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Alzheimer DiseasePhase 1NCT06607900

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

A Phase 1 study testing hUC-MSC-sEV-001 nasal drops in people with Alzheimer Disease and Parkinson Disease, run by Xuanwu Hospital, Beijing at 1 site in China.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
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Trial 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… Read more about this study

Who is it for?
Ages 18 to 80 years
What is being tested?
hUC-MSC-sEV-001 nasal drops
Could I receive a placebo?
No, everyone receives an active treatment
How many people
120 participants
Where
1 siteChina
Study dates
1 Jul 2025to 31 Aug 2028
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.
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  • 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.

Who cannot join

  • 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.
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  • 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:
  • 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:
  • 1. 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:
  • Modified Unified Multiple System Atrophy Rating Scale (UMSARS) Part I score ≥ 14.
  • 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:
  • 1. 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:
  • 1. Diagnosis of non-ALS patients based on clinical presentation and available auxiliary clinical examinations (neurophysiology, MRI or other imaging techniques, laboratory tests, etc.).

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Junwei Hao, MD;PhD contact

86-01-83198277

haojunwei@vip.163.com

China1 site
  • Xuanwu Hospital, Capital Medical University

    Beijing, Beijing Municipality, 100053

    Junwei Hao, MD,PhD, 86-10- 83198277, haojunwei@vip.163.com

Other trials in China

About this study

Official title: 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.

Being tested
hUC-MSC-sEV-001 nasal drops
Medical subject headings
Alzheimer Disease, Parkinson Disease, Lewy Body Disease, Multiple System Atrophy, Amyotrophic Lateral Sclerosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • hUC-MSC-sEV-001 nasal dropsDrug

    hUC-MSC-sEV-001 nasal drops

Study groups in NCT06607900
Study groupRoleWhat they receive
hUC-MSC-sEV-001 groupExperimentalhUC-MSC-sEV-001 nasal drops treatment
Receives: Drug: hUC-MSC-sEV-001 nasal drops

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: 12 months

  2. 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.

    Measured: 12 months

  3. 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.

    Measured: 12 months

  4. 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\&amp;#39;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.

    Measured: 12 months

  5. 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.

    Measured: 12 months

  6. 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).

    Measured: 6 months

Secondary outcomes (17)
  1. Barthel index

    The Barthel Index is used for measuring a person\&amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;amp;#39;s ability to perform basic activities of daily living.Scores typically range from 0 (totally dependent) to 100 (completely independent), with higher scores indicating greater independence in daily functioning.

    Measured: 6 months, 12months

  2. mini mental status exam(MMSE)

    The Mini-Mental State Examination (MMSE) is used for assessing cognitive function. The MMSE is scored out of a total of 30 points. A higher score indicates better cognitive function.

    Measured: 6 months, 12months

  3. EQ-5D-5L

    The EQ-5D-5L is used to measure health-related quality of life. The EQ-5D-5L assesses health on a scale of 0 to 25. A higher score means a lower quality of life.

    Measured: 6 months, 12months

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

    The CDR-SB is used to assess the severity of cognitive impairment and dementia. The total score can range from 0 to 18, where higher scores indicate greater levels of impairment across the domains.

    Measured: 6 months, 12months

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

    The ADCS-ADL is used to assess functional decline in patients with Alzheimer's disease, especially in the mild to moderate stages. The total score ranges from 0 to 78 , with higher scores indicating greater functional independence.

    Measured: 6 months, 12months

  6. multiple system atrophy: Composite Autonomic Symptom Score

    The COMPASS questionnaire is used to evaluate autonomic dysfunction, which can present with a variety of symptoms.Higher scores indicate greater impairment in autonomic function.

    Measured: 6 months, 12months

  7. Clinical Global Impression

    CGI is used to assess the severity of a patient's illness, the change in their condition over time, and the efficacy of treatments. It is scored on a 7-point scale, with a higher score indicating a worsen condition.

    Measured: 6 months, 12months

  8. lewy body dementia: MDS-UPRDS

    Measured: 6 months, 12months

  9. Lewy body dementia:Neuropsychiatric Inventory(NPI)

    NPI is used to assess neuropsychiatric symptoms and behavioral disturbances in individuals. The Total NPI Score ranges from 0 to 144. Higher scores indicate greater overall severity of neuropsychiatric disturbances.

    Measured: 6 months, 12months

  10. frontotemporal dementia: NPI

    Measured: 6 months, 12months

  11. frontotemporal dementia: ADAS-cog

    Measured: 6 months, 12months

  12. Amyotrophic lateral sclerosis: Change in forced vital capacity to predicted value ratio (FVC% pred)

    Spirometry is a physiological test that measures how an individual inhales or exhales volumes of air as a function of time. The primary signal measured in spirometry may be volume or flow. The forced vital capacity (FVC) is the maximal volume of air exhaled with maximally forced effort from a maximal inspiration.

    Measured: 6 months

  13. Amyotrophic lateral sclerosis: Change in the Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40).

    ALSAQ-40 score ranges from 0 to 160 points, and higher scores indicate better quality of life.

    Measured: 6 months

  14. Amyotrophic lateral sclerosis: Change in the Edinburgh Cognitive and Behavioral ALS Screen (ECAS).

    ECAS determine cognitive and behavioral changes of patients suffering from Amyotrophic Lateral Sclerosis.

    With ECAS, ALS-specific (fluency, executive functions and social cognition, language; minimum score = 0, maximum score = 100) and ALS-nonspecific (memory, visuospatial functions; minimum score = 0, maximum score = 36) functions can be analyzed to enable the distinction from other diseases with cognitive and behavioral impairments.

    Measured: 6 months

  15. Amyotrophic lateral sclerosis: Change in the Neurophysiological Index (NI) and Compound Muscle Action Potential (CMAP) score.

    The NI is derived from the CMAP, the DML and the F-wave frequency (CMAP amplitude/DML) x F frequency %), representing aspects of the effects of denervation and reinnervation, of degeneration of the terminal part of the motor axons, and of the excitability of anterior horn cells.

    The negative amplitudes of the CMAP for motor nerve conduction in the median and ulnar nerves in the upper extremities as well as the peroneal and tibial nerves in the lower extremities of ALS patients were recorded (median nerve-wrist, ulnar-wrist, peroneal-ankle, tibial-ankle). These nerves were scored based on the decrease in the amplitudes of each nerve CMAP- 0 (CMAP> =[X-2s]), 1 (50% [X-2s] < CMAP < [X-2s]), 2 (30% [X-2s] < CMAP < 50% [X-2s]), 3 (CMAP < =30% [X-2s]), respectively. The values of X and S for different nerves and different patient age groups, which were applied in the electrophysiology examination room.

    Measured: 6 months

  16. Amyotrophic lateral sclersis: Change in the Rasch Overall ALS Disability Scale (ROADS).

    ROADS score ranges from 0 to 56 points, and higher scores indicate better basic daily function.

    Measured: 6 months

  17. Adverse effects

    Measured: 3months, 6 months, 12months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Sep 2024
  2. Study start1 Jul 2025 (estimated)
  3. Primary completion31 Aug 2027 (estimated)
  4. Study completion31 Aug 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
120 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06607900
Sponsor's protocol ID
XMEC-2024-002
Status last verified
Jul 2025
First submitted
19 Sep 2024
Last updated
8 Jul 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing hUC-MSC-sEV-001 nasal drops in people with Alzheimer Disease and Parkinson Disease, run by Xuanwu Hospital, Beijing at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 23 inclusion and 29 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 8 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Junwei Hao, MD;PhD (86-01-83198277, haojunwei@vip.163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Alzheimer's disease: Change in Alzheimer's disease assessment scale-cognitive section(ADAS-cog) (time frame: 12 months). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jul 2025.

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