Dobbelt-målrettede GD2/B7-H3 CAR-NK-celler til børn med tilbagefaldende eller refraktær neuroblastom (DUAL-NK-NB)
En fase 1/fase 2, åben-label undersøgelse af biomarkørinformeret, allogen dobbelt-måls GD2/B7-H3 (CD276) CAR-NK-celler hos børn og unge voksne med recidiverende eller refraktær neuroblastom
This is Phase 1/Phase 2 study tests allogeneic dual-target GD2/B7-H3 (CD276) CAR-NK cells in children and young adults with relapsed or refractory neuroblastoma. After lymphodepletion, participants receive IV CAR-NK cells;Part A defines the RP2D and Part B estimates preliminary activity
Studieoversigt
Status
Status
Betingelser
Betingelser
Intervention / Behandling
Intervention / Behandling
Detaljeret beskrivelse
The investigational product in this example is a cord blood-derived allogeneic NK-cell therapy engineered to express a dual-target CAR recognizing GD2 and B7-H3, supported by IL-15 to improve short-term persistence and equipped with an inducible safety switch. The study is designed as a multicenter Phase 1/Phase 2 protocol: Part A uses a standard 3+3 dose-escalation approach across predefined dose levels, and Part B expands at the RP2D in a biomarker-characterized population. All participants undergo central review of tumor tissue or marrow for GD2 and B7-H3 expression before treatment. Patients receive protocol-defined lymphodepletion followed by CAR-NK infusion on Day 0, with optional additional infusions on Days 7 and 14 if there is no dose-limiting toxicity (DLT), uncontrolled cytokine release syndrome (CRS), or rapid progression. Formal disease assessment uses revised International Neuroblastoma Response Criteria (rINRC). Correlative studies assess CAR-NK expansion and persistence, cytokine kinetics, tumor-response associations with antigen density, and whether future development should remain dualtarget or shift toward a GD2-dominant or B7-H3-enriched strategy. Long-term follow-up for gene-modified cellular therapy is planned per local regulatory requirements.
Undersøgelsestype
Undersøgelsestype
Tilmelding (Anslået)
Tilmelding
Fase
Fase
- Fase 2
- Fase 1
Kontakter og lokationer
Studiekontakt
Studiekontakt
- Navn: Seni S Lu, Phd
- Telefonnummer: +86 13076790030
- E-mail: Seni-Lu@beijing-biotech.com
Studiesteder
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Guangdong
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Shenzhen, Guangdong, Kina, 518036
- Rekruttering
- Peking University Shenzhen Hospital
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Kontakt:
- Zhen J Peng, Phd
- Telefonnummer: +86 13076790039
- E-mail: Zhen-Peng@beijing-biotech.com
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Deltagelseskriterier
Berettigelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
Inclusion Criteria:
- Age 12 months to 21 years at consent/assent.
- Histologically confirmed neuroblastoma or ganglioneuroblastoma with relapsed, refractory, progressive, or persistent high-risk disease for which no curative standard option is available.
- Measurable or evaluable disease according to revised International Neuroblastoma Response Criteria (rINRC), including MIBG-avid disease, CT/MRI-evaluable soft-tissue disease, and/or bone marrow disease.
- Tumor material available for central assessment of GD2 and B7-H3 expression; at least one target must be positive.
GD2 is treated as the core target and B7-H3 as the complementary target for correlative target-prioritization analyses.
- Prior exposure to standard neuroblastoma therapy, including anti-GD2-based therapy, unless contraindicated, unavailable, or declined for a documented medical reason.
- Lansky or Karnofsky performance score >= 50.
- Life expectancy >= 8 weeks.
- Recovery from clinically significant acute toxicities of prior therapy and protocol-defined washout from chemotherapy, biologics, radiation, and prior cell therapy.
- Adequate organ function: hematologic, renal, hepatic, cardiac, and pulmonary function considered sufficient by protocoldefined laboratory and clinical thresholds.
- Negative pregnancy test for patients of childbearing potential and agreement to use effective contraception during the protocol-defined period.
- Written informed consent from parent/legal guardian and assent from the participant when appropriate.
Exclusion Criteria:
- Active uncontrolled infection, including bacteremia, uncontrolled viral infection, or invasive fungal disease.
- Pregnancy or breastfeeding.
- Active grade >= 2 graft-versus-host disease, or systemic immunosuppression for treatment/prevention of graft-versushost disease within the protocol-defined washout period after prior allogeneic transplant.
- Symptomatic or unstable central nervous system disease requiring urgent medical intervention.
- Prior genetically modified cellular therapy within the protocol-defined washout window, or unresolved clinically significant toxicity from prior cell therapy.
- Active autoimmune disease requiring systemic immunosuppressive therapy.
- Clinically significant uncontrolled cardiovascular, pulmonary, hepatic, renal, or neurologic disorder that would increase study risk.
- Known hypersensitivity to fludarabine, cyclophosphamide, study-product components, or supportive-care medications required by the protocol.
- Known uncontrolled HIV infection or uncontrolled hepatitis B or C.
- Any medical, psychosocial, or logistical condition that, in the investigator's judgment, would make study participation unsafe or would impair protocol adherence.
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Antal våben
Våben og indgreb
Deltagergruppe / ArmDeltagergruppe / Arm |
Intervention / BehandlingIntervention / Behandling |
|---|---|
|
Eksperimentel: EB-DTNB-NK
Participants receive protocol-defined fludarabine/cyclophosphamide lymphodepletion followed by intravenous allogeneic dual-target GD2/B7-H3 CAR-NK cells on Day 0 at the assigned dose level or RP2D. Additional doses on Days 7 and 14 are permitted if predefined safety criteria are met and there is no prohibitive toxicity or rapid progression. |
Allogeneic, cord blood-derived NK cells engineered with a dual-target CAR recognizing GD2 and B7-H3 (CD276), with IL-15 support and an inducible safety switch; administered intravenously on Day 0 with optional repeat dosing on Days 7 and 14 if tolerated.
Andre navne:
Lymphodepleting chemotherapy administered before CAR-NK infusion according to the protocol-defined conditioning regimen.
Andre navne:
Lymphodepleting chemotherapy administered before CAR-NK infusion according to the protocol-definedb conditioning regimen.
Andre navne:
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Hvad måler undersøgelsen?
Primære resultatmål
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Forekomst af dosisbegrænsende toksiciteter (DLT'er) efter første CARNK-infusion ved brug af protokoldefinerede DLT-kriterier.
Tidsramme: 28 dage
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28 dage
|
|
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Incidence and severity of treatment-emergent adverse events
Tidsramme: 12 months
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Incidence and severity of treatment-emergent adverse events, including CRS and ICANS, graded using CTCAE v5.0 and ASTCT consensus criteria. |
12 months
|
Sekundære resultatmål
Sekundære resultatmål
Resultatmål |
Tidsramme |
|---|---|
|
Progressionsfri overlevelse
Tidsramme: 12 måneder
|
12 måneder
|
|
Samlet overlevelse
Tidsramme: 24 måneder
|
24 måneder
|
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Objektiv responsrate efter reviderede International Neuroblastoma Response Criteria (rINRC).
Tidsramme: 12 måneder
|
12 måneder
|
|
Varighed af respons blandt respondenter
Tidsramme: 24 måneder
|
24 måneder
|
Samarbejdspartnere og efterforskere
Sponsor
Sponsor
Datoer for undersøgelser
Studer store datoer
Studiestart (Faktiske)
Studiestart
Primær færdiggørelse (Anslået)
Primær færdiggørelse
Studieafslutning (Anslået)
Studieafslutning
Datoer for studieregistrering
Først indsendt
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Først opslået
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering sendt
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Nøgleord
Yderligere relevante MeSH-vilkår
- Patologiske processer
- Neoplasmer
- Sygdomsegenskaber
- Neoplasmer efter histologisk type
- Neoplasmer, kirtel og epitel
- Neoplasmer, Neuroepithelial
- Neuroektodermale tumorer
- Neoplasmer, kimceller og embryonale
- Neoplasmer, nervevæv
- Neuroektodermale tumorer, primitive, perifere
- Neuroektodermale tumorer, primitive
- Patologiske tilstande, tegn og symptomer
- Tilbagevenden
- Neuroblastom
- Ganglioneuroblastom
- Organiske kemikalier
- Kulbrinter
- Fosforamid -sennep
- Nitrogen sennepsforbindelser
- Sennepsforbindelser
- Kulbrinter, halogeneret
- Phosphoramider
- Organophosphorforbindelser
- Biologiske produkter
- Komplekse blandinger
- Vacciner
- Virale vacciner
- Cyclofosfamid
- fludarabin
- Influenza -vacciner
Andre undersøgelses-id-numre
Andre undersøgelses-id-numre
- EB-CARNK-CRC-103
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Studerer et amerikansk FDA-reguleret enhedsprodukt
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