hUCMSC-EVs — Umbilical Cord MSC-Derived Extracellular Vesicles for Interstitial Lung Disease, Phase 1
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| Trial ID | NCT07570836 ↗ |
|---|---|
| Sponsor | Shanghai Zhongshan Hospital · China |
| Cell Source | 제대 중간엽줄기세포 (UC-MSC) · allogeneic |
| Indication | interstitial-lung-disease |
| Phase | PHASE 1 |
| Status | NOT-YET-RECRUITING |
| Delivery Route | nebulized inhalation |
| Enrollment | 24 |
| Publication | — |
Trial Design
This is an exploratory, randomised, single-blind, placebo-controlled, multiple-dose, dose-escalation Phase 1 trial evaluating the safety and preliminary efficacy of human umbilical cord mesenchymal stem cell-derived extracellular vesicles (hUCMSC-EVs) in patients with interstitial lung disease.
Participants are enrolled sequentially into three dose cohorts: low, intermediate, and high dose. Each cohort comprises 8 participants, randomised 3:1 to hUCMSC-EVs or normal saline placebo within the cohort.
The primary endpoint is the incidence and severity of adverse events from first administration to 12 weeks. Secondary endpoints include changes in FVC, DLCO, six-minute walk distance, and SGRQ.
Clinical Significance
This study illustrates the expansion of pulmonary regenerative medicine from direct MSC administration to cell-free therapy based on MSC-derived extracellular vesicles. It is notable that within the same indication — interstitial lung disease — research on both umbilical cord-derived MSC injection and hUCMSC-EV inhalation is now emerging in parallel.
Reasons hUCMSC-EVs are drawing attention:
- Cell-free therapy — MSC-derived EVs rather than the cells themselves
- Lung-specific delivery route — nebulised inhalation rather than intravenous infusion
- Functional outcome measures — six-minute walk distance included alongside FVC and DLCO
- Placebo-controlled design — comparison against normal saline placebo
Limitations and Discussion
As a Phase 1 study, it is premature to emphasise therapeutic efficacy. Standardisation of EV manufacture, particle characterisation, potency assessment, and lot-to-lot consistency remain important unresolved challenges. How the intra-pulmonary distribution and duration of biological activity of inhaled EVs will be evaluated also represents an important methodological question.