Safety and Preliminary Efficacy of hUCMSC-EVs in Interstitial Lung Disease
A Clinical Study to Explore the Safety and Preliminary Efficacy of Human Umbilical Cord Mesenchymal Stem Cell-Derived Extracellular Vesicles in the Treatment of Interstitial Lung Disease
This is an exploratory, randomized, single-blind, placebo-controlled, multiple-dose, dose-escalation clinical study. The study aims to evaluate the safety and preliminary efficacy of nebulized inhalation of Human Umbilical Cord Mesenchymal Stem Cell-Derived Extracellular Vesicles (hUCMSC-EVs) in patients with interstitial lung disease (ILD).
This study is designed to include three sequential dose cohorts-low, medium, and high-with dose escalation proceeding in an ascending order. Eligible subjects will be enrolled and randomized sequentially, with a planned enrollment of 8 subjects per cohort. Within each cohort, subjects will be randomly assigned in a 3:1 ratio to receive either hUCMSC-EVs or saline placebo.
調査の概要
状態
条件
研究の種類
入学 (推定)
段階
- フェーズ 1
連絡先と場所
研究連絡先
- 名前:Tong, M.D
- 電話番号:86-13764089607
- メール:tong.lin@zs-hospital.sh.cn
研究場所
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Shanghai、中国
- Zhongshan hospital, Fudan university
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コンタクト:
- Tong, M.D
- 電話番号:86-13764089607
- メール:tong.lin@zs-hospital.sh.cn
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主任研究者:
- Yuanlin Song, M.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
Key Inclusion Criteria:
- Age 18 years and older, regardless of gender.
- Diagnosed with interstitial lung disease (ILD).
- High-resolution computed tomography (HRCT) findings within 3 months prior to randomization must be consistent with interstitial pulmonary imaging changes.
- Pulmonary function test within the protocol-defined acceptable range prior to randomization.
- Good compliance, able to understand and cooperate with the required examinations and procedures, and willing to attend follow-up visits as scheduled.
- Voluntary participation and signing of written informed consent form.
Key Exclusion Criteria:
- Inability to tolerate nebulized inhalation therapy.
- Requirement for oxygen supplementation > 15 hours per day during the screening period.
- Allergic diathesis or a history of life-threatening drug hypersensitivity.
- History of malignancy within 5 years.
- Active hepatitis B, hepatitis C, or HIV infection.
- Active systemic infection requiring antibiotic therapy.
- Clinically significant laboratory abnormalities at screening.
- Concomitant significant or severe respiratory disease.
- History of psychiatric illness, epilepsy, or other central nervous system disorders.
- Concomitant severe cardiovascular, hepatic, or renal systemic disease.
- Pregnancy, planned pregnancy in the near future, or breastfeeding.
- Unwillingness to use effective contraception during the study period.
- Any condition that, in the opinion of the investigator, may increase the risk to the participant or interfere with the study outcomes.
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:順次割り当て
- マスキング:独身
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:低用量群
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hUCMSC-EVs , nebulized BID × 7d/cycle, 3 cycles
Nebulized inhalation BID × 7d per cycle, 3 cycles
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実験的:高用量群
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hUCMSC-EVs , nebulized BID × 7d/cycle, 3 cycles
Nebulized inhalation BID × 7d per cycle, 3 cycles
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実験的:Moderate-dose group
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hUCMSC-EVs , nebulized BID × 7d/cycle, 3 cycles
Nebulized inhalation BID × 7d per cycle, 3 cycles
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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 |
時間枠 |
|---|---|
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Incidence and Severity of Adverse Events
時間枠:From first dose to Week 12
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From first dose to Week 12
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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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Change from Baseline in Forced Vital Capacity (FVC)
時間枠:From first dose to Week 24
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Forced Vital Capacity (FVC) will be assessed using standardized spirometry in accordance with American Thoracic Society (ATS)/European Respiratory Society (ERS) guidelines.
Measurements will be performed at Baseline (prior to first dose) and at scheduled post-baseline visits.
Spirometry will be conducted using a calibrated spirometer.
Results will be summarized as absolute change from baseline and percent predicted normal values.
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From first dose to Week 24
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Change from Baseline in Diffusing Capacity of the Lung for Carbon Monoxide (DLCO)
時間枠:From first dose to Week 24
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Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) will be assessed using the single-breath method in accordance with ATS/ERS guidelines.
Hemoglobin-corrected DLCO values will be recorded if applicable.
Measurements will be performed at Baseline and at scheduled post-baseline visits.
Results will be summarized as absolute change from baseline and percent predicted normal values.
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From first dose to Week 24
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Change from Baseline in 6-Minute Walk Test (6MWT) Distance
時間枠:From first dose to Week 24
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From first dose to Week 24
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Change from Baseline in St. George's Respiratory Questionnaire (SGRQ) Total Score
時間枠:From first dose to Week 24
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The St. George's Respiratory Questionnaire (SGRQ) is a disease-specific instrument designed to measure health-related quality of life in patients with chronic respiratory diseases.
The Total Score is calculated as a weighted sum of the domain scores and ranges from 0 to 100, with higher scores indicating worse health-related quality of life.
A change of 4 units is generally considered the minimal clinically important difference (MCID).
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From first dose to Week 24
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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 (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
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