblastic leukemia

Outcome Measures

Primary Outcomes (2)

• Incidence of Dose-Limiting Toxicities (DLTs)

  Number of participants experiencing DLTs during the DLT observation period. DLT is defined as any treatment-emergent adverse event meeting protocol-specified severity criteria assessed per CTCAE v6.0 and ASTCT criteria for CRS/ICANS.

  Up to 21 days post-PID23 infusion

• Incidence and Severity of Treatment-Emergent Adverse Events

  Number and percentage of participants experiencing adverse events (AEs), graded per CTCAE v6.0. Includes all AEs, serious AEs (SAEs), and AEs of special interest (CRS, ICANS, HLH/MAS). Causality assessment performed by the investigator.

  Up to 2 years post-PID23 infusion

Secondary Outcomes (9)

• Objective Response Rate at 3 Months

  At 3 months post-infusion

• Duration of Remission (DOR)

  Up to 2 years post-infusion

• Progression-Free Survival (PFS)

  Up to 2 years post-infusion

• Overall Survival (OS)

  Up to 2 years post-infusion

• Change from Baseline in Bone Marrow Blast Percentage

  Baseline through 2 years post-infusion

Other Outcomes (1)

• Viral Clearance from Body Fluids

  Day 0 through Month 1 or until two consecutive negatives, whichever came first

Study Arms (1)

PID23 Injection (Dose Escalation)

EXPERIMENTAL

Participants receive a single intravenous infusion of PID23 Injection at one of three dose levels (0.8×10⁹, 2×10⁹, or 4×10⁹ TU) according to a rapid titration + standard 3+3 dose-escalation design. The starting dose is 0.8×10⁹ TU, with escalation to higher dose levels guided by safety and pharmacokinetic assessments. All subjects are hospitalized for observation for at least 3 weeks post-infusion and followed for up to 2 years.

Biological: PID23 Injection

Interventions

PID23 Injection BIOLOGICAL

PID23 is an in vivo CAR-T product, a lentiviral vector encoding CD19, BCMA, and CD70 chimeric antigen receptors, administered as a single intravenous infusion

PID23 Injection (Dose Escalation)

Eligibility Criteria

• Age: 3 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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.

Sponsors & Collaborators

• Zhujiang Hospital: lead
• Chongqing Precision Biotech Co., Ltd: collaborator

Study Sites (1)

Department of Hematology, Zhujiang Hospital, Southern Medical University

Guangzhou, Guangdong, 510282, China

Location

MeSH Terms

Conditions

Recurrence; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Leukemia, Myeloid

Central Study Contacts

Yuhua Li, PhD

CONTACT

+86-020-61643188; liyuhua2011gz@163.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Single-arm, open-label, dose-escalation study using rapid titration + standard 3+3 design at 3 dose levels (0.8, 2, 4 ×10⁹ TU). Each level enrolls 1 sentinel subject; after 21 days (DL1) or 14 days (DL2/3) observation, PK and safety data guide decision: escalate to next dose if no DLT, or switch to 3+3 if DLT observed. Under 3+3: 0/3 DLT → escalate; 1/3 → expand to 6; ≥2/3 or ≥2/6 → stop escalation. MTD defined as highest dose with ≤1/6 DLT, but optimal dose determined by integrating safety, efficacy, and PK/PD, not solely MTD. If CAR-T expansion is suboptimal (Cmax \<1/10 of prior mean \~10,000 copies/μg) at day 21 with no DLT and no blast reduction, a second identical dose may be considered after investigator evaluation.

Study Record Dates

• First Submitted: July 6, 2026
• First Posted: July 10, 2026
• Study Start: August 1, 2026
• Primary Completion (Estimated): May 30, 2029
• Study Completion (Estimated): March 31, 2030
• Last Updated: July 10, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)