Nebulized MSC-Exos for Anti-MDA5+ RP-ILD: Safety and Efficacy Trial

April 9, 2025 updated by: Li Shiyue

Safety and Efficacy Study of Nebulized Mesenchymal Stem Cell-Derived Exosomes (MSC-exos) for the Treatment of Anti-MDA5 Positive Dermatomyositis-Associated Rapidly Progressive Interstitial Lung Disease (RP-ILD)

Objective: To assess the safety, tolerability, and efficacy of nebulized MSC-exos-P1 in patients with anti-MDA5 positive dermatomyositis-associated rapidly progressive interstitial lung disease (RP-ILD).

Design: Prospective interventional trial with 10 eligible patients aged 18-75, meeting criteria for RP-ILD and anti-MDA5 positivity. Primary endpoint is safety and tolerability, measured by adverse events within 30 days post-treatment. Secondary endpoints are clinical improvements on days 14 and 28, including serological indicators and chest HRCT scores.

Exclusions: Pregnant/breastfeeding individuals, severe allergies, active pulmonary infections, pulmonary embolism, extracorporeal support treatments, and other specified conditions.

Treatment: Nebulized MSC-exos-P1 daily for 14 days, plus standard care of corticosteroids and immunosuppressants.

Monitoring: Regular vital signs, oxygenation index, and pulmonary function tests. Follow-ups at multiple points up to 12 months.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This single-center, prospective interventional trial aims to evaluate the safety profile and potential efficacy of nebulized mesenchymal stem cell-derived exosomes (MSC-exos-P1) in anti-MDA5 positive dermatomyositis patients with rapidly progressive interstitial lung disease. Anti-MDA5 positive RP-ILD represents a critical clinical challenge with mortality rates exceeding 50% despite aggressive immunosuppressive therapy, highlighting the urgent need for novel treatment approaches.

The trial will enroll 10 eligible patients who will receive a 14-day course of daily nebulized MSC-exos-P1 while continuing standard immunosuppressive therapy. Safety monitoring will include daily vital signs, laboratory tests, and adverse event documentation during the treatment period. Efficacy assessments will measure changes in oxygenation parameters, pulmonary function, inflammatory biomarkers, and CT imaging findings at days 14 and 28 compared to baseline.

The scientific rationale for this intervention is based on preclinical evidence demonstrating the immunomodulatory, anti-inflammatory, and anti-fibrotic properties of MSC-derived exosomes. These nanoparticles have shown the ability to modify alveolar macrophage phenotypes, reduce pro-inflammatory cytokine production, and suppress fibroblast activation - mechanisms that may directly target the pathophysiological processes driving RP-ILD in this patient population. The nebulized delivery system enables direct targeting of affected lung tissue while minimizing systemic exposure.

Study Type

Interventional

Enrollment (Estimated)

10

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
    • Guangdong
      • Guangzhou, Guangdong, China, 510150
        • Recruiting
        • The First Affiliated Hospital of Guangzhou Medical University
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Patients are eligible for inclusion if they meet all of the following criteria:

    1. Positive for anti-MDA5 antibody dermatomyositis (according to the "Chinese Expert Consensus on the Diagnosis and Treatment of Anti-MDA5 Positive Dermatomyositis (2023 Edition)");

    2. Pulmonary lesions meet the diagnostic criteria for RP-ILD.

      Exclusion Criteria:

  • Patients who meet any of the following criteria will be excluded from this study:

    1. Pregnant or breastfeeding women, or women planning pregnancy during the study, or men unwilling to use contraceptive measures throughout the trial period;
    2. History of severe allergies or allergies to the main active ingredients of the trial medication;
    3. Currently suffering from severe pulmonary infections, pneumothorax, or large pleural effusions;
    4. Currently diagnosed with pulmonary embolism;
    5. Currently undergoing mechanical ventilation through tracheal intubation;
    6. Currently undergoing extracorporeal life support treatments such as ECMO, CRRT, PMX-DHP, or plasma exchange;
    7. Currently suffering from severe heart failure, liver, or kidney insufficiency;
    8. Expected to undergo lung transplantation in the near future;
    9. Currently suffering from lung cancer or pulmonary nodules suspected to be early-stage lung cancer;
    10. Suffering from primary immunodeficiency diseases;
    11. Currently suffering from active infectious diseases, including but not limited to HIV positivity, active tuberculosis, etc., and deemed unsuitable for this trial by the researcher;
    12. Use of other trial medications within 28 days before starting treatment, which the researcher judges may interfere with the safety and efficacy assessment of this trial medication;
    13. Other situations deemed not in the best interest of the subject or unsuitable for participation in this study by the researcher, such as poor compliance.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Nebulized MSC-exos for Anti-MDA5+ RP-ILD Treatment
This arm of the study involves the administration of nebulized Mesenchymal Stem Cell-derived Exosomes (MSC-exos-P1) as an intervention for patients diagnosed with Anti-MDA5 Positive Dermatomyositis-Associated Rapidly Progressive Interstitial Lung Disease (RP-ILD).
Drug: MSC-exos Nebulization Therapy
The intervention in this study, "Nebulized MSC-exos for Anti-MDA5+ RP-ILD Treatment," is distinguished by its use of mesenchymal stem cell-derived exosomes (MSC-exos) for direct pulmonary delivery via nebulization. This targeted approach aims to modulate immune responses and reduce inflammation specific to lung diseases, offering a novel therapeutic strategy. This method stands out for its potential to provide a safer and more effective treatment for RP-ILD compared to traditional therapies.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Three-Month Mortality Rate and Safety of Nebulized MSC-exos P1
Time Frame: 3 months post-treatment initiation]
Mortality measured as the percentage of participants who died within three months post-treatment. Safety assessed by number and severity of adverse events using CTCAE v5.0.
3 months post-treatment initiation]

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Oxygen Saturation
Time Frame: Baseline, day 14, and day 28 post-treatment initiation
Oxygen Saturation: SpO2 (%) via pulse oximetry
Baseline, day 14, and day 28 post-treatment initiation
CT Lesion Changes
Time Frame: Baseline, day 14, and day 28 post-treatment initiation
CT Lesion Changes: Semi-quantitative scoring system (0-25)
Baseline, day 14, and day 28 post-treatment initiation
Symptom Improvement
Time Frame: Baseline, day 14, and day 28 post-treatment initiation
Symptom Improvement: VAS (0-10) for dyspnea and cough
Baseline, day 14, and day 28 post-treatment initiation
C-reactive Protein (CRP)
Time Frame: Baseline, day 14, and day 28 post-treatment initiation
Serum C-reactive Protein levels (CRP) (mg/L)
Baseline, day 14, and day 28 post-treatment initiation
Interleukin-6 (IL-6)
Time Frame: Baseline, day 14, and day 28 post-treatment initiation
Interleukin-6 (IL-6) (pg/mL)
Baseline, day 14, and day 28 post-treatment initiation
D-dimer
Time Frame: Baseline, day 14, and day 28 post-treatment initiation
Plasma D-dimer (μg/L)
Baseline, day 14, and day 28 post-treatment initiation

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Li Shiyue

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

April 15, 2025

Primary Completion (Estimated)

December 31, 2026

Study Completion (Estimated)

May 31, 2027

Study Registration Dates

First Submitted

February 13, 2025

First Submitted That Met QC Criteria

April 7, 2025

First Posted (Actual)

April 9, 2025

Study Record Updates

Last Update Posted (Actual)

April 10, 2025

Last Update Submitted That Met QC Criteria

April 9, 2025

Last Verified

April 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ES-2024 -156 -01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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