Anti-MDA5 Positive Dermatomyositis-Associated RP-ILDPhase 1NCT06919380
Nebulized MSC-Exos for Anti-MDA5+ RP-ILD: Safety and Efficacy Trial
A Phase 1 study testing MSC-exos Nebulization Therapy in people with Anti-MDA5 Positive Dermatomyositis-Associated RP-ILD and Rapidly Progressive Interstitial Lung Disease, run by Li Shiyue at 1 site in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Anti-MDA5 Positive Dermatomyositis-Associated RP-ILD, Rapidly Progressive Interstitial Lung Disease
- What is being tested?
- MSC-exos Nebulization Therapy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 15 Apr 2025to 31 May 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of Guangzhou Medical University
Guangzhou, Guangdong, 510150
bingpeng guo, 020-81567301, drkwok@126.com
About this study
Official title: 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)
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.
Read more
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.
Read the detailed description
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.
- Conditions
- Anti-MDA5 Positive Dermatomyositis-Associated RP-ILD, Rapidly Progressive Interstitial Lung Disease
- Being tested
- MSC-exos Nebulization Therapy
- Keywords
- Interstitial Lung Disease, Anti-MDA5 Positive, Rapidly Progressive Interstitial Lung Disease
- Medical subject headings
- Lung Diseases, Interstitial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MSC-exos Nebulization TherapyDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Nebulized MSC-exos for Anti-MDA5+ RP-ILD Treatment | Experimental | 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). Receives: Drug: MSC-exos Nebulization Therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 3 months post-treatment initiation]
Secondary outcomes (6)
Oxygen Saturation
Oxygen Saturation: SpO2 (%) via pulse oximetry
Measured: Baseline, day 14, and day 28 post-treatment initiation
CT Lesion Changes
CT Lesion Changes: Semi-quantitative scoring system (0-25)
Measured: Baseline, day 14, and day 28 post-treatment initiation
Symptom Improvement
Symptom Improvement: VAS (0-10) for dyspnea and cough
Measured: Baseline, day 14, and day 28 post-treatment initiation
C-reactive Protein (CRP)
Serum C-reactive Protein levels (CRP) (mg/L)
Measured: Baseline, day 14, and day 28 post-treatment initiation
Interleukin-6 (IL-6)
Interleukin-6 (IL-6) (pg/mL)
Measured: Baseline, day 14, and day 28 post-treatment initiation
D-dimer
Plasma D-dimer (μg/L)
Measured: Baseline, day 14, and day 28 post-treatment initiation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2025
- Study start15 Apr 2025 (estimated)
- Primary completion31 Dec 2026 (estimated)
- Study completion31 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anti-MDA5 Positive Dermatomyositis-Associated RP-ILDStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Li Shiyue
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Li Shiyue, Guangzhou Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06919380
- Sponsor's protocol ID
- ES-2024 -156 -01
- Status last verified
- Apr 2025
- First submitted
- 13 Feb 2025
- Last updated
- 10 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT06919380
- Acronym / sponsor ID
- ES-2024 -156 -01
- Conditions
- Anti-MDA5 Positive Dermatomyositis-Associated RP-ILD, Rapidly Progressive Interstitial Lung Disease
- Keywords
- Interstitial Lung Disease, Anti-MDA5 Positive, Rapidly Progressive Interstitial Lung Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Li ShiyueUniversity, hospital or other
- Study start
- 15 Apr 2025 (estimated)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 May 2027 (estimated)
- First posted
- 9 Apr 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Nebulized MSC-exos for Anti-MDA5+ RP-ILD Treatment | Experimental | Drug: MSC-exos Nebulization TherapyThis 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). |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Patients are eligible for inclusion if they meet all of the following criteria:
Key exclusion criteria (14 listed)
- Patients who meet any of the following criteria will be excluded from this study:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MSC-exos Nebulization TherapyThe 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. | Drug | Nebulized MSC-exos for Anti-MDA5+ RP-ILD Treatment |
Primary outcome
Three-Month Mortality Rate and Safety of Nebulized MSC-exos P1
Time frame: 3 months post-treatment initiation]
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.
6 secondary outcomes
Oxygen Saturation
Time frame: Baseline, day 14, and day 28 post-treatment initiation
CT Lesion Changes
Time frame: Baseline, day 14, and day 28 post-treatment initiation
Symptom Improvement
Time frame: Baseline, day 14, and day 28 post-treatment initiation
C-reactive Protein (CRP)
Time frame: Baseline, day 14, and day 28 post-treatment initiation
Interleukin-6 (IL-6)
Time frame: Baseline, day 14, and day 28 post-treatment initiation
D-dimer
Time frame: Baseline, day 14, and day 28 post-treatment initiation
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Apr 2025.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MSC-exos Nebulization Therapy in people with Anti-MDA5 Positive Dermatomyositis-Associated RP-ILD and Rapidly Progressive Interstitial Lung Disease, run by Li Shiyue at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact li (020-81567301, drkwok@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Three-Month Mortality Rate and Safety of Nebulized MSC-exos P1 (time frame: 3 months post-treatment initiation]). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Apr 2025.