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Dobbelt-målrettede GD2/B7-H3 CAR-NK-celler til børn med tilbagefaldende eller refraktær neuroblastom (DUAL-NK-NB)

7. november 2026 opdateret af: Beijing Biotech

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

Rekruttering

Betingelser

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

Interventionel

Tilmelding (Anslået)

36

Fase

  • Fase 2
  • Fase 1

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Studiesteder

    • Guangdong
      • Shenzhen, Guangdong, Kina, 518036
        • Rekruttering
        • Peking University Shenzhen Hospital
        • Kontakt:

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

1 år til 21 år (Barn, Voksen)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

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

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Interventionel model: Enkelt gruppeopgave
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / 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:
  • Dual-target GD2/B7-H3 CAR-NK

Lymphodepleting chemotherapy administered before CAR-NK infusion according to the protocol-defined conditioning regimen.

Andre navne:
  • Influenza

Lymphodepleting chemotherapy administered before CAR-NK infusion according to the protocol-definedb conditioning regimen.

Andre navne:
  • Cy

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Forekomst af dosisbegrænsende toksiciteter (DLT'er) efter første CARNK-infusion ved brug af protokoldefinerede DLT-kriterier.
Tidsramme: 28 dage
28 dage
Incidence and severity of treatment-emergent adverse events
Tidsramme: 12 months

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

Resultatmål
Tidsramme
Progressionsfri overlevelse
Tidsramme: 12 måneder
12 måneder
Samlet overlevelse
Tidsramme: 24 måneder
24 måneder
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

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

2. marts 2026

Primær færdiggørelse (Anslået)

14. marts 2027

Studieafslutning (Anslået)

17. juni 2028

Datoer for studieregistrering

Først indsendt

25. marts 2026

Først indsendt, der opfyldte QC-kriterier

25. marts 2026

Først opslået (Faktiske)

30. marts 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

12. november 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

7. november 2026

Sidst verificeret

1. november 2026

Mere information

Begreber relateret til denne undersøgelse

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ingen

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

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