Nebulized MSC-exosomes for treating severe lung disease in dermatomyositis patients

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)

This study is testing if a new inhaled treatment made from stem cell particles can help improve lung function and safety for patients with severe lung disease related to dermatomyositis.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: Guangzhou Medical University (Academic / other)
• Locations: 1 site (Guangzhou, Guangdong)
• Trial ID: NCT06919380 on ClinicalTrials.gov

What this trial studies

This interventional trial aims to evaluate the safety and efficacy of nebulized mesenchymal stem cell-derived exosomes (MSC-exos-P1) in patients with anti-MDA5 positive dermatomyositis-associated rapidly progressive interstitial lung disease (RP-ILD). A total of 10 eligible patients aged 18-75 will receive daily nebulized treatment for 14 days while continuing standard immunosuppressive therapy. The primary focus is on monitoring safety and tolerability, with secondary endpoints assessing clinical improvements through various pulmonary function tests and imaging. The study seeks to address a critical clinical challenge, as current treatments have limited effectiveness and high mortality rates.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-75 who are positive for anti-MDA5 antibodies and have been diagnosed with rapidly progressive interstitial lung disease.

Not a fit: Patients with severe allergies, active pulmonary infections, or those requiring mechanical ventilation will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve lung function and survival rates in patients with a severe form of interstitial lung disease.

How similar studies have performed: While the use of MSC-derived exosomes is a novel approach in this specific context, preclinical evidence suggests potential efficacy, though clinical success in similar studies remains to be fully established.

Eligibility criteria

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.

Where this trial is running

Guangzhou, Guangdong

• The First Affiliated Hospital of Guangzhou Medical University — Guangzhou, Guangdong, China (Recruiting)

Study contacts

• Study coordinator: li

Conditions Anti-MDA5 Positive Dermatomyositis-Associated RP-ILD; Rapidly Progressive Interstitial Lung Disease; Interstitial Lung Disease; Anti-MDA5 Positive