IASO207 Injection for Active Refractory Systemic Lupus Erythematosus (SLE)
An Exploratory Clinical Study on the Treatment of Active Refractory Systemic Lupus Erythematosus With IASO207 Injection
調査の概要
詳細な説明
This study is a single-arm, single-center, open-label, first-in-human (FIH) clinical trial designed to preliminarily evaluate the safety and efficacy of IASO207 injection, an in vivo chimeric antigen receptor T-cell (CAR-T) therapy, in patients with active, refractory systemic lupus erythematosus (SLE), and to determine the recommended dose for subsequent clinical development.
A standard "3+3" dose-escalation design will be employed during the dose-escalation phase, with three predefined dose levels: 5.0 × 10⁸ TU, 1.0 × 10⁹ TU, and 2.0 × 10⁹ TU. Each dose cohort will enroll 3-6 subjects, with a single administration of IASO207 injection.
The primary objective is to assess the safety and tolerability of IASO207 across different dose levels. Secondary and exploratory objectives include the characterization of pharmacokinetics, pharmacodynamics, and immunogenicity, as well as the preliminary evaluation of clinical efficacy in patients with active refractory SLE. The results of this study are expected to inform dose selection and support subsequent clinical trials.
研究の種類
入学 (推定)
段階
- 初期フェーズ 1
連絡先と場所
研究連絡先
- 名前:Xiangyu Zhao, M.D,Ph.D
- 電話番号:010-88325531
- メール:Zhao_xy@bjmu.edu.cn
研究連絡先のバックアップ
- 名前:Meng Lv, M.D., Ph.D
- 電話番号:010-88316617
- メール:drlvmeng@bjmu.edu.cn
研究場所
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-
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Beijing、中国、100044
- 募集
- Peking University People's Hospital
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副調査官:
- Meng Lv, M.D, Ph.D
-
コンタクト:
- Meng Lv, M.D,Ph.D
- 電話番号:010-88316617
- メール:drlvmeng@bjmu.edu.cn
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コンタクト:
- Jing He, M.D, Ph.D
- 電話番号:010-88316617
- メール:hejing1105@126.com
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主任研究者:
- Xiangyu Zhao, M.D, Ph.D
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主任研究者:
- Jing He, M.D, Ph.D
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-
参加基準
適格基準
就学可能な年齢
- 大人
- 高齢者
健康ボランティアの受け入れ
説明
Inclusion Criteria:
- Subjects aged 18-75 years old, regardless of gender.
- Subjects diagnosed with active refractory SLE: a) Confirmed by the 2019 European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR) classification criteria or the 2012 Systemic Lupus Erythematosus Collaborative Clinic (SLICC) criteria for at least 24 weeks; b) SLE Disease Activity Index 2000 (SLEDAI-2K) score ≥ 8, with at least 1 organ system having a BILAG-2004 A-class activity score at screening, or 2 organ systems having a BILAG-2004 B-class activity score; c) Previously treated with standardized glucocorticoids and at least two immunosuppressants/adjusters, antimalarial drugs or biologics for at least 3 months.
- Positive for disease-related pathogenic antibodies: Anti-nuclear antibody (ANA) positive and/or anti-dsDNA positive and/or anti-Smith positive.
- Allowed to use ≤ 20mg/d prednisone or equivalent dose of corticosteroids at screening, and use at a stable dose for at least 2 weeks. Note: Local or inhaled corticosteroids (or its immunomodulators) can be used concurrently;
- If antimalarial treatment has been initiated for ≥ 12 weeks before screening and is used at a stable dose for at least 8 weeks, it is allowed to continue in the study (maximum hydroxychloroquine dose ≤ 400mg/d).
- If immunosuppressants have been used before screening, they must be used at a stable dose for at least 4 weeks.
- Laboratory tests must meet the following conditions: a) Blood routine: Absolute neutrophil count (ANC) ≥ 1.0×10^9/L; Absolute lymphocyte count (ALC) ≥ 0.3×10^9/L; Hemoglobin ≥ 60 g/L; Platelets ≥ 50×10^9/L b) Liver function: Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5× upper limit of normal (ULN); Serum total bilirubin ≤ 1.5×ULN (Gilbert's syndrome ≤ 3.0×ULN).
- The subject and their spouse agree to take effective contraceptive measures (excluding safe period contraception) from the time of signing the informed consent form by the subject until one year after IASO207 injection treatment.
- The subject must agree to sign or personally write and present the signed informed consent form approved by the ethics committee before starting any screening procedure.
Exclusion Criteria:
Disease-related:
- Diagnosed with drug-induced SLE.
- Complicated with other autoimmune diseases that may affect the assessment of the study, including but not limited to Sjögren's syndrome, psoriasis, rheumatoid arthritis.
- Had a catastrophic antiphospholipid syndrome or developed a severe antiphospholipid syndrome within 1 year before screening.
The study disease involved neurological symptoms with a BILAG-2004 activity score of class A.
Other medical history-related:
- Known primary immunodeficiency (congenital or acquired).
- The subject has uncontrollable active fungal, viral, bacterial or other infections (existing persistent infection-related signs/symptoms, not improved after appropriate anti-infection treatment) or requires intravenous anti-infection drug treatment for infection.
- Positive hepatitis B surface antigen (HBsAg), or positive hepatitis B core antibody (HBcAb) and abnormal peripheral blood hepatitis B virus (HBV) DNA detection (defined as HBV DNA quantification above the normal reference range of the testing center or positive HBV DNA qualitative detection); positive hepatitis C virus (HCV) antibody and positive peripheral blood hepatitis C virus (HCV) RNA; positive Human Immunodeficiency Virus (HIV) antibody; positive cytomegalovirus (CMV) DNA detection; positive Treponema pallidum specific antibody and positive rapid plasma reagin test for syphilis.
- Severe heart disease: including but not limited to unstable angina pectoris and/or myocardial infarction within 12 months of screening, any congestive heart failure (NYHA classification ≥ III), and a history of severe arrhythmia; or left ventricular ejection fraction (LVEF) < 45%.
- Severe asthma or chronic obstructive pulmonary disease (COPD), with stable treatment considered for mild or moderate asthma or COPD by the investigator and sponsor; or resting arterial blood oxygen saturation < 91%.
- Evaluated by the investigator as having a severe bleeding risk.
- Evaluated by the investigator as having severe liver dysfunction.
- History of severe kidney disease; or eGFR < 30 mL/min/1.73m2 calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula.
- Acute cerebrovascular disease events occurred within 6 months before enrollment, including transient ischemic attack or stroke history.
- Any severe and/or uncontrolled comorbid diseases as assessed by the investigator.
- Had a malignant tumor within 5 years before screening, excluding cured cervical carcinoma in situ, basal cell or squamous cell skin cancer, locally advanced prostate cancer after radical surgery, breast duct carcinoma in situ after radical surgery, or papillary thyroid carcinoma after radical surgery.
- Had a clear history of mental disorder or a history of substance abuse for mental disorders that cannot be quit.
Known allergy to the components of IASO207 injection or to the supportive drugs required for the toxicity management of CAR-T cell therapy (such as tocilizumab).
Previous and concurrent treatments:
- History of organ transplantation.
- History of autologous or allogeneic stem cell transplantation.
- History of cell therapy or CD19-targeted drug treatment.
- Received targeted CD20 biologic therapy within 12 weeks before screening, such as rituximab, obinutuzumab.
- Received plasma exchange or immunoadsorption treatment within 12 weeks before screening.
- Had or planned to have major surgery or surgical treatment caused by any reason within 12 weeks before screening.
- Have participated in the treatment of other investigational drugs (except for placebo) within the previous 4 weeks or 5 half-lives (whichever is longer);
- Have received a BAFF antagonist, such as belimumab or tixagevimab, within the previous 4 weeks;
- Have received live attenuated vaccine within the previous 4 weeks;
- Pregnant or lactating women;
- Other situations deemed unsuitable for inclusion by the investigators.
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)
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
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実験的:IASO207 Injection
IASO207 Injection will be infused at 5.0×10^8 TU or 1.0×10^9 TU or 2.0×10^9 TU after enrollment
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IASO207 is a third-generation replication-deficient self-inactivating lentiviral vector that carries the gene encoding a second-generation anti-human CD19 chimeric antigen receptor (CD19 CAR) containing the 4-1BB co-stimulatory factor.
IASO207 has been engineered through surface modification of the lentiviral envelope to enable it to selectively bind and transduce T cells within the body, thereby directly generating CD19 CAR-T cells in vivo.
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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 |
|---|---|---|
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Safety endpoint - Adverse Events (AEs)
時間枠:up to 2 years from IASO207 Injection infusion
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Incidence and severity of adverse events as assessed by NCI-CTCAE v5.0 (except CRS and ICANS assessed according to the criteria of 2019 ASTCT criteria).
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up to 2 years from IASO207 Injection infusion
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Safety endpoint - The incidence of dose-limiting toxicity (DLT)
時間枠:28 days from IASO207 Injection infusion
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Percentage of participants who experienced DLT within 28 days after IASO207 administration
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28 days from IASO207 Injection infusion
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Pharmacokinetic Endpoint - Cmax (viral particle)
時間枠:Up to 7 days from IASO207 Injection infusion
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Maximum concentration (Cmax) of viral particle titer in peripheral blood will be observed and calculated.
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Up to 7 days from IASO207 Injection infusion
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Pharmacokinetic Endpoint - Cmax (VCN)
時間枠:up to 2 years from IASO207 Injection infusion
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The maximum concentration (Cmax) of lentiviral vector copy number (VCN) in peripheral blood after infusion.
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up to 2 years from IASO207 Injection infusion
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Pharmacodynamic Endpoint - Ferritin
時間枠:up to 2 years from IASO207 Injection infusion
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Changes in the levels of Ferritin.
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up to 2 years from IASO207 Injection infusion
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Pharmacodynamic Endpoint - IL-6
時間枠:up to 2 years from IASO207 Injection infusion
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Changes in the levels of IL-6.
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up to 2 years from IASO207 Injection infusion
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Efficacy endpoint - Response rate of SRI-4 after infusion
時間枠:up to 2 years from IASO207 Injection infusion
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SLE Responder Index (SRI)-4 is defined as follows with all criteria compared with Baseline: 1. ≥ 4-point reduction in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score.
2. No worsening of the overall condition (< 0.3 point increase in Physician's Global Assessment [PhGA]).
3.
No new British Isles Lupus Assessment Group (BILAG) A or more than 1 new BILAG B disease activity scores.
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up to 2 years from IASO207 Injection infusion
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Efficacy endpoint - lupus low disease activity state (LLDAS) rate through 2 years after infusion
時間枠:up to 2 years from IASO207 Injection infusion
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Proportions of subjects achieving LLDAS at each time point
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up to 2 years from IASO207 Injection infusion
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Efficacy endpoint - Definitions of Remission in SLE (DORIS) rate through 2 years after infusion
時間枠:up to 2 years from IASO207 Injection infusion
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Proportions of subjects achieving remission according to the DORIS as assessed by SLEDAI-2K Scale, PhGA score and concomitant medication use
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up to 2 years from IASO207 Injection infusion
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Efficacy endpoint - The changes in SLEDAI-2K scores
時間枠:up to 2 years from IASO207 Injection infusion
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At each visit site, the patients were scored using the SLEDAI-2K, and then the changes in scores compared between each visit point and the baseline score were evaluated
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up to 2 years from IASO207 Injection infusion
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Efficacy endpoint - The changes in PGA scores
時間枠:up to 2 years from IASO207 Injection infusion
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At each visit site, the patients were scored using the PGA, and then the changes in scores compared between each visit point and the baseline score were evaluated
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up to 2 years from IASO207 Injection infusion
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7. Pharmacokinetic Endpoint - Tmax (viral particle)
時間枠:Up to 7 days from IASO207 Injection infusion
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Peak time (Tmax) of viral particle titer in peripheral blood will be observed and analyzed.
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Up to 7 days from IASO207 Injection infusion
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Pharmacokinetic Endpoint - AUC0-7 (viral particle)
時間枠:Up to 7 days from IASO207 Injection infusion
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AUC0-7d refers to the area under the concentration-time curve from day 0 to day 7
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Up to 7 days from IASO207 Injection infusion
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Pharmacokinetic Endpoint - Cmax (CAR-T cells)
時間枠:up to 2 years from IASO207 Injection infusion
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The maximum concentration (Cmax) of CAR-T cells in peripheral blood after infusion
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up to 2 years from IASO207 Injection infusion
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Pharmacokinetic Endpoint - Tmax (CAR-T cells)
時間枠:up to 2 years from IASO207 Injection infusion
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The time for CAR-T cells to reach the maximum concentration (Tmax) after infusion
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up to 2 years from IASO207 Injection infusion
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Pharmacokinetic Endpoint - AUC (CAR-T cells)
時間枠:up to 2 years from IASO207 Injection infusion
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Area under the curve of 28 days, 90 days, 180 days, and the last time point of PK measurement (AUC0-28d, AUC0-90d, AUC0-180d, AUC0-last) for CAR-T cells.
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up to 2 years from IASO207 Injection infusion
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Pharmacokinetic Endpoint - Tmax (VCN)
時間枠:up to 2 years from IASO207 Injection infusion
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The time for VCN to reach the maximum concentration (Tmax) after infusion
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up to 2 years from IASO207 Injection infusion
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Pharmacokinetic Endpoint - AUC (VCN)
時間枠:up to 2 years from IASO207 Injection infusion
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Area under the curve of 28 days, 90 days, 180 days, and the last time point (AUC0-28d, AUC0-90d, AUC0-180d, AUC0-last) for VCN
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up to 2 years from IASO207 Injection infusion
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Pharmacodynamic Endpoint - Pathogenic antibodies
時間枠:up to 2 years from IASO207 Injection infusion
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Changes in serum levels of pathogenic antibodies such as ANA, anti-dsDNA, and anti-Smith.
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up to 2 years from IASO207 Injection infusion
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Pharmacodynamic Endpoint - Complement levels
時間枠:up to 2 years from IASO207 Injection infusion
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Changes in measures of C3, C4.
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up to 2 years from IASO207 Injection infusion
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Pharmacodynamic Endpoint - Immune cell subsets
時間枠:up to 2 years from IASO207 Injection infusion
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Changes in the levels of Immune cell subsets
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up to 2 years from IASO207 Injection infusion
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Pharmacodynamic Endpoint - Serum immunoglobulin
時間枠:up to 2 years from IASO207 Injection infusion
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Changes in serum immunoglobulin (IgG, IgA, IgM) levels from baseline.
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up to 2 years from IASO207 Injection infusion
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Pharmacodynamic Endpoint - CRP
時間枠:up to 2 years from IASO207 Injection infusion
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Changes in the levels of CRP
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up to 2 years from IASO207 Injection infusion
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その他の成果指標
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Immunogenicity
時間枠:up to 2 years from IASO207 Injection infusion
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Prevalence and titer of confirmed human anti-CAR antibodies in peripheral blood.
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up to 2 years from IASO207 Injection infusion
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Replication competent lentivirus (RCL)
時間枠:up to 2 years from IASO207 Injection infusion
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Prevalence of replication-competent lentivirus (RCL) in peripheral blood.
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up to 2 years from IASO207 Injection infusion
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Virus shedding
時間枠:Up to 7 days from IASO207 Injection infusion
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Presence of viral shedding in body fluid samples (urine, feces, and saliva)
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Up to 7 days from IASO207 Injection infusion
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協力者と研究者
捜査官
- 主任研究者:XiangYu Zhao, M.D, Ph.D、Peking University People's Hospital
- 主任研究者:Jing He, M.D, Ph.D、Peking University People's Hospital
研究記録日
主要日程の研究
研究開始 (推定)
一次修了 (推定)
研究の完了 (推定)
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
キーワード
追加の関連 MeSH 用語
その他の研究ID番号
- IASO207CI002
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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