ClinicalTrials.gov record NCT06607900

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 7, 2026

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

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 1An early-stage study, usually small, mainly to check safety, side effects and how much of a treatment can be given. Whether it works is not the main question yet.
Age18 years old to 80 years old
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 1, multi-center, open-label, single-arm "basket" study gives all participants hUC-MSC-sEV-001 nasal drops and evaluates safety and early efficacy signals. The study includes people with Alzheimer Disease, Parkinson Disease, Lewy Body Dementia, Multiple System Atrophy, Fronto-temporal Dementia, or Amyotrophic Lateral Sclerosis. Outcomes are measured with disease-specific clinical scales over months of follow-up; there is no placebo or comparison group.

What it is studying. Whether hUC-MSC-sEV-001 nasal drops are safe and to collect preliminary data on possible changes in symptoms or function across the listed neurodegenerative diseases.

What the researchers hope to learn. To assess the safety and tolerability of hUC-MSC-sEV-001 nasal drops and to collect preliminary efficacy data using disease-specific rating scales and functional measures. The study will record adverse events and other safety data and gather early outcome measurements at time points specified in the protocol (e.g., 6 and 12 months).

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • hUC-MSC-sEV-001 nasal drops

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • General Criteria:
  • 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.

Exclusion criteria

  • 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:
  • 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.
  • 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:
  • 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.
  • 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:
  • 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.
  • 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:
  • 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.
  • 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:
  • 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 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:
  • 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.
  • Diagnosis of non-ALS patients based on clinical presentation and available auxiliary clinical examinations (neurophysiology, MRI or other imaging techniques, laboratory tests, etc.).

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive the investigational product (hUC‑MSC‑sEV‑001) as nasal drops and attend regular study visits over months for safety and disease assessments. Visits will include disease‑specific rating scales and questionnaires (for example ADAS‑cog, MDS‑UPDRS, ALSFRS‑R), brain MRI scans, blood tests, and, for ALS patients, lung function tests and neurophysiology/nerve assessments as specified. The study team will monitor for adverse effects and collect the measures used in the trial; participants must follow contraception and vaccination rules and avoid other investigational drug studies within 30 days of dosing. Participants (or their legal guardian) must sign informed consent and be able to comply with dosing and follow‑up requirements.

How the study is structured. An interventional Phase 1 trial in which every participant receives the investigational nasal-drop drug (single-group, no blinding). It is a basket design: several different neurodegenerative diseases are enrolled and each disease is assessed with its own clinical scales over scheduled follow-up visits.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on multiple system atrophy

Clinical trials76 trials listed for multiple system atrophyEvery trial the registry lists for multiple system atrophy, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in multiple system atrophy researchLatest monthly update: September 2026. Includes: Tube feeding. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jul 1, 2025 (estimated)
Primary completion
Aug 31, 2027 (estimated)
Study completion
Aug 31, 2028 (estimated)
Target enrollment
120 participants (estimated)
Last updated on registry
Jul 8, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Xuanwu Hospital, Beijing

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

written informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
investigational product
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
creatinine clearance
A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
absolute neutrophil count
The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
Contraindications
A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
MRI
A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
Women of childbearing potential
Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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