小児再発または難治性神経芽腫のためのデュアルターゲットGD2/B7-H3 CAR-NK細胞 (DUAL-NK-NB)
再発または難治性神経芽腫を有する小児および若年成人におけるバイオマーカー情報に基づく同種異系二重標的GD2/B7-H3(CD276)CAR-NK細胞の第1相/第2相、非盲検試験
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
調査の概要
状態
状態
条件
条件
介入・治療
介入・治療
詳細な説明
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.
研究の種類
研究の種類
入学 (推定)
入学
段階
段階
- フェーズ2
- フェーズ 1
連絡先と場所
研究連絡先
研究連絡先
- 名前:Seni S Lu, Phd
- 電話番号:+86 13076790030
- メール:Seni-Lu@beijing-biotech.com
研究場所
-
-
Guangdong
-
Shenzhen、Guangdong、中国、518036
- 募集
- Peking University Shenzhen Hospital
-
コンタクト:
- Zhen J Peng, Phd
- 電話番号:+86 13076790039
- メール:Zhen-Peng@beijing-biotech.com
-
-
参加基準
適格基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
説明
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.
研究計画
研究はどのように設計されていますか?
デザインの詳細
- 主な目的:処理
- 割り当て:なし
- 介入モデル:単一グループの割り当て
- マスキング:なし(オープンラベル)
アーム数
武器と介入
参加者グループ / アーム参加者グループ / アーム |
介入・治療介入・治療 |
|---|---|
|
実験的: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.
他の名前:
Lymphodepleting chemotherapy administered before CAR-NK infusion according to the protocol-defined conditioning regimen.
他の名前:
Lymphodepleting chemotherapy administered before CAR-NK infusion according to the protocol-definedb conditioning regimen.
他の名前:
|
この研究は何を測定していますか?
主要な結果の測定
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
初回CARNK投与後の用量制限毒性(DLT)の発生率(プロトコルで定義されたDLT基準を使用)。
時間枠:28日
|
28日
|
|
|
Incidence and severity of treatment-emergent adverse events
時間枠: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
|
二次結果の測定
二次結果の測定
結果測定 |
時間枠 |
|---|---|
|
無増悪生存
時間枠:12ヶ月
|
12ヶ月
|
|
全生存
時間枠:24ヶ月
|
24ヶ月
|
|
改訂国際神経芽腫反応基準(rINRC)による客観的奏効率。
時間枠:12ヶ月
|
12ヶ月
|
|
応答者の間での応答期間
時間枠:24ヶ月
|
24ヶ月
|
協力者と研究者
研究記録日
主要日程の研究
研究開始 (実際)
研究開始
一次修了 (推定)
一次修了
研究の完了 (推定)
研究の完了
試験登録日
最初に提出
最初に提出
QC基準を満たした最初の提出物
QC基準を満たした最初の提出物
最初の投稿 (実際)
最初の投稿
学習記録の更新
投稿された最後の更新 (実際)
投稿された最後の更新
QC基準を満たした最後の更新が送信されました
QC基準を満たした最後の更新が送信されました
最終確認日
最終確認日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
その他の研究ID番号
- EB-CARNK-CRC-103
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
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