Safety and Preliminary Efficacy of hUCMSC-EVs in Interstitial Lung Disease

NCT07570836 | Not Yet Recruiting Phase 1

• 1 other identifier: ACE001-RI001
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Interstitial Lung Disease (ILD)

Outcome Measures

Primary Outcomes (1)

• Incidence and Severity of Adverse Events

  From first dose to Week 12

Secondary Outcomes (4)

• Change from Baseline in Forced Vital Capacity (FVC)

  From first dose to Week 24

• Change from Baseline in Diffusing Capacity of the Lung for Carbon Monoxide (DLCO)

  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

  From first dose to Week 24

Study Arms (3)

Low-dose group

EXPERIMENTAL

Biological: Human Umbilical Cord Mesenchymal Stem Cell-Derived Extracellular Vesicles (hUCMSC-EVs); Biological: Normal Saline Placebo

Moderate-dose group

EXPERIMENTAL

Biological: Human Umbilical Cord Mesenchymal Stem Cell-Derived Extracellular Vesicles (hUCMSC-EVs); Biological: Normal Saline Placebo

High-dose group

EXPERIMENTAL

Biological: Human Umbilical Cord Mesenchymal Stem Cell-Derived Extracellular Vesicles (hUCMSC-EVs); Biological: Normal Saline Placebo

Interventions

Human Umbilical Cord Mesenchymal Stem Cell-Derived Extracellular Vesicles (hUCMSC-EVs) BIOLOGICAL

hUCMSC-EVs , nebulized BID × 7d/cycle, 3 cycles

High-dose group; Low-dose group; Moderate-dose group

Normal Saline Placebo BIOLOGICAL

Nebulized inhalation BID × 7d per cycle, 3 cycles

High-dose group; Low-dose group; Moderate-dose group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Shanghai Zhongshan Hospital: lead

Study Sites (1)

Zhongshan Hospital, Fudan University

Shanghai, China

Location

MeSH Terms

Conditions

Lung Diseases, Interstitial

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases

Central Study Contacts

Tong, M.D

CONTACT

86-13764089607; tong.lin@zs-hospital.sh.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: The study is designed as a dose-escalation trial comprising three sequential dose cohorts: low-dose, medium-dose, and high-dose. Escalation will proceed from the lowest to the highest dose level. Within each cohort, subjects will be randomized in a 3:1 ratio to receive either investigational product or placebo.

Study Record Dates

• First Submitted: April 17, 2026
• First Posted: May 6, 2026
• Study Start: April 30, 2026
• Primary Completion (Estimated): April 30, 2027
• Study Completion (Estimated): September 30, 2028
• Last Updated: May 6, 2026

Record last verified: 2026-05

Locations

CN (1)