Neuromyelitis Optica Spectrum DisordersEarly Phase 1NCT06939166
Universal Chimeric Antigen Receptor T-Cell （UCAR T-cell） Therapy Targeting CD19/B Cell Maturation Antigen （CD19/BCMA） in Patients With r/r Neurological Autoimmune Diseases
A Early Phase 1 study testing UCAR T-cell in people with Neuromyelitis Optica Spectrum Disorders and Myasthenia Gravis, run by Tianjin Huanhu Hospital at 1 site in China.
Trial summary
This is an open label, single-site, dose-escalation study in up to 12 participants with relapsed or refractory Neurological Autoimmune Diseases. This study aims to evaluate the safety and efficacy of the treatment with universal CD19/BCMA CAR T-cells. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neuromyelitis Optica Spectrum Disorders, Myasthenia Gravis, Multiple Sclerosis
- What is being tested?
- UCAR T-cell
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 17 Jun 2025to Oct 2027
- Phase
- Early 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 years.
- Flow cytometry detected positive B cell CD19 or BCMA in the patient's peripheral blood.
- Subjects with relapsed or refractory neurological autoimmune diseases, Including neuromyelitis optica spectrum disorders(NMOSD), myasthenia gravis(MG), multiple sclerosis(MS)，Autoimmune encephalitis(AE) and chronic inflammatory demyelinating Polyradiculoneuropathy(CIDP).
- Female subjects of childbearing potential and male subjects with partners of childbearing potential must use medically approved contraception or abstinence during the study treatment period and for at least 6 months after the end of the study treatment; Female subjects of childbearing potential must have a negative Human chorionic gonadotropin (HCG) test within 7 days before study enrollment and not be lactating.
- Willing to participate in this clinical study, sign an informed consent form, have good compliance, and cooperate with follow-up.
Who cannot join
- Subjects with a history of severe drug allergies or allergic tendencies.
- History of malignancy within five years.
- Subjects with insufficient cardiac function.
- Subjects who are positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with peripheral blood HBV DNA >the upper limit of detection; subjects positive for hepatitis C virus (HCV) antibody and peripheral blood HCV RNA; individuals positive for human immunodeficiency virus (HIV) antibody; individuals positive for syphilis testing.
- Pregnant women or women planning to conceive.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Tianjin Huanhu Hospital
Tianjin
Jialing Wu, 86-18622271026, wywjl2009@hotmail.com
tjzge@163.com
About this study
Official title: A Clinical Study Evaluating the Safety and Preliminary Efficacy of Universal Allogeneic CAR T-cell Therapy Targeting CD19 and BCMA in Patients With Relapsed / Refractory Neurological Autoimmune Diseases
This is an open label, single-site, dose-escalation study in up to 12 participants with relapsed or refractory Neurological Autoimmune Diseases. This study aims to evaluate the safety and efficacy of the treatment with universal CD19/BCMA CAR T-cells.
Read the detailed description
This is an investigator-initiated trial to evaluate the safety and efficacy of universal CD19/BCMA CAR T-cells in Relapsed or Refractory Neurological Autoimmune Diseases.
Study intervention consists of a single infusion of universal CAR T-cells administered intravenously after a lymphodepleting therapy regimen consisting of fludarabine and cyclophosphamide.
Interim analysis will be performed when participants finish the visit 90 days after CAR T-cell infusion.
- Conditions
- Neuromyelitis Optica Spectrum Disorders, Myasthenia Gravis, Multiple Sclerosis, Chronic Inflammatory Demyelinating Polyradiculoneuropathy, Autoimmune Encephalitis
- Being tested
- UCAR T-cell
- Keywords
- Universal Allogeneic CAR T-cells
- Medical subject headings
- Neuromyelitis Optica, Myasthenia Gravis, Multiple Sclerosis, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating, 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.
UCAR T-cellDrug
Universal allogeneic anti-CD19/BCMA CAR T-cells
| Study group | Role | What they receive |
|---|---|---|
| UCAR T-cell group | Experimental | Universal allogeneic CD19/BCMA CAR T-cells Receives: Drug: UCAR T-cell |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number and severity of dose-limiting toxicity (DLT) events
DLT will be graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0, and the ASTCT Consensus Grading for Cytokine Release Syndrome and Neurologic Toxicity Associated with Immune Effector Cells.
Measured: Within 28 Days After UCAR T-cell Infusion
The total number, incidence, and severity of AEs
Measured: Up to 90 days After UCAR T-cell Infusion
Secondary outcomes (6)
NMOSD、MS: Expanded Disability Status Scale (EDSS) score
EDSS and its associated functional system (FS) score provide a system for quantifying disability and monitoring changes in the level of disability over time. EDSS is a scale for assessing neurologic impairment in multiple sclerosis (MS). It consists of 7 FS (visual FS, brainstem FS, pyramidal FS, cerebellar FS, sensory FS, bowel and bladder FS, and cerebral FS) which are used to derive EDSS score ranging from 0 (normal neurological exam) to 10 (death from MS). A negative change from baseline indicates improvement. A participant was considered to have a worsening in overall EDSS score of at least 2 if baseline EDSS score was 0, or at least 1 point if baseline EDSS score is 1 to 5, or at least 0.5 point if baseline EDSS score is 5.5 or more.
Measured: Up to 24 Months After UCAR T-cell Infusion
NMOSD、MS: Modified Rankin Scale
Modified Rankin Scale (mRS) is a profoundly valid and reliable measure of disability and is broadly utilized for assessing stroke outcomes and degree of disability. We characterized a favorable outcome as mRS ranging from zero up to two, while unfavorable outcome ranging for 3 up to 6.
Measured: Up to 24 Months After UCAR T-cell Infusion
MG: Quantitative Myasthenia Gravis Score (QMG)
The QMG score is a 13-item scale used to quantify disease severity in myasthenia gravis. The scale measures ocular, bulbar, respiratory, and limb function, grading each finding, and ranges from 0 (no myasthenic findings) to 39 (maximal myasthenic deficits).
Measured: Up to 24 Months After UCAR T-cell Infusion
MG: Myasthenia Gravis Activities if Daily Living (MG-ADL) Score
The MG-ADL is an eight-question survey of symptom severity, with each response graded from 0 (normal) to 3 (most severe). Two questions concern ocular, three oropharyngeal, one respiratory, and two extremity functions. Cumulative MG-ADL scores range from 0 to 24
Measured: Up to 24 Months After UCAR T-cell Infusion
CIDP: Inflammatory Neuropathy Cause and Treatment (INCAT) Score
The INCAT score comprises two parts, the arm score and the leg score. Based on a patient's level of impairment in their arms and legs, each part is scored between 0 and 5 points, resulting in an INCAT total score between 0 and 10.
Measured: Up to 24 Months After UCAR T-cell Infusion
AE: Change in CASE
The changes of Clinical Assessment Scale in Autoimmune Encephalitis (CASE) score from baseline.
Measured: Up to 24 Months After UCAR T-cell Infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2025
- Study start17 Jun 2025
- Primary completionApr 2026 (estimated)
- Study completionOct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromyelitis Optica Spectrum DisordersStudies 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tianjin Huanhu Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Shanghai Xiniao Biotech Co., Ltd.
- Responsible party
- Principal investigator, Jialing Wu, Tianjin Huanhu Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06939166
- Sponsor's protocol ID
- QH-HH-01
- Status last verified
- Dec 2025
- First submitted
- 6 Apr 2025
- Last updated
- 15 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Study Evaluating the Safety and Preliminary Efficacy of Universal Allogeneic CAR T-cell Therapy Targeting CD19 and BCMA in Patients With Relapsed / Refractory Neurological Autoimmune Diseases
- ClinicalTrials.gov ID
- NCT06939166
- Acronym / sponsor ID
- QH-HH-01
- Conditions
- Neuromyelitis Optica Spectrum Disorders, Myasthenia Gravis, Multiple Sclerosis, Chronic Inflammatory Demyelinating Polyradiculoneuropathy, Autoimmune Encephalitis
- Keywords
- Universal Allogeneic CAR T-cells
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Tianjin Huanhu HospitalUniversity, hospital or other
- Collaborators
- Shanghai Xiniao Biotech Co., Ltd.
- Study start
- 17 Jun 2025 (actual)
- Primary completion
- Apr 2026 (estimated)
- Study completion
- Oct 2027 (estimated)
- First posted
- 22 Apr 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 an open label, single-site, dose-escalation study in up to 12 participants with relapsed or refractory Neurological Autoimmune Diseases. This study aims to evaluate the safety and efficacy of the treatment with universal CD19/BCMA CAR T-cells.
Detailed description
This is an investigator-initiated trial to evaluate the safety and efficacy of universal CD19/BCMA CAR T-cells in Relapsed or Refractory Neurological Autoimmune Diseases.
Study intervention consists of a single infusion of universal CAR T-cells administered intravenously after a lymphodepleting therapy regimen consisting of fludarabine and cyclophosphamide.
Interim analysis will be performed when participants finish the visit 90 days after CAR T-cell infusion.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| UCAR T-cell group | Experimental | Drug: UCAR T-cellUniversal allogeneic CD19/BCMA CAR T-cells |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Age ≥ 18 years.
- Flow cytometry detected positive B cell CD19 or BCMA in the patient's peripheral blood.
- Subjects with relapsed or refractory neurological autoimmune diseases, Including neuromyelitis optica spectrum disorders(NMOSD), myasthenia gravis(MG), multiple sclerosis(MS)，Autoimmune encephalitis(AE) and chronic inflammatory demyelinating Polyradiculoneuropathy(CIDP).
- Female subjects of childbearing potential and male subjects with partners of childbearing potential must use medically approved contraception or abstinence during the study treatment period and for at least 6 months after the end of the study treatment; Female subjects of childbearing potential must have a negative Human chorionic gonadotropin (HCG) test within 7 days before study enrollment and not be lactating.
- Willing to participate in this clinical study, sign an informed consent form, have good compliance, and cooperate with follow-up.
Key exclusion criteria (5 listed)
- Subjects with a history of severe drug allergies or allergic tendencies.
- History of malignancy within five years.
- Subjects with insufficient cardiac function.
- Subjects who are positive for hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) with peripheral blood HBV DNA >the upper limit of detection; subjects positive for hepatitis C virus (HCV) antibody and peripheral blood HCV RNA; individuals positive for human immunodeficiency virus (HIV) antibody; individuals positive for syphilis testing.
- Pregnant women or women planning to conceive.
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 |
|---|---|---|
| UCAR T-cellUniversal allogeneic anti-CD19/BCMA CAR T-cells | Drug | UCAR T-cell group |
Primary outcome
The number and severity of dose-limiting toxicity (DLT) events
Time frame: Within 28 Days After UCAR T-cell Infusion
DLT will be graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0, and the ASTCT Consensus Grading for Cytokine Release Syndrome and Neurologic Toxicity Associated with Immune Effector Cells.
The total number, incidence, and severity of AEs
Time frame: Up to 90 days After UCAR T-cell Infusion
6 secondary outcomes
NMOSD、MS: Expanded Disability Status Scale (EDSS) score
Time frame: Up to 24 Months After UCAR T-cell Infusion
NMOSD、MS: Modified Rankin Scale
Time frame: Up to 24 Months After UCAR T-cell Infusion
MG: Quantitative Myasthenia Gravis Score (QMG)
Time frame: Up to 24 Months After UCAR T-cell Infusion
MG: Myasthenia Gravis Activities if Daily Living (MG-ADL) Score
Time frame: Up to 24 Months After UCAR T-cell Infusion
CIDP: Inflammatory Neuropathy Cause and Treatment (INCAT) Score
Time frame: Up to 24 Months After UCAR T-cell Infusion
AE: Change in CASE
Time frame: Up to 24 Months After UCAR T-cell Infusion
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 15 Dec 2025.
Related trials for Neuromyelitis Optica Spectrum Disorders
RecruitingObservational study
Observational Safety Study in Women With Neuromyelitis Optica Spectrum Disorder (NMOSD) Exposed to UPLIZNA® During Pregnancy
Neuromyelitis Optica Spectrum Disorder, Pregnancy Related
RecruitingPhase 2
Efgartigimod for the Treatment of Acute Optic Neuritis
Optic Neuritis
Not yet recruiting
Neurogenetics and Multi-omics of Neuroinflammatory Diseases in Multiplex Families to Improve Patients Health
Multiple Sclerosis, Neuromyelitis Optica Spectrum Disorder
RecruitingPhase 1
A Study to Assess the Safety and Clinical Activity of Azer-cel in Participants With B-cell Mediated Autoimmune Disorders
B-cell Mediated Autoimmune Disorders
Active, not recruitingObservational study
Biobank For MS And Other Demyelinating Diseases
Multiple Sclerosis, Transverse Myelitis
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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing UCAR T-cell in people with Neuromyelitis Optica Spectrum Disorders and Myasthenia Gravis, run by Tianjin Huanhu Hospital 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 5 inclusion and 5 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: looking for participants now, as of the sponsor's last update on 15 Dec 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 Jialing Wu (86-18622271026, wywjl2009@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The number and severity of dose-limiting toxicity (DLT) events (time frame: Within 28 Days After UCAR T-cell Infusion). 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 15 Dec 2025.