Human Placenta-derived 3D Mesenchymal Stem Cells(Guojianqingke) (3D MSC - QK01)
A Phase I/IIa Clinical Trial on the Safety, Tolerability, and Preliminary Efficacy of Human Placental-Derived 3D Mesenchymal Stem Cell Injection Administered Via the Intravenous Route in Patients With Acute Ischemic Stroke (AIS): A Randomized, Double-Blind, Placebo-Controlled Study
調査の概要
詳細な説明
This study consists of two sequential phases:
Phase I (Dose Escalation): A single ascending dose design to evaluate safety and tolerability of intravenous human placental-derived 3D MSC injection in AIS patients, and to establish the MTD and RP2D.
Phase IIa (Expansion): A randomized, double-blind, placebo-controlled evaluation of preliminary efficacy at the RP2D.
Study Duration: Screening within 72 hours of stroke onset; single-day treatment; follow-up visits through Day 720.
研究の種類
入学 (推定)
段階
- フェーズ2
- フェーズ 1
連絡先と場所
研究連絡先
- 名前:Zhao Dong
- 電話番号:18910685535
- メール:dong_zhaozhao@126.com
研究場所
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Beijing、中国
- 募集
- Chinese People's Liberation Army General Hospital, First Medical Center
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コンタクト:
- Zhao Dong
- 電話番号:18910685535
- メール:dong_zhaozhao@126.com
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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:
- Age ≥18 years and ≤80 years; any gender
- Body weight 45-90 kg
- Diagnosed with acute ischemic stroke, with onset between 6 and 72 hours (inclusive) prior to enrollment; received thrombolysis or not planned for thrombolysis and no planned thrombectomy
- NIHSS score 6-20, with NIHSS item 1a (Level of Consciousness) <2
- Participant or legally authorized representative able to understand and provide written informed consent
Exclusion Criteria:
- Significant pre-stroke disability (pre-stroke modified Rankin Scale [mRS] score ≥2);
- History of intracerebral hemorrhage, subarachnoid hemorrhage, or hemorrhagic transformation after this ischemic stroke (imaging re-evaluation before planned dosing shows new bleeding within the infarct area accompanied by neurological deterioration [e.g., NIHSS total score increased ≥4 points from admission], judged by the investigator as unsuitable for clinical trial participation); or presence of cerebrovascular malformation, multiple sclerosis, severe traumatic brain injury history, encephalitis, or other conditions causing stroke-like symptoms
- Uncontrolled systemic diseases, including but not limited to: hypertension (systolic BP >180 mmHg and/or diastolic BP ≥120 mmHg), diabetes (diabetic acute complications such as ketoacidosis, hyperosmolar hyperglycemic state, lactic acidosis, or hypoglycemic coma within 3 months, or difficult-to-control diabetes [blood glucose >16.8 mmol/L or <2.8 mmol/L]), renal disease (eGFR <30 mL/min/1.73m²), hepatic failure (Child-Pugh Class C), severe heart failure (NYHA Class IV), severe chronic respiratory disease
- History of seizure (except secondary epilepsy not currently requiring drug treatment)
- History of brain tumor or malignancy within the past 5 years, including concurrent second primary malignancy, except: a) radically excised non-melanoma skin cancer; b) radically treated cervical carcinoma in situ; c) radically treated papillary thyroid carcinoma; d) radically treated localized prostate cancer; e) radically treated ductal carcinoma in situ of the breast
History of any of the following:
- Active or uncontrolled autoimmune disease (e.g., antiphospholipid antibody syndrome)
- Protein C or protein S deficiency
- Sickle cell anemia
- Deep vein thrombosis
- Pulmonary embolism
- Cerebrovascular malformation (e.g., moyamoya disease)
- Any concomitant disease or physical condition (e.g., severe arthritis, amputation, blindness, severe disability from prior stroke) that, in the investigator's judgment, would significantly interfere with accurate assessment of mRS, NIHSS, or BI scores
- Major surgery within the past 30 days (e.g., thoracotomy, cardiac surgery, abdominal surgery, intracranial surgery)
Currently severe illness, including:
- Severe heart failure (NYHA Class III-IV)
- Severe febrile illness (any fever within 14 days before dosing requiring systemic anti-infective treatment)
- Primary or secondary immunodeficiency disease, or long-term or recent high-dose immunosuppressant or systemic corticosteroid therapy before screening
- Hemorrhagic disorder or bone marrow transplantation
- Any comorbidity that the investigator believes may shorten survival or limit ability to complete the study
- Uncontrolled depression affecting daily life before stroke, dementia that may affect clinical assessment, or other neurological or psychiatric disorders that the investigator believes may affect study assessment
- Uncontrolled active infection; or systemic anti-infective treatment within 7 days before dosing that the investigator assesses may shortly convert to uncontrolled active infection
Organ function meeting any of the following:
Hematology:
Absolute neutrophil count (ANC) <1.0×10⁹/L Platelets (PLT) <75×10⁹/L Hemoglobin (Hb) <80 g/L
Hepatic/Renal Function:
Alanine aminotransferase (ALT) >2.5×ULN Aspartate aminotransferase (AST) >2.5×ULN Total bilirubin (TBIL) >1.5×ULN Creatinine >1.5×ULN
Coagulation:
Phase I: Not receiving anticoagulant or antithrombotic therapy: PT and APTT >1.25×ULN, INR >1.4; Receiving anticoagulant or antithrombotic therapy: PT and APTT >1.5×ULN, INR >3.0 Phase IIa: Not receiving anticoagulant therapy: APTT >2.0×ULN or PT >2.0×ULN; Receiving anticoagulant therapy: judged by investigator to have severe bleeding risk
- Clinically significant uncorrected electrolyte disturbances (e.g., hyperkalemia, hypernatremia) that the investigator believes may affect study assessment
- Unable to undergo head CT/MRI for any reason (e.g., cardiac pacemaker, metal implants, claustrophobia)
- History of drug abuse or alcohol abuse within the past year
- Allergy to bovine or porcine products, human serum albumin products, or known allergy to gentamicin
- Participation in another investigational drug, device study, or stem cell/immune cell therapy within 3 months before treatment
- History of blood transfusion or vaccination with attenuated/live vaccine within 3 months before screening
- Pregnant or lactating women; or participants with pregnancy plans during the study period, or unwilling to use effective contraception; or females of childbearing potential with positive pregnancy test; or female participants on long-term oral contraceptives (continuous use >30 days)
- Other reasons deemed by the investigator as unsuitable for participation or inability to complete study procedures (e.g., lack of willingness)
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:ランダム化
- 介入モデル:並列代入
- マスキング:4倍
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:Human Placental-Derived 3D Mesenchymal Stem Cell Injection
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This product is administered by intravenous infusion. The investigator will calculate the dosage based on the dose group assigned to each participant. Preparation: Remove the product from liquid nitrogen and immediately thaw in a 37±1°C water bath. After thawing, add 40 mL of pre-cooled (2-8°C) compound electrolyte injection to the product bag and mix well. Administration: The recommended infusion time is 30 minutes (±5 minutes) per bag. |
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プラセボコンパレーター:Cell Culture Medium (Cell-Free)
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This product is administered by intravenous infusion. The investigator will calculate the dosage based on the dose group assigned to each participant. Preparation: Remove the product from liquid nitrogen and immediately thaw in a 37±1°C water bath. After thawing, add 40 mL of pre-cooled (2-8°C) compound electrolyte injection to the product bag and mix well. Administration: The recommended infusion time is 30 minutes (±5 minutes) per bag. |
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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Proportion of Participants with Modified Rankin Scale (mRS) Score of 0-2
時間枠:Day 90 post-treatment
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Functional outcome assessed by the Modified Rankin Scale (mRS), a validated clinician-reported 7-point ordinal scale (minimum = 0, maximum = 6). Higher scores indicate worse functional outcome (0 = no symptoms, 6 = death). A score of 0-2 indicates functional independence. Assessment conducted by a trained, blinded neurologist via structured interview using the standardized mRS questionnaire. % of participants with mRS score 0-2 |
Day 90 post-treatment
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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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Proportion of Participants with mRS Score 0-2 at Day 28
時間枠:Day 28 post-treatment
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Functional outcome assessed by the Modified Rankin Scale (mRS), a validated clinician-reported 7-point ordinal scale (minimum = 0, maximum = 6). Higher scores indicate worse functional outcome (0 = no symptoms, 6 = death). A score of 0-2 indicates functional independence. Assessment conducted by a trained, blinded neurologist via structured interview using the standardized mRS questionnaire. % of participants with mRS score 0-2 |
Day 28 post-treatment
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Proportion of Participants with mRS Score 0-2 at Day 180
時間枠:Day 180 post-treatment
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Functional outcome assessed by the Modified Rankin Scale (mRS), a validated clinician-reported 7-point ordinal scale (minimum = 0, maximum = 6). Higher scores indicate worse functional outcome (0 = no symptoms, 6 = death). A score of 0-2 indicates functional independence. Assessment conducted by a trained, blinded neurologist via structured interview using the standardized mRS questionnaire. % of participants with mRS score 0-2 |
Day 180 post-treatment
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Proportion of Participants with mRS Score 0-2 at Day 270
時間枠:Day 270 post-treatment
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Functional outcome assessed by the Modified Rankin Scale (mRS), a validated clinician-reported 7-point ordinal scale (minimum = 0, maximum = 6). Higher scores indicate worse functional outcome (0 = no symptoms, 6 = death). A score of 0-2 indicates functional independence. Assessment conducted by a trained, blinded neurologist via structured interview using the standardized mRS questionnaire. % of participants with mRS score 0-2 |
Day 270 post-treatment
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Proportion of Participants with mRS Score 0-2 at Day 360
時間枠:Day 360 post-treatment
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Functional outcome assessed by the Modified Rankin Scale (mRS), a validated clinician-reported 7-point ordinal scale (minimum = 0, maximum = 6). Higher scores indicate worse functional outcome (0 = no symptoms, 6 = death). A score of 0-2 indicates functional independence. Assessment conducted by a trained, blinded neurologist via structured interview using the standardized mRS questionnaire. % of participants with mRS score 0-2 |
Day 360 post-treatment
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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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Incidence of Dose-Limiting Toxicity (DLT)
時間枠:Throughout the dose-escalation phase (Phase I only)
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Dose-Limiting Toxicity assessed by clinical evaluation and laboratory monitoring according to predefined criteria based on NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. DLT is defined as: Grade ≥3 non-hematologic toxicity, Grade ≥4 hematologic toxicity lasting >7 days, or any toxicity requiring dose delay >14 days. Assessment includes: physical examination, vital signs monitoring, hematology panel, blood chemistry, and coagulation tests. % of participants with DLT events |
Throughout the dose-escalation phase (Phase I only)
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Incidence of Serious Adverse Events (SAE)
時間枠:From first dose through Day 720
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Serious Adverse Events assessed by clinical monitoring, laboratory evaluation, and radiographic imaging according to NCI-CTCAE version 5.0 and ICH E2A regulatory definitions. SAE includes: death, life-threatening event, hospitalization, disability/incapacity, congenital anomaly, or other medically important event. Assessment tools include: clinical examination, automated vital signs monitors, laboratory analyzers, and CT/MRI imaging. % of participants with SAEs |
From first dose through Day 720
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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)
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
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