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.

Study Overview

• Status: Not yet recruiting
• Conditions: Interstitial Lung Disease (ILD)
• Intervention / Treatment: Biological: Human Umbilical Cord Mesenchymal Stem Cell-Derived Extracellular Vesicles (hUCMSC-EVs); Biological: Normal Saline Placebo

• Study Type: Interventional
• Enrollment (Estimated): 24
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Tong, M.D; Phone Number: 86-13764089607; Email: tong.lin@zs-hospital.sh.cn

Study Locations

• China
  • Shanghai, China
    • Zhongshan Hospital, Fudan University
    • Contact: Tong, M.D; Phone Number: 86-13764089607; Email: tong.lin@zs-hospital.sh.cn
    • Principal Investigator: Yuanlin Song, M.D

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Single

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Low-dose group	Biological: Human Umbilical Cord Mesenchymal Stem Cell-Derived Extracellular Vesicles (hUCMSC-EVs); hUCMSC-EVs , nebulized BID × 7d/cycle, 3 cycles; Biological: Normal Saline Placebo; Nebulized inhalation BID × 7d per cycle, 3 cycles
Experimental: High-dose group	Biological: Human Umbilical Cord Mesenchymal Stem Cell-Derived Extracellular Vesicles (hUCMSC-EVs); hUCMSC-EVs , nebulized BID × 7d/cycle, 3 cycles; Biological: Normal Saline Placebo; Nebulized inhalation BID × 7d per cycle, 3 cycles
Experimental: Moderate-dose group	Biological: Human Umbilical Cord Mesenchymal Stem Cell-Derived Extracellular Vesicles (hUCMSC-EVs); hUCMSC-EVs , nebulized BID × 7d/cycle, 3 cycles; Biological: Normal Saline Placebo; Nebulized inhalation BID × 7d per cycle, 3 cycles

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence and Severity of Adverse Events	From first dose to Week 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from Baseline in Forced Vital Capacity (FVC)	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.	From first dose to Week 24
Change from Baseline in Diffusing Capacity of the Lung for Carbon Monoxide (DLCO)	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.	From first dose to Week 24
Change from Baseline in 6-Minute Walk Test (6MWT) Distance		From first dose to Week 24
Change from Baseline in St. George's Respiratory Questionnaire (SGRQ) Total Score	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).	From first dose to Week 24

Collaborators and Investigators

• Sponsor: Shanghai Zhongshan Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): April 30, 2026
• Primary Completion (Estimated): April 30, 2027
• Study Completion (Estimated): September 30, 2028

Study Registration Dates

• First Submitted: April 17, 2026
• First Submitted That Met QC Criteria: May 1, 2026
• First Posted (Actual): May 6, 2026

Study Record Updates

• Last Update Posted (Actual): May 6, 2026
• Last Update Submitted That Met QC Criteria: May 1, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lung Diseases; Lung Diseases, Interstitial
• Other Study ID Numbers: ACE001-RI001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No