ClinicalTrials.gov record NCT06919380

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

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)

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 1An early-stage study, usually small, mainly to check safety, side effects and how much of a treatment can be given. Whether it works is not the main question yet.
Age18 years old to 75 years old
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

A Phase 1, open-label study testing daily nebulized mesenchymal stem cell–derived exosomes (MSC-exos-P1) for 14 days in people with anti‑MDA5 positive dermatomyositis who have rapidly progressive interstitial lung disease. Ten participants aged 18–75 will receive the investigational inhaled treatment alongside standard immunosuppressive care. The main goal is to assess safety and tolerability; early clinical, imaging, and blood-marker changes will be measured up to 28 days, with follow-up continuing to 12 months.

What it is studying. The study is testing whether inhaled MSC-exos-P1 are safe and tolerated in people with anti‑MDA5+ dermatomyositis-associated rapidly progressive ILD, and it will collect early data on clinical measures (including oxygen levels and symptoms), chest CT lesion scores, and blood markers to look for signs of effect. Primary safety assessments include adverse events and three-month mortality; secondary assessments include measures at baseline, day 14, and day 28.

What the researchers hope to learn. To evaluate the safety and tolerability of nebulized MSC-exos-P1 in this patient group; to measure three-month mortality and adverse events; and to collect preliminary data on oxygenation, CT lesion scores, symptoms, and inflammatory biomarkers to inform later studies.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • MSC-exos Nebulization Therapy

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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 criteria

  • 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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive daily nebulized MSC-exos-P1 treatment for 14 days while continuing standard care with corticosteroids and immunosuppressants. They will have close monitoring during treatment (daily vital signs, laboratory tests, and recording of adverse events) and assessments at baseline, day 14, and day 28, including oxygenation checks, pulmonary function tests, symptom scores, blood markers, and chest CT scans. There are additional follow-up visits extending up to 12 months to monitor safety and outcomes. The study team documents and follows any side effects throughout the study period.

How the study is structured. Phase 1 interventional treatment study. Single-group, open-label (no masking). All participants (target n=10) receive daily nebulized MSC-exos for 14 days in addition to their usual corticosteroid and immunosuppressant care. Assessments include daily safety monitoring during treatment, clinical and imaging measures at baseline, day 14 and day 28, and longer follow-up visits up to 12 months.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on dermatomyositis

Clinical trials59 trials listed for dermatomyositisEvery trial the registry lists for dermatomyositis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in dermatomyositis researchLatest monthly update: September 2026. Includes: Upadacitinib case reports. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Apr 15, 2025 (estimated)
Primary completion
Dec 31, 2026 (estimated)
Study completion
May 31, 2027 (estimated)
Target enrollment
10 participants (estimated)
Last updated on registry
Apr 10, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Li Shiyue

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

  • The First Affiliated Hospital of Guangzhou Medical UniversityRecruiting

    Guangzhou, Guangdong, 510150 · China

Terms used on this page · explanations by MedicalUpdates.ai

Primary endpoint
The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
adverse events
Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
endpoints
Something the study measures to judge its results, such as a symptom score, a blood test or survival. Outcomes are grouped as primary, secondary and sometimes exploratory.
HRCT
An X-ray scan that builds detailed cross-section images of the body. 'High-resolution' CT (HRCT) is used to look closely at the lungs.
corticosteroids
Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
immunosuppressants
A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
biomarkers
Something measurable in the body, such as a blood test result or an imaging finding, that indicates how a condition or treatment is behaving.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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