Nebulized MSC-Exos for Anti-MDA5+ RP-ILD: Safety and Efficacy Trial
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)
Li Shiyue
10 participants
Apr 15, 2025
INTERVENTIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria3
- Patients are eligible for inclusion if they meet all of the following criteria:
- Positive for anti-MDA5 antibody dermatomyositis (according to the "Chinese Expert Consensus on the Diagnosis and Treatment of Anti-MDA5 Positive Dermatomyositis (2023 Edition)");
- Pulmonary lesions meet the diagnostic criteria for RP-ILD.
Exclusion Criteria14
- Patients who meet any of the following criteria will be excluded from this study:
- Pregnant or breastfeeding women, or women planning pregnancy during the study, or men unwilling to use contraceptive measures throughout the trial period;
- History of severe allergies or allergies to the main active ingredients of the trial medication;
- Currently suffering from severe pulmonary infections, pneumothorax, or large pleural effusions;
- Currently diagnosed with pulmonary embolism;
- Currently undergoing mechanical ventilation through tracheal intubation;
- Currently undergoing extracorporeal life support treatments such as ECMO, CRRT, PMX-DHP, or plasma exchange;
- Currently suffering from severe heart failure, liver, or kidney insufficiency;
- Expected to undergo lung transplantation in the near future;
- Currently suffering from lung cancer or pulmonary nodules suspected to be early-stage lung cancer;
- Suffering from primary immunodeficiency diseases;
- 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;
- 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;
- 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.
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Interventions
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.
Locations(1)
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NCT06919380