Who may be eligible for the hUC-MSC-Exo Therapy for Autoimmune Encephalitis trial?

Inclusion Criteria: 1. Aged 18-65 years, both male and female are eligible; 2. Diagnosis of autoimmune encephalitis within 3 months of onset (meeting the 2016 Graus and Dalmau diagnostic criteria), with positive serum/cerebrospinal fluid anti-NMDAR antibodies or anti-LGI1 antibodies; 3. Modified Rankin Scale (mRS) score ≥ 2 at enrollment; 4. The subject or legally authorized representative is able to sign the informed consent form; 5. Subjects of childbearing potential must agree to practice strict contraception during the study period. Exclusion Criteria: 1. Pre-morbid modified Rankin Scale (mRS) score ≥ 2; 2. Known allergy to any component of the investigational product or history of severe allergic reactions; 3. Presence of neurological or psychiatric disorders (e.g., cerebrovascular disease, Parkinson's disease, severe depression) deemed by the investigator to potentially impair trial participation or study assessments; 4. Current or history of any clinically significant systemic diseases judged by the investigator as unsuitable for inclusion, including but not limited to: * Severe cardiovascular diseases (e.g., congestive heart failure, severe arrhythmia, myocardial infarction) * Hepatic diseases (e.g., cirrhosis) * Renal diseases (e.g., requiring hemodialysis or peritoneal dialysis) * Hematological diseases (e.g., hemophilia with bleeding tendency) * Endocrine disorders (e.g., poorly controlled diabetes with blood glucose \>16.8 mmol/L or \<2.8 mmol/L, or with severe complications) * Immune system disorders (active or uncontrolled systemic autoimmune diseases, primary/secondary immunodeficiency) * Malignancies; 5. Anatomical nasal abnormalities, nasal mucosal damage, severe rhinitis, or other nasal conditions affecting drug administration; 6. Requiring nasogastric tube placement; 7. Organ function meeting any of the following criteria: 1. Absolute neutrophil count (ANC) \ 2.5×ULN and/or alanine aminotransferase (ALT) \>2.5×ULN, total bilirubin (TBIL) \>1.5×ULN 3. Creatinine \>1.5×ULN 4. Without anticoagulant/antiplatelet therapy: International normalized ratio (INR) \>1.7 or activated partial thromboplastin time (APTT) \>1.25×ULN With anticoagulant/antiplatelet therapy: INR \>3.0 or APTT \>1.5×ULN; 8. Positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with detectable HBV-DNA; or positive for hepatitis C antibody (HCVAb), Treponema pallidum antibody (TPAb/RPR), or human immunodeficiency virus antibody (HIV); 9. Pregnant or lactating patients; 10. Contraindications for MRI (e.g., metal implants such as pacemakers, claustrophobia); 11. Participation in any clinical trial involving investigational drugs within 3 months prior to dosing (or within 5 half-lives of last dose, whichever is longer); 12. Major trauma or surgery within 3 months prior to dosing, or planned surgery during the trial (excluding laparoscopy or minor procedures \>4 weeks before baseline; excluding thymoma/teratoma surgery); 13. History of drug abuse or alcoholism within 1 year prior to dosing; 14. Previous treatment with stem cells or derivatives; 15. Any other condition that may increase patient risk or interfere with result interpretation, as determined by the investigator.

ClinicalTrials.gov record NCT07131683

hUC-MSC-Exo Therapy for Autoimmune Encephalitis

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

Human Umbilical Cord Mesenchymal Stem Cell-derived ExoSomes for Autoimmune Encephalitis: a Phase I/IIa Clinical Trial (MESAE)

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

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 1/2A study that combines safety and dose-finding (phase 1) with a first look at whether the treatment has an effect (phase 2).

Age18 years old to 65 years old

SexAll

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 I/IIa interventional study tests intranasal human umbilical cord mesenchymal stem cell‑derived exosomes (hUC‑MSC‑Exo) in people with autoimmune encephalitis. It starts with an open‑label dose‑escalation phase to find a tolerated dose, then moves to a randomized, double‑blind, placebo‑controlled expansion comparing the chosen dose with a placebo mimic. Treatment is given as a nasal spray daily for 7 days then weekly for 3 weeks, with safety and clinical follow‑up through 24 weeks.

What it is studying. The trial is testing the safety, tolerance, and preliminary efficacy of intranasal hUC‑MSC‑Exo in people with autoimmune encephalitis. The study will identify dose‑limiting toxicities and a maximal tolerated dose during dose escalation, then compare the selected dose with a placebo mimic in a randomized, blinded expansion. Primary outcomes include drug‑related dose‑limiting toxicities and the modified Rankin Scale at 24 weeks.

What the researchers hope to learn. Scientifically, the study aims to assess safety and tolerability, identify dose‑limiting toxicities and the maximal tolerated dose, and collect preliminary data on clinical outcomes (mRS, CASE, ADL, MMSE, MoCA) and relapse rate, as well as adverse events up to 24 weeks.

What this study is about

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

This is a phase I/IIa study to investigate the safety and preliminary efficacy of intranasal admnistration of human umbilical mesenchymal stem cell-derived exosome (hUC-MSC-Exo) for patients with autoimmune encephalitis.

Interventions being studied

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

Biological: Human umbilical cord mesenchymal stem cell derived exosomes
Other: Placebo Control

Eligibility

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

Inclusion criteria

Aged 18-65 years, both male and female are eligible;
Diagnosis of autoimmune encephalitis within 3 months of onset (meeting the 2016 Graus and Dalmau diagnostic criteria), with positive serum/cerebrospinal fluid anti-NMDAR antibodies or anti-LGI1 antibodies;
Modified Rankin Scale (mRS) score ≥ 2 at enrollment;
The subject or legally authorized representative is able to sign the informed consent form;
Subjects of childbearing potential must agree to practice strict contraception during the study period.

Exclusion criteria

Pre-morbid modified Rankin Scale (mRS) score ≥ 2;
Known allergy to any component of the investigational product or history of severe allergic reactions;
Presence of neurological or psychiatric disorders (e.g., cerebrovascular disease, Parkinson's disease, severe depression) deemed by the investigator to potentially impair trial participation or study assessments;
Current or history of any clinically significant systemic diseases judged by the investigator as unsuitable for inclusion, including but not limited to:
Severe cardiovascular diseases (e.g., congestive heart failure, severe arrhythmia, myocardial infarction)
Hepatic diseases (e.g., cirrhosis)
Renal diseases (e.g., requiring hemodialysis or peritoneal dialysis)
Hematological diseases (e.g., hemophilia with bleeding tendency)
Endocrine disorders (e.g., poorly controlled diabetes with blood glucose >16.8 mmol/L or <2.8 mmol/L, or with severe complications)
Immune system disorders (active or uncontrolled systemic autoimmune diseases, primary/secondary immunodeficiency)
Malignancies;
Anatomical nasal abnormalities, nasal mucosal damage, severe rhinitis, or other nasal conditions affecting drug administration;
Requiring nasogastric tube placement;
Organ function meeting any of the following criteria:
Absolute neutrophil count (ANC) <1.5×10⁹/L, platelets (PLT) <100×10⁹/L, hemoglobin (Hb) <90 g/L
Aspartate aminotransferase (AST) >2.5×ULN and/or alanine aminotransferase (ALT) >2.5×ULN, total bilirubin (TBIL) >1.5×ULN
Creatinine >1.5×ULN
Without anticoagulant/antiplatelet therapy: International normalized ratio (INR) >1.7 or activated partial thromboplastin time (APTT) >1.25×ULN With anticoagulant/antiplatelet therapy: INR >3.0 or APTT >1.5×ULN;
Positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with detectable HBV-DNA; or positive for hepatitis C antibody (HCVAb), Treponema pallidum antibody (TPAb/RPR), or human immunodeficiency virus antibody (HIV);
Pregnant or lactating patients;
Contraindications for MRI (e.g., metal implants such as pacemakers, claustrophobia);
Participation in any clinical trial involving investigational drugs within 3 months prior to dosing (or within 5 half-lives of last dose, whichever is longer);
Major trauma or surgery within 3 months prior to dosing, or planned surgery during the trial (excluding laparoscopy or minor procedures >4 weeks before baseline; excluding thymoma/teratoma surgery);
History of drug abuse or alcoholism within 1 year prior to dosing;
Previous treatment with stem cells or derivatives;
Any other condition that may increase patient risk or interfere with result interpretation, as determined by the investigator.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

Dose Escalation Phase: A multicenter, single-arm, open-label study will be conducted to evaluate the safety, tolerance, and dose exploration of multiple administrations of hUC-MSC-Exo for treating AE. Three dose cohorts (2.5×10¹⁰, 5.0×10¹⁰, and 1.0×10¹¹ particles) will be enrolled with 3-6 subjects each. Administration will be intranasal, once daily for 7 consecutive days, followed by once weekly for 3 consecutive weeks, resulting in a total treatment period of 4 weeks. After the last subject in each cohort completes the final dose and undergoes a 21-day safety assessment, a decision will be made regarding progression to the next higher dose cohort for further evaluation of safety and tolerance. The maximal tolerance dose (MTD) will be determined. Case Expansion Phase: A multicenter, randomized, double-blind, placebo-controlled study will enroll 20 subjects randomly assigned to either the experimental group (exosome group) or the control group (exosome mimetic group) in a 1:1 ratio. The dosage for the experimental group will be determined by the Data Safety Monitoring Board (DSMB) based on the safety and efficacy data obtained during the dose exploration phase.

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 will receive an intranasal study spray (either hUC‑MSC‑Exo at the trial dose or a placebo/mimetic) once daily for 7 days then once weekly for 3 weeks. They will have screening tests (including blood tests and infection screening) to confirm eligibility, regular safety checks and medical assessments during treatment, and follow‑up visits with clinical rating scales (mRS, CASE, ADL, MMSE, MoCA) and adverse‑event monitoring for up to 24 weeks. In the dose‑escalation portion, small cohorts have extra safety observation between dose levels; in the expansion portion participants are randomized and treated double‑blind (neither participant nor investigators know which spray is given).

How the study is structured. Two‑part interventional study. Phase 1: multicenter, single‑arm, open‑label dose escalation with three dose cohorts (2.5×10¹⁰, 5.0×10¹⁰, 1.0×10¹¹ particles), given intranasally once daily for 7 days then once weekly for 3 weeks; 3–6 subjects per cohort to assess safety and maximal tolerated dose. Phase 2a (case expansion): multicenter, randomized, parallel assignment, double‑blind, placebo‑controlled expansion (1:1) comparing the exosome dose chosen by the Data Safety Monitoring Board with an exosome mimetic placebo; masking described as quadruple. Follow‑up and outcome assessments occur through 24 weeks.

Questions you could ask

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

Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
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?
No sites are listed on the registry yet. Where will the study take place?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on autoimmune encephalitis

Clinical trials40 trials listed for autoimmune encephalitisEvery trial the registry lists for autoimmune encephalitis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in autoimmune encephalitis researchLatest monthly update: September 2026. Includes: Efgartigimod reports. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Nov 1, 2025 (estimated)
Primary completion: Jun 30, 2027 (estimated)
Study completion: Aug 31, 2027 (estimated)
Target enrollment: 38 participants (estimated)
Last updated on registry: Aug 20, 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
Principal investigator: cuilili, Principle investigator
Xuanwu Hospital, Beijing
Collaborators: ECHO Biotech

Participating sites

Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins.

Terms used on this page · explanations by MedicalUpdates.ai

phase I: An 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.
enrollment: The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
legally authorized representative: A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
informed consent form: 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.
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.
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.
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.
ULN: 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.
5 half-lives: The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
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.
Dose Escalation: Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
single-arm: All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
open-label: No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
cohort: A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
MTD: The highest dose found in a dose-escalation study that can be given without too many dose-limiting side effects.
randomized: Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
double-blind: Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
placebo-controlled: A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
control group: The group the study treatment is compared with. It may receive a placebo, an existing treatment (active comparator), a sham procedure, or no intervention.
1:1: How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.

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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