Tests stem cell vesicle nasal drops in several neurodegenerative diseases

Trial details

Who runs it

Xuanwu Hospital, Beijing

Trial code

NCT06607900

Record updated

8 July 2025

Original description

To evaluate the safety and preliminary efficacy of human umbilical cord mesenchymal stem cell-derived small extracellular vesicles hUC-MSC-sEV-001 nasal drops in multiple neurodegenerative diseases, including Alzheimer's disease, Parkinson's disease, multiple system atrophy, Lewy body dementia, frontotemporal dementia, and amyotrophic lateral sclerosis.Read full original descriptionShow less

A Multi-center, Open, Single-arm, Basket-design Clinical Trial to Evaluate the Safety and Efficacy of Human Umbilical Cord Mesenchymal Stem Cell-derived Small Extracellular Vesicles hUC-MSC-sEV-001 Nasal Drops for the Treatment of Multiple Neurodegenerative Diseases

To evaluate the safety and preliminary efficacy of human umbilical cord mesenchymal stem cell-derived small extracellular vesicles hUC-MSC-sEV-001 nasal drops in multiple neurodegenerative diseases, including Alzheimer's disease, Parkinson's disease, multiple system atrophy, Lewy body dementia, frontotemporal dementia, and amyotrophic lateral sclerosis.

Inclusion: Age 18-80 years (inclusive), any gender; Subjects or their legal guardians voluntarily sign a written informed consent form and are able to comply with the study requirements for dosing and follow-up; Probable AD as defined by the 2011 National Institute on Aging-Alzheimer's Association (NIA-AA) criteria; Clinical Dementia Rating (CDR) score ≤ 1.0; Subjects have an identified, reliable caregiver; Stable treatment regimen for at least 1 month prior to dosing; Diagnosis of PD according to the 2015 Movement Disorder Society (MDS) clinical diagnostic criteria for PD; Hoehn and Yahr stage ≤ 3; Stable treatment regimen for at least 1 month prior to dosing; Diagnosis of possible or probable multiple system atrophy; Time since diagnosis < 3 years from baseline, with an expected survival of at least 3 years; Stable treatment regimen for at least 1 month prior to dosing; Meets the revised consensus criteria for DLB (Fourth Consensus Report of the DLB Consortium, 2017); Severity of motor symptoms must be ≤ stage 3 on the Hoehn and Yahr scale; Clinical Dementia Rating (CDR) score ≤ 1.0; Stable treatment regimen for at least 1 month prior to dosing; Meets the 2017 International Research Society (IRS) diagnostic criteria for FTD; Clinical Dementia Rating (CDR) score ≤ 1.0; Stable treatment regimen for at least 1 month prior to dosing; Diagnosis of Amyotrophic Lateral Sclerosis (ALS) meeting the diagnostic criteria (Revised El Escorial Criteria, 2000 or Airlie House Criteria) at the level of definite, probable, or laboratory-supported probable ALS; Disease duration ≥ 6 months and ≤ 2 years (from the first occurrence of any ALS symptom); Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score: each individual item score ≥ 2 points (with the three respiratory items - Dyspnea, Orthopnea, and Respiratory Insufficiency - all scoring 4 points); Stable treatment regimen for at least 1 month prior to dosing.

Exclusion: Subjects who have received allogeneic mesenchymal progenitor cell therapy or its derived small extracellular vesicles; Subjects with abnormal nasal anatomy, nasal damage, severe rhinitis, or other nasal conditions that may affect the administration of the investigational product; Contraindications to MRI examination (e.g., metal implants) or inability to tolerate MRI (e.g., claustrophobia); Vaccination within 1 month prior to the first dose or planned during the period from enrollment until the end of follow-up; Participation in other clinical drug studies within the past 30 days; Parkinsonism other than PD, including but not limited to progressive supranuclear palsy (PSP), multiple system atrophy (MSA), vascular parkinsonism, drug-induced parkinsonism, essential tremor, primary dystonia; Multiple System Atrophy (MSA) Specific Criteria:; Suffering from other uncontrolled diseases that may interfere with the study results, including but not limited to severe local infection, systemic infection, or immunodeficiency; Combined with malignant tumors, hematological malignancies, or other serious systemic diseases; Clinically significant history of allergic reactions, especially drug allergic reactions; HBsAg positive, or HBcAb positive with HBV DNA positive, hepatitis C virus (HCV) antibody positive, peripheral blood HCV RNA positive, HIV antibody positive; CMV DNA positive, syphilis serology positive; Any condition that, in the investigator's judgment, may compromise the subject's ability to understand and/or comply with the study procedures and/or follow-up; Alzheimer's Disease (AD) Specific Criteria:; Major history of significant brain injury with persistent neurological impairment (with or without) or known structural brain abnormalities; Cognitive impairment due to other causes: central nervous system infection, Creutzfeldt-Jakob disease, traumatic dementia, other physical/chemical factors (e.g., drug intoxication, alcoholism, carbon monoxide poisoning), major systemic illnesses (e.g., hepatic encephalopathy, pulmonary encephalopathy), intracranial space-occupying lesions (e.g., subdural hematoma, brain tumor), endocrine disorders (e.g., thyroid disease, parathyroid disease), vitamin B12 or folate deficiency, or any other known causes; Parkinson's Disease (PD) Specific Criteria:; Presence of any condition that, in the investigators' judgment, affects the diagnosis or assessment of MSA; Dementia with Lewy Bodies (DLB) Specific Criteria:; Patients currently diagnosed with any primary psychiatric disorder (e.g., schizophrenia, bipolar disorder, or major depressive episode) according to DSM-V; Patients with clinically significant or unstable systemic illness and exposure to toxicants within the past 5 years; Frontotemporal Dementia (FTD) Specific Criteria:; Diagnosis of severe central nervous system diseases other than FTD that may be the cause of the patient's FTD symptoms or may affect the study objectives; Amyotrophic Lateral Sclerosis (ALS) Specific Criteria:; Severe renal insufficiency: creatinine clearance (CrCl) < 30 mL/min (calculated by Cockcroft-Gault formula), or other known severe renal diseases; Peripheral blood hemoglobin (HGB) < 100 g/L, absolute neutrophil count (NEUT) < 1.5 × 10⁹/L, platelet count (PLT) < 100 × 10⁹/L, white blood cell count (WBC) < 4.0 × 10⁹/L or ≥ 12 × 10⁹/L, serum albumin < 30 g/L; alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≥ 3 × upper limit of normal (ULN); Diagnosis of non-ALS patients based on clinical presentation and available auxiliary clinical examinations (neurophysiology, MRI or other imaging techniques, laboratory tests, etc.); Subjects requiring nasogastric tube insertion; Women of childbearing potential not intending to use effective contraception during the trial or within 90 days after the last dose and with a positive pregnancy test record; pregnant or lactating women; men who are sexually active during the trial or within 90 days after the last dose and not intending to use effective contraception; or men planning to donate sperm during the trial or within 90 days after the last dose; Modified Unified Multiple System Atrophy Rating Scale (UMSARS) Part I score ≥ 14.

General Criteria:.

ClinicalTrials.gov NCT06607900