Autoimmune EncephalitisPhase 1/2MESAENCT07131683
hUC-MSC-Exo Therapy for Autoimmune Encephalitis
A Phase 1/2 study testing Human umbilical cord mesenchymal stem cell derived exosomes in people with Autoimmune Encephalitis, run by Xuanwu Hospital, Beijing.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Autoimmune Encephalitis
- What is being tested?
- Human umbilical cord mesenchymal stem cell derived exosomes
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 38 participants
- Where
- No sites listed yet
- Study dates
- 1 Nov 2025to 31 Aug 2027
- Phase
- Phase 1/2
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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)
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- 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.
About this study
Official title: Human Umbilical Cord Mesenchymal Stem Cell-derived ExoSomes for Autoimmune Encephalitis: a Phase I/IIa Clinical Trial (MESAE)
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.
Read the detailed description
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.
- Conditions
- Autoimmune Encephalitis
- Being tested
- Human umbilical cord mesenchymal stem cell derived exosomes, Placebo Control
- Keywords
- mesenchymal stem cell, exosome, autoimmune encephalitis, safety, efficacy
- Medical subject headings
- Autoimmune Diseases of the Nervous System
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human umbilical cord mesenchymal stem cell derived exosomesBiological
Also called hUC-MSC-Exo
Nasal spray of hUC-MSC-Exo (low dose: 2.5×10^10 particles, mid-dose: 5.0×10^10 particles, high-dose: 1.0×10^11 particles), once per day for 7 days, then once per week for 3 weeks
Placebo ControlOther
Also called hUC-MSC-Exo mimics
Nasal spray of placebo control, once per day for 7 days, then once per week for 3 weeks
| Study group | Role | What they receive |
|---|---|---|
| Exosome group | Experimental | Not described Receives: Biological: Human umbilical cord mesenchymal stem cell derived exosomes |
| Control group | Placebo comparator | Not described Receives: Other: Placebo Control |
What is being measured
The primary outcome is the main question the study is designed to answer.
DLT events
Drug-related dose limiting toxicity (DLT)
Measured: Within 24 weeks after drug administration
mRS
modified Ranking Scale (mRS), which ranges from 0 to 6, with higher scores indicating worse outcome, and a score of 6 representing death.
Measured: 24 weeks ±10 days
Secondary outcomes (9)
AE and SAE
Adverse events and severe adverse events
Measured: Within 24 weeks after drug administration
mRS
modified Ranking Scale (mRS), which ranges from 0 to 6, with higher scores indicating worse outcome, and a score of 6 representing death.
Measured: 5 weeks (±3 days), 12 weeks (±5 days)
Percentage of mRS 0-2
Percentage of mRS 0-2. modified Ranking Scale (mRS), which ranges from 0 to 6, with higher scores indicating worse outcome, and a score of 6 representing death.
Measured: 5 weeks (±3 days), 12 weeks (±5 days), 24 weeks (±10 days)
Percentage of mRS improvement ≧ 1
Percentage of mRS improvement ≧ 1. modified Ranking Scale (mRS), which ranges from 0 to 6, with higher scores indicating worse outcome, and a score of 6 representing death.
Measured: 5 weeks (±3 days), 12 weeks (±5 days), 24 weeks (±10 days)
CASE score change compared to baseline
Clinical Assessment Scale in Autoimmune Encephalitis (CASE), ranging from 0 to 27 points, with higher score indicating more severe symptoms
Measured: 5 weeks (±3 days), 12 weeks (±5 days), 24 weeks (±10 days)
ADL score change compared to baseline
Activity of daily living (ADL) score, ranging from 0 to 100, with higher score indicating better daily living abilities
Measured: 5 weeks (±3 days), 12 weeks (±5 days), 24 weeks (±10 days)
MMSE score change compared to baseline
mini-mental state examination (MMSE), ranging from 0 to 30, with higher score indicating better cognition
Measured: 5 weeks (±3 days), 12 weeks (±5 days), 24 weeks (±10 days)
MoCA score change compared to baseline
Montreal Cognitive Assessment Scale (MoCA), ranging from 0 to 30, with higher score indicating better cognition
Measured: 5 weeks (±3 days), 12 weeks (±5 days), 24 weeks (±10 days)
Relapse rate of autoimmne encephalits
Measured: Within 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2025
- Study start1 Nov 2025 (estimated)
- Primary completion30 Jun 2027 (estimated)
- Study completion31 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autoimmune EncephalitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 38 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Xuanwu Hospital, Beijing
- Sponsor type
- University, hospital or other
- Collaborators
- ECHO Biotech
- Responsible party
- Principal investigator, cuilili, Xuanwu Hospital, Beijing
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07131683
- Sponsor's protocol ID
- KS2025069
- Other IDs
- BRWEP2024W022010110
- Acronym
- MESAE
- Status last verified
- Aug 2025
- First submitted
- 7 Aug 2025
- Last updated
- 20 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Human Umbilical Cord Mesenchymal Stem Cell-derived ExoSomes for Autoimmune Encephalitis: a Phase I/IIa Clinical Trial (MESAE)
- ClinicalTrials.gov ID
- NCT07131683
- Acronym / sponsor ID
- MESAE / KS2025069
- Conditions
- Autoimmune Encephalitis
- Keywords
- mesenchymal stem cell, exosome, autoimmune encephalitis, safety, efficacy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 38 participants (estimated)
- Lead sponsor
- Xuanwu Hospital, BeijingUniversity, hospital or other
- Collaborators
- ECHO Biotech
- Study start
- 1 Nov 2025 (estimated)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 31 Aug 2027 (estimated)
- First posted
- 20 Aug 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Exosome group | Experimental | Biological: Human umbilical cord mesenchymal stem cell derived exosomes |
| Control group | Placebo comparator | Other: Placebo Control |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (26 listed)
- 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:
- Anatomical nasal abnormalities, nasal mucosal damage, severe rhinitis, or other nasal conditions affecting drug administration;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Human umbilical cord mesenchymal stem cell derived exosomesAlso: hUC-MSC-ExoNasal spray of hUC-MSC-Exo (low dose: 2.5×10^10 particles, mid-dose: 5.0×10^10 particles, high-dose: 1.0×10^11 particles), once per day for 7 days, then once per week for 3 weeks | Biological | Exosome group |
| Placebo ControlAlso: hUC-MSC-Exo mimicsNasal spray of placebo control, once per day for 7 days, then once per week for 3 weeks | Other | Control group |
Primary outcome
DLT events
Time frame: Within 24 weeks after drug administration
Drug-related dose limiting toxicity (DLT)
mRS
Time frame: 24 weeks ±10 days
modified Ranking Scale (mRS), which ranges from 0 to 6, with higher scores indicating worse outcome, and a score of 6 representing death.
9 secondary outcomes
AE and SAE
Time frame: Within 24 weeks after drug administration
mRS
Time frame: 5 weeks (±3 days), 12 weeks (±5 days)
Percentage of mRS 0-2
Time frame: 5 weeks (±3 days), 12 weeks (±5 days), 24 weeks (±10 days)
Percentage of mRS improvement ≧ 1
Time frame: 5 weeks (±3 days), 12 weeks (±5 days), 24 weeks (±10 days)
CASE score change compared to baseline
Time frame: 5 weeks (±3 days), 12 weeks (±5 days), 24 weeks (±10 days)
ADL score change compared to baseline
Time frame: 5 weeks (±3 days), 12 weeks (±5 days), 24 weeks (±10 days)
MMSE score change compared to baseline
Time frame: 5 weeks (±3 days), 12 weeks (±5 days), 24 weeks (±10 days)
MoCA score change compared to baseline
Time frame: 5 weeks (±3 days), 12 weeks (±5 days), 24 weeks (±10 days)
Relapse rate of autoimmne encephalits
Time frame: Within 24 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Human umbilical cord mesenchymal stem cell derived exosomes in people with Autoimmune Encephalitis, run by Xuanwu Hospital, Beijing.
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 5 inclusion and 26 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 20 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
DLT events (time frame: Within 24 weeks after drug administration). The study lists 2 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 20 Aug 2025.