Clinical Safety and Efficacy of Umbilical Cord Mesenchymal Stromal Cells-derived Exosomes in Patients With Parkinson's Disease

Condition(s) studied

Investigational drug(s) / intervention(s)

Umbilical cord mesenchymal stromal cell-derived exosomes (UCMSC-Exo): UCMSC-Exo is an open-label product of exosomes from umbilical cord mesenchymal stromal cells (UCMSCs) in saline. It is a transparent liquid given by intranasal spray, not intravenously, and contains no living cells. Each dose is 0.4 mL total (200 µL/nostril; 2 sprays/nostril at \~100 µL/spray), twice weekly (Mon/Thu) for 12 weeks (24 doses), with usual dopaminergic therapy continued. A 3+3 design tests low (5×10\^8 particles/mL), medium (1×10\^9 particles/mL), and high (2×10\^9 particles/mL) concentrations; volume is identical, concentration differs. Escalation proceeds only with no DLT; 1 DLT expands a cohort to 6; \>1 DLT stops escalation. Store at -80°C; use within 2 hours after thawing. Key distinctions: acellular UCMSC-derived exosomes, intranasal route, fixed twice-weekly 12-week regimen, sequential dose escalation, no placebo.

Study summary

The goal of this clinical trial is to learn if intranasal umbilical cord mesenchymal stromal cell-derived exosomes (UCMSC-Exo) are safe and can help treat early Parkinson's disease in adults aged 50-75. It will also learn about the safety and tolerability of UCMSC-Exo. This is a single-center, open-label, dose-escalation study using a 3+3 design; there is no placebo and no blinding.

The main questions it aims to answer are:

1. What local and systemic adverse events and serious adverse events occur with intranasal UCMSC-Exo?
2. Does intranasal UCMSC-Exo improve motor and non-motor symptoms, biomarkers, and imaging measures in Parkinson's disease?
3. Researchers will compare low-, medium-, and high-dose UCMSC-Exo groups sequentially to assess safety and preliminary efficacy.

Participants will:

1. Receive UCMSC-Exo by nasal spray twice weekly (Monday/Thursday) for 12 weeks (24 administrations) at their assigned dose.
2. Continue their usual dopaminergic medication during the study.
3. Visit the clinic for safety assessments at weeks 4, 12, 16, and 48, and for efficacy assessments at weeks 12, 16, 24, 36, and 48.
4. Undergo blood sampling, symptom questionnaires, and PET-MR imaging at scheduled time points.
5. Be followed for a total of 48 weeks, including the 12-week treatment period and 36 weeks after treatment.

Eligibility

Primary outcome measure(s)

• Incidence, severity, and relatedness of local and systemic adverse events and serious adverse events after intranasal UCMSC-Exo in early Parkinson's disease — From first dose through Week 48; safety assessments at Weeks 4, 12, 16, and 48
  Primary safety outcome: incidence, severity, and relationship to study intervention of local and systemic adverse events (AEs) and serious adverse events (SAEs) after intranasal UCMSC-Exo. Local AEs include pain, itching, sneezing, bleeding, allergy, and hyposmia. Systemic AEs include fever, systemic allergic reactions (rash, redness), abnormal inflammatory markers (WBC, CRP), hepatic impairment (ALT/AST \>2× ULN), renal impairment (serum creatinine \> ULN), anemia (Hb \<120 g/L in men; \<110 g/L in women), and abnormal tumor markers. SAEs include severe allergic reactions (edema, asthma). Safety is assessed by physical examination, vital signs, laboratory tests (CBC/CRP, liver and renal function, electrolytes, fasting glucose, IgE, EKG), and AE/SAE questionnaires at Weeks 4, 12, 16, and 48; tumor markers, cardiac markers, DIC, and lipids are assessed at Week 48.

Trial sites (1)

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