hUC-MSC-sEV-001 Nasal Drops for Neurodegenerative Diseases

NCT06607900 | Not Yet Recruiting Phase 1

• 1 other identifier: XMEC-2024-002
• Study Type: interventional
• Target: 120
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Alzheimer Disease; Parkinson Disease; Lewy Body Dementia; Multiple System Atrophy; Fronto-temporal Dementia; Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (6)

• Alzheimer's disease: Change in Alzheimer's disease assessment scale-cognitive section（ADAS-cog）

  ADAS-Cog is comprised of 11 modalities that evaluate memory, praxis, and language deficiencies. The total score range is 0 to 70 points, with higher scores indicating greater cognitive impairment.

  12 months

• Parkinson's disease: Change in MDS Unified-Parkinson Disease Rating Scale(MDS-UPDRS)

  The MDS-UPDRS is defined by 4 Parts, each composed by a different number of items. The MDS-UPDRS has a minimum score of 0 and a maximum score of 260. The higher the score, the more severe the impairment.

  12 months

• multiple system atrophy: Unified Multiple System Atrophy Rating Scale（Part I and Part II）

  UMSARS is a clinical tool designed to assess the severity and progression of MSA. The scale is divided into multiple parts, with Part I focusing on functional disability based on the history and self-report symptoms and Part II the severity of motor symptoms through a physical examination. The higher score, the severer impairment.

  12 months

• lewy body dementia: Alzheimer's Disease Cooperative Study-Clinical Global Impression of Change (ADCS-CGIC)

  ADCS-CGIC provides a clinician-rated, subjective evaluation of a patient\'s cognitive, behavioral, and functional performance based on interviews and observations. The scoring typically uses a 7-point scale. The lower score, the better changes in overall condition.

  12 months

• frontotemporal dementia: CDR Dementia Staging Instrument PLUS National Alzheimer's Coordinating Center Behavior and Language Domains（CDR+NACC FTLD）

  The CDR Dementia Staging Instrument provides a global assessment of cognitive and functional decline, while the NACC Behavior and Language Domains offer a more nuanced view of neuropsychiatric symptoms and language impairments.

  12 months

• Amyotrophic lateral sclerosis: Change in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)

  The ALSFRS-R is rating scale (ratings 0 = can't do, to 4 = normal ability) used to determine participants' assessment of their capability and independence in 12 functional activities. This is a validated scale, both in person and by phone, which provides a total score from four sub-scores which assess speech and swallowing (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). Total scores range from 0 (most impaired) to 48 (normal ability).

  6 months

Secondary Outcomes (17)

• Barthel index

  6 months, 12months

• mini mental status exam(MMSE)

  6 months, 12months

• EQ-5D-5L

  6 months, 12months

• Alzheimer's disease: sum of boxes of the CDR（CDR-SB）

  6 months, 12months

• Alzheimer's disease: Alzheimer's Disease Cooperative Study - Activities of Daily Living （ADCS-ADL）

  6 months, 12months

Study Arms (1)

hUC-MSC-sEV-001 group

EXPERIMENTAL

hUC-MSC-sEV-001 nasal drops treatment

Drug: hUC-MSC-sEV-001 nasal drops

Interventions

hUC-MSC-sEV-001 nasal drops DRUG

hUC-MSC-sEV-001 nasal drops

hUC-MSC-sEV-001 group

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• Subjects requiring nasogastric tube insertion.
• 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.
• 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).
• 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.
• Contraindications to MRI examination (e.g., metal implants) or inability to tolerate MRI (e.g., claustrophobia).
• 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.
• 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.
• 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:

Sponsors & Collaborators

• Xuanwu Hospital, Beijing: lead

Study Sites (1)

Xuanwu Hospital, Capital Medical University

Beijing, Beijing Municipality, 100053, China

Location

MeSH Terms

Conditions

Alzheimer Disease; Parkinson Disease; Lewy Body Disease; Multiple System Atrophy; Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Dementia; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders; Parkinsonian Disorders; Basal Ganglia Diseases; Movement Disorders; Synucleinopathies; Primary Dysautonomias; Autonomic Nervous System Diseases; Spinal Cord Diseases; Motor Neuron Disease; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Junwei Hao, MD;PhD

CONTACT

86-01-83198277; haojunwei@vip.163.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 19, 2024
• First Posted: September 23, 2024
• Study Start: July 1, 2025
• Primary Completion (Estimated): August 31, 2027
• Study Completion (Estimated): August 31, 2028
• Last Updated: July 8, 2025

Record last verified: 2025-07

Locations

CN (1)