A Phase 1 interventional study of MSC-exos Nebulization Therapy in Anti-MDA5 Positive Dermatomyositis-Associated RP-ILD and Rapidly Progressive Interstitial Lung Disease, sponsored by Li Shiyue. Recruiting at 1 site in China. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2025-04-10.
Sponsored by Li Shiyue · Phase 1, Interventional, and Treatment
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.
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.
171 studies on the registry are indexed under Dermatomyositis; 61 are open to participants now.
This study's planned enrollment of 10 is below the median of 31 across 125 interventional studies indexed under Dermatomyositis.
Browse Dermatomyositis studies →This is the only study on the registry with Li Shiyue as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Patients are eligible for inclusion if they meet all of the following criteria:
Exclusion Criteria:
Patients who meet any of the following criteria will be excluded from this study:
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.
Three-Month Mortality Rate and Safety of Nebulized MSC-exos P1
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.
Time frame: 3 months post-treatment initiation]
Oxygen Saturation
Oxygen Saturation: SpO2 (%) via pulse oximetry
Time frame: Baseline, day 14, and day 28 post-treatment initiation
CT Lesion Changes
CT Lesion Changes: Semi-quantitative scoring system (0-25)
Time frame: Baseline, day 14, and day 28 post-treatment initiation
Symptom Improvement
Symptom Improvement: VAS (0-10) for dyspnea and cough
Time frame: Baseline, day 14, and day 28 post-treatment initiation
C-reactive Protein (CRP)
Serum C-reactive Protein levels (CRP) (mg/L)
Time frame: Baseline, day 14, and day 28 post-treatment initiation
Interleukin-6 (IL-6)
Interleukin-6 (IL-6) (pg/mL)
Time frame: Baseline, day 14, and day 28 post-treatment initiation
D-dimer
Plasma D-dimer (μg/L)
Time frame: Baseline, day 14, and day 28 post-treatment initiation
Plan to share: No
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