小児の再発性/難治性急性骨髄性白血病を治療するためのNK520の安全性と有効性
再発/難治性急性骨髄性白血病の小児患者に対するNK520の安全性と有効性を評価するためのオープンな単一施設探索研究
This study will evaluate the safety and efficacy of NK520 in the treatment of pediatric relapsed/refractory acute myeloid leukemia. NK520 will be administered by intravenous injection. The safety and efficacy of this treatment will be evaluated.
調査の概要
詳細な説明
This open label, single-arm study aims to evaluate the efficacy and safety of allogenic natural killer (NK) cells in pediatric relapsed/refractory acute myeloid leukemia. Allogenic NK cells will be infused once a week. After infusion, the investigators will observe the characteristics of dose limited toxicity (DLT), and determine the maximum tolerable dose(MTD). To provide basis for the dosage and treatment plan of cell products in follow-up clinical trials.
研究の種類
入学 (実際)
段階
- 初期フェーズ 1
連絡先と場所
研究場所
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-
Shanghai Municipality
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Shanghai、Shanghai Municipality、中国、200127
- Shanghai Children Medical Center
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参加基準
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:
- Participants must be between 6 and 18 years;
- Diagnostic Criteria: Meet the 2022 World Health Organization (WHO) diagnostic criteria for acute myelocytic leukemia (AML), unsuitable for current treatments or patients with relapsed/refractory AML after ≥2 lines of therapy. The definition of relapsed/refractory acute myeloid leukemia is based on the 2017 Chinese Guidelines for Diagnosis and Treatment: a. Relapsed AML: Diagnosis is confirmed when leukemia cells reappear in the peripheral blood or bone marrow blast cells exceed 5% after complete remission (CR) (excluding reasons such as bone marrow regeneration post-consolidation chemotherapy) or there is extramedullary infiltration by leukemia cells; b. Refractory AML: Initial cases unresponsive after two cycles of standard regimen treatment; recurrence within 12 months after CR and consolidation therapy; recurrence beyond 12 months with ineffectiveness of conventional chemotherapy; those who have relapsed twice or more; or persistent extramedullary leukemia;
- For participants under 16 years old, Lansky performance status must be ≥50%; for participants aged 16 or older, Karnofsky performance status must be ≥50%;
- Expected survival of at least 12 weeks;
- Normal Organ Function.
Exclusion Criteria:
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Acute promyelocytic leukemia, chronic myeloid leukemia, acute mixed lineage leukemia, or known central nervous system leukemia;
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AML associated with congenital syndromes, such as Down syndrome, Fanconi anemia, Bloom syndrome, Kostmann syndrome, or congenital aplastic anemia;
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Severe bleeding tendency or coagulation disorders, or currently receiving thrombolytic therapy;
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Human immunodeficiency virus (HIV)-infected individuals, or individuals with known active syphilis infection;
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Receipt of live attenuated vaccines within 2 weeks before the first dose or planned during the study period;
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Participation in another clinical trial and receipt of investigational drug within 4 weeks prior to the first dose;
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Receipt of immune-modulatory drugs (including thymosin, interferons, except for local use to manage conditions like pleural or ascites fluid) within 2 weeks before the first dose;
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At screening, positive hepatitis B or C viral markers as follows:
- Hepatitis B surface antigen (HBsAg) positive with serum hepatitis B virus-deoxyribonucleic acid (HBV-DNA) level ≥1×10^3 copies/mL or above normal range;
- Positive for hepatitis C virus (HCV) antibodies;
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Any other condition or situation in which the investigator deems the patient unsuitable for participation in this study.
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：非ランダム化
- 介入モデル：順次割り当て
- マスキング：なし（オープンラベル）
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
実験的：Group A(low-dose group)
NK520: 5x10^7 NK/kg |
The number of NK520 cell infused for each dosing should be calculated base on the body weight of subject. NK520 will be administered through intravenous infusion.
他の名前：
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実験的：Group B(medium-dose group)
NK520: 1.5×10^8NK/kg |
The number of NK520 cell infused for each dosing should be calculated base on the body weight of subject. NK520 will be administered through intravenous infusion.
他の名前：
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実験的：Group C(high-dose group)
NK520: 3×10^8NK/kg |
The number of NK520 cell infused for each dosing should be calculated base on the body weight of subject. NK520 will be administered through intravenous infusion.
他の名前：
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この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Dose-Limiting Toxicity
時間枠：From the first infusion of NK520 to 4 weeks after last infusion of NK520
|
To evaluate the DLT during N520 treatment |
From the first infusion of NK520 to 4 weeks after last infusion of NK520
|
|
Complete Response Rate (CRR)
時間枠：from the date of first infusion of NK510 up to 104 weeks
|
Effectiveness Metrics |
from the date of first infusion of NK510 up to 104 weeks
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Overall response rate (ORR)
時間枠：from the date of first infusion of NK510 up to 104 weeks
|
Effectiveness Metrics |
from the date of first infusion of NK510 up to 104 weeks
|
|
Duration of Response (DOR)
時間枠：From the date of enrollment up to 104 weeks
|
Effectiveness Metrics |
From the date of enrollment up to 104 weeks
|
|
Event-Free Survival (EFS)
時間枠：From date of enrollment up to 104 weeks, or progression, or date of death, whichever came first.
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Effectiveness Metrics |
From date of enrollment up to 104 weeks, or progression, or date of death, whichever came first.
|
|
Overall Survival (OS)
時間枠：From date of enrollment up to 104 weeks, or date of death, whichever came first.
|
Effectiveness Metrics |
From date of enrollment up to 104 weeks, or date of death, whichever came first.
|
協力者と研究者
捜査官
- 主任研究者：wenting Hu、Shanghai children's medical center
研究記録日
主要日程の研究
研究開始 (実際)
一次修了 (実際)
研究の完了 (実際)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
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
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
- NK520-01
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
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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