Neuromyelitis Optica Spectrum DisorderPhase 1/2NCT06561009
Safety and Efficacy of BAFFR CART for Relapsed/ Refractory Neuromyelitis Optica Spectrum Disorder
A Phase 1/2 study testing Anti-BAFFR CART in people with Neuromyelitis Optica Spectrum Disorder, run by Tianjin Medical University General Hospital.
Trial summary
This is an open-label, single-arm, dose-escalation study in up to 20 participants with relapsed/refractory Neuromyelitis Optica Spectrum Disorders (NMOSD). The aim is to evaluate the safety and efficacy of the treatment with BAFFR CART. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Neuromyelitis Optica Spectrum Disorder
- What is being tested?
- Anti-BAFFR CART
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- 1 Dec 2026to 1 Oct 2028
- 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 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Male or female subjects aged 18-60 years;
- Patients must be diagnosed as AQP4-IgG-positive NMOSD;
- At least one immunosuppressant has been used for over a year with poorly controlled symptoms;
- Clinical evidence of at least two relapses in the last 12 months or three relapses in the last 24 months and one relapse in the preceding 12 months before screening.
- Subjects and their partners must be willing to use effective and reliable methods of contraception, devices or medicines, within one year before BAFFR CART cells infusion.
- Subjects must provide written informed consent before the study begins and comply with the requirements of the study protocol.
Who cannot join
- Subjects have received B cell deletion treatment within 6 months before screening;
- Chronic and active hepatitis B (HBV), hepatitis C (HCV), Human Immunodeficiency Virus (HIV) infection, CMV or syphilis infections concurrently.
- Subjects with Papovaviruses infection.
- Subjects have received live attenuated vaccine vaccination within 8 weeks before screening; or plan to receive live vaccine vaccination within 8 weeks after treatment;
- History of psychoactive drug abuse and failed to withdraw, or have a history of psychiatric disorders.
- Pregnant or lactating women.
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- Subjects with severe heart, liver, kidney or bone marrow function disorder.
- Allergic constitution or a history of severe allergies.
- Subjects with conditions adjudicated by the investigator as unsuitable for lymphodepletion or cell infusion.
About this study
This is an open-label, single-arm, dose-escalation study in up to 20 participants with relapsed/refractory Neuromyelitis Optica Spectrum Disorders (NMOSD). The aim is to evaluate the safety and efficacy of the treatment with BAFFR CART.
- Conditions
- Neuromyelitis Optica Spectrum Disorder
- Being tested
- Anti-BAFFR CART
- Medical subject headings
- Neuromyelitis Optica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Anti-BAFFR CARTDrug
Anti-BAFFR CART
| Study group | Role | What they receive |
|---|---|---|
| Participant Group | Experimental | BAFFR CART cells Receives: Drug: Anti-BAFFR CART |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of dose-limiting toxicity (DLT)
Incidence of dose-limiting toxicity (DLT) will be evaluated within the first 28 days following BAFFR CART cells infusion.
Measured: Up to 28 days
Incidence and severity of adverse events
Adverse events will be evaluated following the chemotherapy preparative regimen and infusion of BAFFR CART cells within the first 28 days.
Measured: Up to 28 days
Secondary outcomes (8)
Proportion of BAFFR CART cells in peripheral blood
The proportion of BAFFR CART cells in peripheral blood will be detected after infusion.
Measured: Up to 1 years
Annualized relapse rate (ARR)
ARR is defined as the number of attacks divided by the total participant-years after infusion.
Measured: Up to 1 years
Changes of B cell levels in bone marrow and peripheral blood
The changes of B cell levels in bone marrow and peripheral blood after BAFFR CART infusion.
Measured: Up to 1 years
Changes of AQP4 antibody titers
Changes of AQP4 antibody titers from baseline over 1 year after BAFFR CART infusion.
Measured: Up to 1 years
Changes of cytokine in peripheral blood
Changes of cytokine(including IL-6, IFN-γ, TNF-α, and serum BAFF) in peripheral blood from baseline over 1 year after BAFFR CART infusion.
Measured: Up to 1 years
Changes of Expanded Disability Status Scale (EDSS) score
Participant will be 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 1 years
Changes of visual acuity
Corrected visual acuity is determine by Snellen E chart held at a distance of 5 meters.
Measured: Up to 1 years
Accumulated total active MRI lesions
The changes of T2 lesions and/or enhancing T1 Lesions as detected by brain Magnetic Resonance Imaging (MRI).
Measured: Up to 1 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2024
- Study start1 Dec 2026 (estimated)
- Primary completion1 Oct 2027 (estimated)
- Study completion1 Oct 2028 (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 DisorderStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tianjin Medical University General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Qiang Liu, Tianjin Medical University General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06561009
- Sponsor's protocol ID
- IRB2024-YX-220-01
- Status last verified
- Oct 2026
- First submitted
- 23 Jul 2024
- Last updated
- 16 Oct 2026
- 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 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of BAFFR CART for Relapsed/ Refractory Neuromyelitis Optica Spectrum Disorder
- ClinicalTrials.gov ID
- NCT06561009
- Acronym / sponsor ID
- IRB2024-YX-220-01
- Conditions
- Neuromyelitis Optica Spectrum Disorder
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Tianjin Medical University General HospitalUniversity, hospital or other
- Study start
- 1 Dec 2026 (estimated)
- Primary completion
- 1 Oct 2027 (estimated)
- Study completion
- 1 Oct 2028 (estimated)
- First posted
- 19 Aug 2024 (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-arm, dose-escalation study in up to 20 participants with relapsed/refractory Neuromyelitis Optica Spectrum Disorders (NMOSD). The aim is to evaluate the safety and efficacy of the treatment with BAFFR CART.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participant Group | Experimental | Drug: Anti-BAFFR CARTBAFFR CART cells |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- Male or female subjects aged 18-60 years;
- Patients must be diagnosed as AQP4-IgG-positive NMOSD;
- At least one immunosuppressant has been used for over a year with poorly controlled symptoms;
- Clinical evidence of at least two relapses in the last 12 months or three relapses in the last 24 months and one relapse in the preceding 12 months before screening.
- Subjects and their partners must be willing to use effective and reliable methods of contraception, devices or medicines, within one year before BAFFR CART cells infusion.
Key exclusion criteria (9 listed)
- Subjects have received B cell deletion treatment within 6 months before screening;
- Chronic and active hepatitis B (HBV), hepatitis C (HCV), Human Immunodeficiency Virus (HIV) infection, CMV or syphilis infections concurrently.
- Subjects with Papovaviruses infection.
- Subjects have received live attenuated vaccine vaccination within 8 weeks before screening; or plan to receive live vaccine vaccination within 8 weeks after treatment;
- History of psychoactive drug abuse and failed to withdraw, or have a history of psychiatric disorders.
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 |
|---|---|---|
| Anti-BAFFR CARTAnti-BAFFR CART | Drug | Participant Group |
Primary outcome
Incidence of dose-limiting toxicity (DLT)
Time frame: Up to 28 days
Incidence of dose-limiting toxicity (DLT) will be evaluated within the first 28 days following BAFFR CART cells infusion.
Incidence and severity of adverse events
Time frame: Up to 28 days
Adverse events will be evaluated following the chemotherapy preparative regimen and infusion of BAFFR CART cells within the first 28 days.
8 secondary outcomes
Proportion of BAFFR CART cells in peripheral blood
Time frame: Up to 1 years
Annualized relapse rate (ARR)
Time frame: Up to 1 years
Changes of B cell levels in bone marrow and peripheral blood
Time frame: Up to 1 years
Changes of AQP4 antibody titers
Time frame: Up to 1 years
Changes of cytokine in peripheral blood
Time frame: Up to 1 years
Changes of Expanded Disability Status Scale (EDSS) score
Time frame: Up to 1 years
Changes of visual acuity
Time frame: Up to 1 years
Accumulated total active MRI lesions
Time frame: Up to 1 years
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 16 Oct 2026.
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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 Anti-BAFFR CART in people with Neuromyelitis Optica Spectrum Disorder, run by Tianjin Medical University General Hospital.
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 6 inclusion and 9 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 16 Oct 2026. 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. 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?
Incidence of dose-limiting toxicity (DLT) (time frame: Up to 28 days). 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 16 Oct 2026.