NCT06919380

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

Recruiting now Phase 1 Last updated 10 April 2025

What this trial tests

Phase 1 trial testing MSC-exos Nebulization Therapy in Anti-MDA5 Positive Dermatomyositis-Associated RP-ILD in 10 participants. Currently enrolling.

Timeline

15 April 2025

Primary endpoint
31 December 2026

31 May 2027

Quick facts

Lead sponsor	Li Shiyue
Phase	Phase 1
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	10
Start date	15 April 2025
Primary completion	31 December 2026
Estimated completion	31 May 2027
Sites	1 location across China

Drugs / interventions tested

• MSC-exos Nebulization Therapy — full drug profile →

Conditions studied

• Anti-MDA5 Positive Dermatomyositis-Associated RP-ILD — all drugs for Anti-MDA5 Positive Dermatomyositis-Associated RP-ILD →
• Rapidly Progressive Interstitial Lung Disease — all drugs for Rapidly Progressive Interstitial Lung Disease →

Sponsor

Li Shiyue — full company profile →

Who can join

Adults 18 to 75, any sex, with Anti-MDA5 Positive Dermatomyositis-Associated RP-ILD or Rapidly Progressive Interstitial Lung Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

1. Rethinking miRNAs in MSC-sEV therapeutics: implications for manufacture, mechanism of action, and development of robust potency CQAs.
   Tan TT, Lim SK. · · 2025 · cited 1× · PMID 41551610 · DOI 10.20517/evcna.2025.55
2. Inhalable Exosomes in Respiratory Therapies with the Transformative Potential.
   Gou J, Zhang L, Wang G, Li Z, et al · · 2025 · cited 1× · PMID 41341867 · DOI 10.2147/ijn.s545306
3. Targeting immunosenescence in lung diseases: mechanistic insights and clinical interventions.
   Zhang Y, Yu X, Li P, Ouyang Y, et al · · 2026 · PMID 41952158 · DOI 10.1186/s12916-026-04833-9
4. The roles and clinical applications of mesenchymal stem cells and their exosomes in hematologic diseases.
   Deng M, Zhang X, Zhang Y. · · 2025 · PMID 41444972 · DOI 10.1186/s13287-025-04880-8
5. Advanced cell-derived drug delivery systems for pulmonary diseases: from bench to bedside.
   Li F, Xu W, Wu J, Zhang J, et al · · 2025 · PMID 41025153 · DOI 10.1080/10717544.2025.2564814
6. Mesenchymal stem cell-derived extracellular vesicles: current advances in preparation and therapeutic applications for neurological disorders.
   Li S, Zhang J, Sun L, Yang Z, et al · · 2025 · PMID 40900731 · DOI 10.3389/fcell.2025.1626996