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Dual-Targeted Umbilical Cord Blood CAR-T Cells Against CD19 and CD22 for the Treatment of Relapsed or Refractory Acute B-Cell Lymphoblastic Leukemia

24 september 2026 uppdaterad av: Jin Wang, Ruijin Hospital

A Single-Center Phase I Clinical Trial of Dual-Targeted Umbilical Cord Blood CAR-T Cells Against CD19 and CD22 for the Treatment of Relapsed or Refractory Acute B-Cell Lymphoblastic Leukemia

This investigator-initiated, prospective, single-arm, phase I dose-escalation trial aims to evaluate the safety and tolerability of anti-CD19/CD22 dual-target cord blood-derived CAR-T cells in adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia who have failed prior therapies, with the goal of reducing antigen-escape relapse and overcoming poor autologous T-cell fitness through the use of universally available umbilical cord blood T cells.

Studieöversikt

Detaljerad beskrivning

Patients with relapsed/refractory B-cell acute lymphoblastic leukemia who meet eligibility criteria will receive fludarabine (30 mg/m²/d) and cyclophosphamide (300 mg/m²/d) for three consecutive days as lymphodepleting conditioning, followed by a single intravenous infusion of anti-CD19/CD22 dual-target cord blood-derived CAR-T cells. Dose assignment follows a "3+3" escalation design across three dose levels (4×10⁶, 8×10⁶, and 12×10⁶ CAR-T/kg, ±20%). Dose-limiting toxicity will be assessed during the first 14 days post-infusion to determine the maximum tolerated dose. After CAR-T infusion, patients will be monitored for safety and efficacy at protocol-specified time points through 3 months, with long-term follow-up continuing up to 15 years post-infusion. A total of 18 patients will be enrolled.

Studietyp

Interventionell

Inskrivning (Beräknad)

18

Fas

  • Fas 1

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

Studieorter

    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, Kina, 200025
        • Rekrytering
        • Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
        • Kontakt:

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

18 år till 75 år (Vuxen, Äldre vuxen)

Tar emot friska volontärer

Nej

Kön som är behöriga för studier

Allt

Beskrivning

Inclusion Criteria:

  • The patient or their legally authorized guardian has signed the informed consent form (ICF), indicating understanding of the purpose and procedures of this clinical trial and willingness to participate.

  • Age between 18 and 75 years, male or female.

  • Diagnosis of relapsed/refractory B-cell acute lymphoblastic leukemia (R/R B-ALL) according to the Chinese Guidelines for Diagnosis and Treatment of Adult Acute Lymphoblastic Leukemia (2024 edition).

  • Leukemic cells confirmed to express CD19 and/or CD22 by flow cytometry.

  • ECOG performance status 0-2.

  • Life expectancy ≥12 weeks.

  • Adequate organ function at screening, meeting all of the following laboratory criteria:

    1. Hematology: absolute neutrophil count (ANC) ≥1×10⁹/L; absolute lymphocyte count (ALC) ≥0.3×10⁹/L; platelet count ≥20×10⁹/L; hemoglobin ≥60 g/L.
    2. Hepatic function: ALT and AST ≤2.5× upper limit of normal (ULN); total bilirubin ≤1.5× ULN.
    3. Renal function: creatinine clearance (CrCl) ≥40 mL/min (by Cockcroft-Gault formula).
    4. Coagulation: fibrinogen ≥1.0 g/L; activated partial thromboplastin time (APTT) ≤1.5× ULN; prothrombin time (PT) ≤1.5× ULN.
    5. Oxygen saturation >91%.
    6. Left ventricular ejection fraction (LVEF) ≥50%.
  • The subject and their spouse agree to use effective contraceptive measures (excluding rhythm method) from ICF signing through 1 year after CAR-T cell infusion.

Exclusion Criteria:

  • Active graft-versus-host disease (GVHD) or autoimmune disease requiring long-term immunosuppressive therapy.
  • Use of therapeutic doses of corticosteroids (defined as prednisone or equivalent >20 mg/day) within 7 days prior to screening. Physiologic replacement, topical, and inhaled steroids are permitted.
  • Hypertension not controllable with medication.
  • Severe cardiac disease, including but not limited to: unstable angina, myocardial infarction (within 6 months prior to screening), congestive heart failure (NYHA class ≥III), or severe arrhythmia.
  • Unstable systemic disease as judged by the investigator, including but not limited to: severe hepatic, renal, or metabolic disease requiring medication.
  • Malignancy other than B-ALL within 5 years prior to screening, except for adequately treated carcinoma in situ of the cervix, basal cell or squamous cell skin cancer, localized prostate cancer after radical surgery, or ductal carcinoma in situ of the breast after radical surgery.
  • History of solid organ transplantation.
  • Planned surgery within 2 weeks after study treatment (subjects scheduled for local anesthesia surgery may participate).
  • Receipt of other interventional investigational drugs within 1 month prior to ICF signing.
  • Uncontrolled active infection.
  • Positive for HBsAg, or positive for HBcAb with detectable HBV DNA in peripheral blood; positive for HCV antibody with detectable HCV RNA; positive for HIV antibody; positive for CMV DNA; positive for syphilis serology.
  • Pregnant or breastfeeding women.
  • Psychiatric illness, consciousness disorder, or central nervous system disease.
  • Other conditions deemed unsuitable for enrollment by the investigator.

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: N/A
  • Interventionsmodell: Enskild gruppuppgift
  • Maskning: Ingen (Open Label)

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: Anti-CD19/CD22 Dual-Target Cord Blood CAR-T Cells

A single intravenous infusion of anti-CD19/CD22 dual-target umbilical cord blood-derived CAR-T cells administered on Day 0, following lymphodepleting chemotherapy with fludarabine (30 mg/m²/day) and cyclophosphamide (300 mg/m²/day) for three consecutive days. Dose escalation follows a 3+3 design across three dose levels (4.0×10⁶, 8.0×10⁶, and 12.0×10⁶ CAR-T/kg, ±20%). All subjects receive the same investigational product; no comparator arm is included.

Genetically modified umbilical cord blood-derived T cells expressing chimeric antigen receptors targeting both CD19 and CD22. Administered as a single intravenous infusion on Day 0 following lymphodepleting chemotherapy. Dose levels: 4.0×10⁶, 8.0×10⁶, and 12.0×10⁶ CAR-T/kg (±20%) according to 3+3 dose escalation design.

30 mg/m² intravenous infusion over 30 minutes once daily for 3 consecutive days as lymphodepleting preconditioning prior to CAR-T cell infusion.

300 mg/m² intravenous infusion over 30 minutes once daily for 3 consecutive days as lymphodepleting preconditioning prior to CAR-T cell infusion.

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Incidence and Severity of Dose-Limiting Toxicity (DLT)
Tidsram: Within 14 days after CAR-T cell infusion

DLT is defined as treatment-related adverse events occurring within 14 days post-infusion. Non-hematologic DLT: Grade ≥3 toxicity not reducible to ≤ Grade 1 within 72 hours. Hematologic DLT: Grade 4 toxicity (excluding lymphopenia) persisting >21 days, not attributable to underlying disease. Predefined exclusion criteria include tumor lysis syndrome, electrolyte disturbances, hypogammaglobulinemia, transient laboratory abnormalities, febrile neutropenia, and others per protocol. CRS/ICANS graded per ASTCT 2019, aGVHD per modified Glucksberg, other AEs per CTCAE v5.0.

Within 14 days after CAR-T cell infusion

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Objective Response Rate (ORR)
Tidsram: 1 and 3 months post-infusion

ORR is the proportion of subjects achieving CR/CRi at 1 and 3 months post-infusion. Efficacy assessed by bone marrow examination per international acute leukemia response criteria.

1 and 3 months post-infusion
Minimal Residual Disease (MRD) Negativity Rate
Tidsram: 1 and 3 months post-infusion

MRD negativity rate is the proportion of subjects achieving MRD-negative status by flow cytometry at 1 and 3 months post-infusion.

1 and 3 months post-infusion
Complete Remission (CR) Rate
Tidsram: 1 and 3 months post-infusion

CR rate is the proportion achieving CR. Efficacy assessed by bone marrow examination per international acute leukemia response criteria.

1 and 3 months post-infusion
Progression-Free Survival (PFS)
Tidsram: From infusion up to 24 months

PFS is defined as time from infusion to disease progression, relapse, or death. Median PFS and 12-/24-month PFS rates will be estimated by Kaplan-Meier method.

From infusion up to 24 months
Overall Survival (OS)
Tidsram: From infusion up to 24 months

OS is defined as time from infusion to death from any cause. Median OS and 12-/24-month OS rates will be estimated by Kaplan-Meier method.

From infusion up to 24 months
CAR-T Cell Expansion and Persistence
Tidsram: Up to 90 days post-infusion

CAR-T cell levels and vector copy number (VCN) in peripheral blood will be measured.

Up to 90 days post-infusion
CAR-T Cell Expansion and Persistence
Tidsram: Up to 90 days post-infusion

PK parameters: Cmax(Peak Plasma Concentration)

Up to 90 days post-infusion
CAR-T Cell Expansion and Persistence
Tidsram: Up to 90 days post-infusion

PK parameters: Tmax

Up to 90 days post-infusion
CAR-T Cell Expansion and Persistence
Tidsram: Up to 90 days post-infusion

PK parameters: AUC₀-₂₈d, AUC₀-₉₀d.

Up to 90 days post-infusion
CAR-T Cell Expansion and Persistence
Tidsram: At 3 and 6 months

Proportion of subjects with detectable CAR-T cells at 3 and 6 months, and duration of persistence will be recorded.

At 3 and 6 months

Samarbetspartners och utredare

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Sponsor

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

13 maj 2026

Primärt slutförande (Beräknad)

30 mars 2028

Avslutad studie (Beräknad)

30 mars 2028

Studieregistreringsdatum

Först inskickad

26 juni 2026

Först inskickad som uppfyllde QC-kriterierna

17 september 2026

Första postat (Faktisk)

24 september 2026

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

25 september 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

24 september 2026

Senast verifierad

1 september 2026

Mer information

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