- ICH GCP
- Rejestr badań klinicznych w USA
- Badanie kliniczne NCT07838714
Dual-Targeted Umbilical Cord Blood CAR-T Cells Against CD19 and CD22 for the Treatment of Relapsed or Refractory Acute B-Cell Lymphoblastic Leukemia
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.
Przegląd badań
Status
Interwencja / Leczenie
Szczegółowy opis
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.
Typ studiów
Zapisy (Szacowany)
Faza
- Faza 1
Kontakty i lokalizacje
Kontakt w sprawie studiów
- Nazwa: Weiyang Liu
- Numer telefonu: +86 13817506393
- E-mail: lwy21758760@sjtu.edu.cn
Lokalizacje studiów
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Shanghai Municipality
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Shanghai, Shanghai Municipality, Chiny, 200025
- Rekrutacyjny
- Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
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Kontakt:
- Jin Wang, PhD
- Numer telefonu: +86 13524945202
- E-mail: jinwang@shsmu.edu.cn
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Kryteria uczestnictwa
Kryteria kwalifikacji
Wiek uprawniający do nauki
Akceptuje zdrowych ochotników
Płeć kwalifikująca się do nauki
Opis
Inclusion Criteria:
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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.
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Age between 18 and 75 years, male or female.
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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).
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Leukemic cells confirmed to express CD19 and/or CD22 by flow cytometry.
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ECOG performance status 0-2.
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Life expectancy ≥12 weeks.
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Adequate organ function at screening, meeting all of the following laboratory criteria:
- 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.
- Hepatic function: ALT and AST ≤2.5× upper limit of normal (ULN); total bilirubin ≤1.5× ULN.
- Renal function: creatinine clearance (CrCl) ≥40 mL/min (by Cockcroft-Gault formula).
- Coagulation: fibrinogen ≥1.0 g/L; activated partial thromboplastin time (APTT) ≤1.5× ULN; prothrombin time (PT) ≤1.5× ULN.
- Oxygen saturation >91%.
- Left ventricular ejection fraction (LVEF) ≥50%.
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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.
Plan studiów
Jak projektuje się badanie?
Szczegóły projektu
- Główny cel: Leczenie
- Przydział: Nie dotyczy
- Model interwencyjny: Zadanie dla jednej grupy
- Maskowanie: Brak (otwarta etykieta)
Broń i interwencje
Grupa uczestników / Arm |
Interwencja / Leczenie |
|---|---|
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Eksperymentalny: 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. |
Co mierzy badanie?
Podstawowe miary wyniku
Miara wyniku |
Opis środka |
Ramy czasowe |
|---|---|---|
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Incidence and Severity of Dose-Limiting Toxicity (DLT)
Ramy czasowe: Within 14 days after CAR-T cell infusion
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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
|
Miary wyników drugorzędnych
Miara wyniku |
Opis środka |
Ramy czasowe |
|---|---|---|
|
Objective Response Rate (ORR)
Ramy czasowe: 1 and 3 months post-infusion
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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
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Minimal Residual Disease (MRD) Negativity Rate
Ramy czasowe: 1 and 3 months post-infusion
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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
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Complete Remission (CR) Rate
Ramy czasowe: 1 and 3 months post-infusion
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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
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Progression-Free Survival (PFS)
Ramy czasowe: From infusion up to 24 months
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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
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Overall Survival (OS)
Ramy czasowe: From infusion up to 24 months
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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
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CAR-T Cell Expansion and Persistence
Ramy czasowe: Up to 90 days post-infusion
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CAR-T cell levels and vector copy number (VCN) in peripheral blood will be measured. |
Up to 90 days post-infusion
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CAR-T Cell Expansion and Persistence
Ramy czasowe: Up to 90 days post-infusion
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PK parameters: Cmax(Peak Plasma Concentration) |
Up to 90 days post-infusion
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CAR-T Cell Expansion and Persistence
Ramy czasowe: Up to 90 days post-infusion
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PK parameters: Tmax |
Up to 90 days post-infusion
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CAR-T Cell Expansion and Persistence
Ramy czasowe: Up to 90 days post-infusion
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PK parameters: AUC₀-₂₈d, AUC₀-₉₀d. |
Up to 90 days post-infusion
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CAR-T Cell Expansion and Persistence
Ramy czasowe: At 3 and 6 months
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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
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Współpracownicy i badacze
Sponsor
Daty zapisu na studia
Główne daty studiów
Rozpoczęcie studiów (Rzeczywisty)
Zakończenie podstawowe (Szacowany)
Ukończenie studiów (Szacowany)
Daty rejestracji na studia
Pierwszy przesłany
Pierwszy przesłany, który spełnia kryteria kontroli jakości
Pierwszy wysłany (Rzeczywisty)
Aktualizacje rekordów badań
Ostatnia wysłana aktualizacja (Rzeczywisty)
Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości
Ostatnia weryfikacja
Więcej informacji
Terminy związane z tym badaniem
Słowa kluczowe
Dodatkowe istotne warunki MeSH
- Nowotwory
- Choroby układu odpornościowego
- Infekcje
- Choroby wirusowe
- Nowotwory według typu histologicznego
- Infekcje wirusami DNA
- Choroby limfatyczne
- Zaburzenia limfoproliferacyjne
- Zaburzenia immunoproliferacyjne
- Chłoniak nieziarniczy
- Chłoniak z komórek B
- Chłoniak
- Zakażenia wirusem Epsteina-Barra
- Infekcje Herpesviridae
- Zakażenia wirusem nowotworowym
- Choroby hemowe i limfatyczne
- Chłoniak Burkitta
- Organiczne chemikalia
- Węglowodory
- Mostki fosforamidu
- Związki musztardy azotu
- Związki musztardy
- Węglowodory, uboczne
- Fosforamidy
- Związki okorosfosforowe
- Cyklofosfamid
- Fludarabina
Inne numery identyfikacyjne badania
- RJ-ALL 2026-B01
Informacje o lekach i urządzeniach, dokumenty badawcze
Bada produkt leczniczy regulowany przez amerykańską FDA
Bada produkt urządzenia regulowany przez amerykańską FDA
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