Xuanwu Hospital in Beijing is launching a multi-center, open-label, single-arm Phase I clinical trial to assess the safety and efficacy of hUC-MSC-sEV-001 nasal drops—a therapy derived from human umbilical cord mesenchymal stem cell small extracellular vesicles—for several neurodegenerative diseases.
The trial will take place at multiple sites in China and aims to enroll approximately 120 participants.
Neurodegenerative diseases such as Alzheimer’s disease, Parkinson’s disease, multiple system atrophy, Lewy body dementia, frontotemporal dementia, and amyotrophic lateral sclerosis (ALS) are characterized by progressive loss of neurological function and currently lack curative treatments.
Researchers are seeking new therapeutic strategies, including regenerative approaches, to slow or halt disease progression and improve patient quality of life. In this trial, all participants will receive the therapy (hUC-MSC-sEV-001) as nasal drops. According to the study protocol, the therapy is tested in a “basket” design—meaning a single investigational drug is tested across multiple diseases sharing common pathological mechanisms. There is no control or placebo arm.
The focus is on evaluating:
- Safety and tolerability of the nasal-administered stem cell-derived vesicles across all targeted neurodegenerative conditions.
- Potential efficacy, as measured by disease-specific clinical assessments at set time points (6 and 12 months).
The primary objectives, as reported by the investigators, are to measure:
- Changes in the ADAS-cog score for Alzheimer’s disease (evaluating cognitive impairment over 12 months).
- Change in the MDS-UPDRS for Parkinson’s disease (assessing motor and non-motor impairment at 12 months).
- Unified Multiple System Atrophy Rating Scale (UMSARS) Parts I and II for multiple system atrophy (12 months).
- ADCS-Clinical Global Impression of Change for Lewy body dementia (12 months).
- CDR Dementia Staging Instrument plus NACC domains for frontotemporal dementia (12 months).
- ALS Functional Rating Scale-Revised (ALSFRS-R) for ALS (6 months).
Secondary objectives include a broad set of functional and quality-of-life measures, such as the Barthel Index, MMSE, EQ-5D-5L, disease-specific cognitive and behavioral scores, spirometry for ALS, and regular adverse event tracking at 3, 6, and 12 months.
The researchers note that extracellular vesicles (EVs) from mesenchymal stem cells may modulate inflammation, support cell survival, and facilitate repair in the nervous system, potentially offering benefits across a spectrum of neurodegenerative conditions. Early-stage studies of stem cell-derived EVs have suggested safety in other contexts, but their utility as nasal drops for these diseases has not yet been demonstrated in a large clinical population. The trial is scheduled to begin recruitment in July 2025, with primary completion expected in August 2027 and full study completion in August 2028.
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