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- Klinische proef NCT07696481
Clinical Study on the Safety and Efficacy of PID23 Injection in Patients With Relapsed/Refractory Acute Leukemia (R/R AL)
A Single-center, Single-arm, Open-label Clinical Study on the Safety and Efficacy of PID23 Injection in Treating Patients With Relapsed/Refractory Acute Leukemia
Studie Overzicht
Toestand
Conditie
Interventie / Behandeling
Studietype
Inschrijving (Geschat)
Fase
- Vroege fase 1
Contacten en locaties
Studiecontact
- Naam: Yuhua Li, PhD
- Telefoonnummer: +86-020-61643188
- E-mail: liyuhua2011gz@163.com
Studie Locaties
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Guangdong
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Guangzhou, Guangdong, China, 510282
- Department of Hematology, Zhujiang Hospital, Southern Medical University
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Contact:
- Yuhua Li, PhD
- Telefoonnummer: +86-020-62782316
- E-mail: liyuhua2011gz@163.com
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Contact:
- E-mail: zhoulijanice@foxmail.com
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Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
- Kind
- Volwassen
- Oudere volwassene
Accepteert gezonde vrijwilligers
Beschrijving
Inclusion Criteria:
- 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.
4.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.
9. 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.
Exclusion Criteria:
- 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.
5.Grade 2-4 acute graft-versus-host disease (GVHD) or moderate-to-severe chronic GVHD within 4 weeks prior to screening.
6.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).
9.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.
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: NVT
- Interventioneel model: Opdracht voor een enkele groep
- Masker: Geen (open label)
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
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Experimenteel: PID23 Injection (Dose Escalation)
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.
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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
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Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
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Incidence of Dose-Limiting Toxicities (DLTs)
Tijdsspanne: Up to 21 days post-PID23 infusion
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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.
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Up to 21 days post-PID23 infusion
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Incidence and Severity of Treatment-Emergent Adverse Events
Tijdsspanne: Up to 2 years post-PID23 infusion
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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.
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Up to 2 years post-PID23 infusion
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Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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Objective Response Rate at 3 Months
Tijdsspanne: At 3 months post-infusion
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Proportion of participants achieving complete remission (CR) or CR with incomplete blood count recovery (CRi) at 3 months post-infusion.
CR defined as bone marrow blasts <5%, no peripheral blasts, ANC ≥1.0×10⁹/L, platelets ≥100×10⁹/L, no extramedullary disease.
CRi defined as meeting all CR criteria except platelet <100×10⁹/L and/or ANC <1.0×10⁹/L.
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At 3 months post-infusion
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Duration of Remission (DOR)
Tijdsspanne: Up to 2 years post-infusion
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Time from first documented CR or CRi to first documented disease progression (PD) or death from any cause, whichever occurs first.
Participants who do not achieve CR/CRi are not applicable for this analysis.
Assessed per protocol until 2 years post-infusion.
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Up to 2 years post-infusion
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Progression-Free Survival (PFS)
Tijdsspanne: Up to 2 years post-infusion
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Time from PID23 infusion to first documented disease progression (PD) or death from any cause, whichever occurs first.
Participants alive without progression are censored at the date of last disease assessment.
Assessed per protocol through 2 years post-infusion or until event.
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Up to 2 years post-infusion
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Overall Survival (OS)
Tijdsspanne: Up to 2 years post-infusion
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Time from PID23 infusion to death from any cause.
Participants alive at the end of follow-up are censored at the date of last known survival status.
Assessed through 2 years post-infusion or until death, whichever occurs first.
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Up to 2 years post-infusion
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Change from Baseline in Bone Marrow Blast Percentage
Tijdsspanne: Baseline through 2 years post-infusion
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Change from baseline in percentage of bone marrow blasts assessed by bone marrow morphology.
Measured at screening and at protocol-specified follow-up time points (M1, M2, M3, and every 3 months thereafter).
Assessed per protocol schedule.
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Baseline through 2 years post-infusion
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Maximum Concentration (Cmax) of CAR-T Cells in Peripheral Blood
Tijdsspanne: Day 0 through 2 years post-infusion
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Maximum concentration (Cmax) of CAR-T cells in peripheral blood, measured as CAR DNA copy number by quantitative PCR (copies/μg DNA) and/or percentage of CAR+ cells by flow cytometry.
Blood samples collected at protocol-specified time points: 0h, 30min, 2h, 6h, 12h, D2, D4, D7, D10, D14, D21, M1, M2, M3, and long-term follow-up.
Samples analyzed by the technology partner.
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Day 0 through 2 years post-infusion
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Time to Maximum Concentration (Tmax) of CAR-T Cells
Tijdsspanne: Day 0 through 2 years post-infusion
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Time to reach maximum concentration (Tmax) of CAR-T cells in peripheral blood, measured as CAR DNA copy number by quantitative PCR.
Blood samples collected at protocol-specified time points from immediately post-infusion through long-term follow-up.
Tmax determined from the concentration-time profile.
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Day 0 through 2 years post-infusion
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Area Under the Curve (AUC0-28d) of CAR-T Cells
Tijdsspanne: Day 0 through Day 28
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Area under the concentration-time curve from Day 0 to Day 28 (AUC0-28d) of CAR-T cells in peripheral blood, measured as CAR DNA copy number by quantitative PCR.
Calculated using the linear trapezoidal rule.
Blood samples collected at protocol-specified time points through Day 28.
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Day 0 through Day 28
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Change from Baseline in Serum Cytokine Levels
Tijdsspanne: Baseline through Month 1
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Change from baseline in serum cytokine concentrations, including but not limited to IL-6, IL-1β, IL-2/IL-2R, IL-8, IL-10, TNF-α, IFN-γ, CRP, and ferritin.
Blood samples collected at protocol-specified time points: screening, D0 (pre-infusion), D2, D4, D7, D10, D14, and M1.
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Baseline through Month 1
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Andere uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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Viral Clearance from Body Fluids
Tijdsspanne: Day 0 through Month 1 or until two consecutive negatives, whichever came first
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Detection of viral vector sequences in body fluid samples including blood, saliva, urine, and stool.
Viral clearance is defined as two consecutive negative results.
Samples collected at protocol-specified time points: 0h, 30min, 2h, 6h, 12h, D2, D4, D7, D10, D14, D21, and M1.
Samples may be stored at -80°C and shipped in batches to the technology partner for analysis.
Viral clearance assessed until two consecutive negative results are obtained.
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Day 0 through Month 1 or until two consecutive negatives, whichever came first
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Medewerkers en onderzoekers
Sponsor
Medewerkers
Studie record data
Bestudeer belangrijke data
Studie start (Geschat)
Primaire voltooiing (Geschat)
Studie voltooiing (Geschat)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Werkelijk)
Updates van studierecords
Laatste update geplaatst (Werkelijk)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Trefwoorden
Aanvullende relevante MeSH-voorwaarden
- Pathologische processen
- Neoplasmata
- Ziekte attributen
- Ziekten van het immuunsysteem
- Neoplasmata per histologisch type
- Hematologische ziekten
- Lymfatische ziekten
- Lymfoproliferatieve aandoeningen
- Immunoproliferatieve aandoeningen
- Leukemie, myeloïde
- Leukemie, Lymfoïde
- Leukemie
- Pathologische aandoeningen, tekenen en symptomen
- Hemische en lymfatische ziekten
- Herhaling
- Leukemie, myeloïde, acuut
- Voorlopercel lymfoblastische leukemie-lymfoom
Andere studie-ID-nummers
- 2026-KY-163-01
Plan Individuele Deelnemersgegevens (IPD)
Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?
Beschrijving IPD-plan
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
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