AQP4+ NMOSDPhase 1NCT07573332
A Safety and Tolerability Study of CC- 97540 (BMS-086353) in Anti-Aquaporin 4 Antibody Positive Neuromyelitis Optica Patients
A Phase 1 study testing CC-97540 (BMS-986353), Fludarabine and Cyclophosphamide in people with AQP4+ NMOSD, run by University of Texas Southwestern Medical Center at 1 site in United States.
Trial summary
The goal of this clinical trial is to find out if the investigational medicine BMS-986353 is safe and well tolerated in adults with anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD). Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- AQP4+ NMOSD
- What is being tested?
- CC-97540 (BMS-986353), Fludarabine and Cyclophosphamide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Sep 2026to 1 Aug 2031
- Phase
- 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 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Signed written informed consent
- Participants must have signed and dated an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved written ICF in accordance with regulatory, local, and institutional guidelines. This must be obtained before the performance of any protocol-related procedures that are not part of normal patient care.
- Age 18 to 60 inclusive
- Diagnosis of anti-AQP4 antibody positive NMOSD by 2015 Wingerchuk IPND criteria
- No relapses within last 12 months
- Stable ravulizumab or satralizumab dosing for at least 6 months
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- Up to date on meningococcal vaccines and enrolled in any required REMS program
- EDSS score of 6.5 or less
- At least one eye best visual acuity 20/200 or better
- Peripheral B cell count by flow cytometry of >5%
- Positive anti-AQP4 antibody test on cell based assay at screening
- Patient has adequate vascular access for leukapheresis
- ALT and AST <1.5x upper limit of normal
Who cannot join
- History of active meningococcal disease
- Presence of active, clinically significant concomitant CNS pathology, other than NMOSD, that may confound the ability to interpret study results, including but not limited to, seizure disorder, traumatic brain injuries, delirium, Parkinson's disease, psychosis, Neuro-Behcet's disease, Guillain-Barré syndrome, metabolic or infectious cause of myelopathy, genetically-inherited progressive CNS disorder, ischemic cerebrovascular disorders, including, but not limited to, transient ischemic attack, subarachnoid hemorrhage, cerebral thrombosis, cerebral embolism, or cerebral hemorrhage; CNS sarcoidosis; history of anti- myelin oligodendrocyte glycoprotein antibody-associated disorder or a diagnosis of progressive multifocal leukoencephalopathy (PML) or history of PML; or of infectious or autoimmune encephalitis or meningitis under prior DMT.
- Any significant medical condition, laboratory test abnormality or psychiatric Active infection requiring treatment
- Hypersensitivity or allergy to fludarabine, cyclophosphamide, excipients of CC- 97540, ravulizumab, tocilizumab or satralizumab.
- condition that would pose a risk the participant's safety from participating in the study.
- Active autoimmune condition other than NMOSD that requires immunotherapy
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- History of any one of the following cardiovascular conditions within the 6 months prior to screening: Class III or IV heart failure as defined by the New York Heart Association, myocardial infarction, unstable angina, angioplasty or stenting, or other clinically significant cardiac disease.
- Uncontrolled medical, psychological, familial, sociological, or geographical conditions that do not permit compliance with the protocol, as judged by the investigator; or unwillingness or inability to follow the procedures required in the protocol.
- Prior history of malignancies or lymphoproliferative disease, unless the participant has been free of the disease for ≥ 2 years. The following are allowed:
- Basal or squamous cell carcinoma of the skin.
- Carcinoma in situ of the cervix or breast.
- Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis clinical staging system) or prostate cancer that is curative.
- Other completely resected Stage I solid tumor with low risk for recurrence.
- Active syphilis, any human immunodeficiency virus, human lymphocytic T-cell virus type 1 and/or type 2, or active or latent tuberculosis infection.
- Known chronic, active hepatitis B or C virus (HBV/HCV) infection or untreated prior HCV infection (positive HCV IgG result). Participants who had HCV but have received an antiviral treatment and show no detectable HCV viral RNA for 6 months are eligible. Participants with no active hepatitis B infection (eg, hepatitis B surface antigen [HBsAg] negative, anti-hepatitis B core antibody [HBcAb]positive) who are under adequate prophylaxis against HBV re-activation may be eligible; such participants must be screened using real-time polymerase chain reaction (PCR) measurement of HBV DNA levels or viral load; those who are PCR positive will be excluded. Participants who have received HBV vaccine and are hepatitis B surface antibody (HBsAb) positive, HBcAb negative, and HBsAg negative are eligible for study entry.
- Uncontrolled or active systemic fungal, bacterial, viral, or other infection despite appropriate anti-infective treatment at the time of leukapheresis, or within 72hours before LD chemotherapy, or 5 days before CC-97540 (BMS-986353) administration.
- Use of any live vaccines against infectious disease within 6 weeks prior to the start of lymphodepleting therapy, during CC-97540 (BMS-986353) infusion, and until immune recovery.
- Previous severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection within 10 days for mild or asymptomatic infections or 20 days for severe/critical illness prior to leukapheresis or initiation of LD chemotherapy. Acute symptoms must have resolved and based on investigator assessment there are no sequelae that would place the participant at a higher risk of receiving study treatment.
- Any condition that confounds the ability to interpret data from the study.
- Participants who are not up to date on all recommended vaccinations per local/national Health Authority (eg, Centers for Disease Control and Prevention) or institutional guidelines for immunocompromised individuals. For vaccines requiring more than one dose, the full series (eg, both doses of a 2-dose series) should be completed prior to leukapheresis or initiation of LD chemotherapy when feasible and when a delay in leukapheresis or initiation of LD chemotherapy would not put the study participant at risk.
- An answer of "yes" to items 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS) at Screening (for the past 6-months interval).
- Pregnancy or nursing women
- Prior CAR T therapy
- Stem cell transplantation within one year of screening
- Positive Quantiferon test
- Prisoners or participants who are involuntarily incarcerated.
- Contraindications against MRI imaging, including non-MRI compatible metal implants, cardiac pacemakers, cochlear implants, ventricular shunt systems; metal particles in the body that pose a risk to the participant, including large tattoos in regions affected by cranial/neck MRI scanning, and severe claustrophobia. Contraindications against gadolinium-based contrast agents, including known hypersensitivity to gadolinium-based contrast agents.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
UT Southwestern
Dallas, Texas, 75390
Manuel Huichapa, 214-645-8216, Manuel.Huichapa@utsouthwestern.edu
About this study
Official title: A Multicenter, Phase 1, Safety and Tolerability Study of CC-97540 (BMS- 986353) in Anti-Aquaporin 4 Antibody Positive Neuromyelitis Optica Patients
The goal of this clinical trial is to find out if the investigational medicine BMS-986353 is safe and well tolerated in adults with anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a long-term autoimmune condition that affects the optic nerves and spinal cord and can lead to relapses. Most people with NMOSD have antibodies against AQP4, which are linked to future disease activity.
The main questions this study aims to answer are:
Read more
"-" Is CC-97540 (BMS-986353) safe and well tolerated, based on how many participants experience serious side effects that limit dosing (called dose-limiting toxicities)?
"-" Does CC-97540 (BMS-986353) show early signs of benefit, based on how many participants no longer have detectable AQP4 antibodies in their blood (called sero-reversion)?
Participants are adults aged 18 to 60 years with AQP4 antibody-positive NMOSD who are currently clinically stable on ravulizumab or satralizumab. Approved NMOSD treatments reduce relapses by changing how the immune system works, but they do not remove the cells that make AQP4 antibodies. This study is designed to see whether BMS-986353 can target these cells without the need for long-term immune suppression.
Participants will:
"-" Receive CC-97540 (BMS-986353) as part of the study "-" Continue their current NMOSD therapy "-" Attend study visits for safety checks, exams, and lab tests
- Conditions
- AQP4+ NMOSD
- Being tested
- CC-97540 (BMS-986353), Fludarabine, Cyclophosphamide, Tocilizumab
- Keywords
- anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder, AQP4+ NMOSD
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CC-97540 (BMS-986353)Biological
CD19 CAR T therapy
FludarabineDrug
Lymphodepleting Chemotherapy
CyclophosphamideDrug
Lymphodepleting Chemotherapy
TocilizumabDrug
management of CRS
| Study group | Role | What they receive |
|---|---|---|
| Participants who are AQP4 antibody-positive NMOSD treated with CC-97540 (BMS- 986353) | Experimental | Not described Receives: Biological: CC-97540 (BMS-986353); Drug: Fludarabine; Drug: Cyclophosphamide; Drug: Tocilizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety and tolerability of CC-97540 (BMS-986353) in participants with anti-AQP4 antibody positive NMO as determined by the proportion of patients experiencing DLTs
Proportion of patients experiencing DLTs at 4, 12 and 52 weeks post- infusion with CC-97540 (BMS-986353)
Measured: From infusion to 52 weeks post-infusion
To evaluate the preliminary efficacy of CC-97540 (BMS-986353) in participants with anti-AQP4 antibody positive NMO as determined by the proportion of patients who sero-revert.
Proportion of patients seronegative for the anti-AQP4 antibody at 6 months post-infusion with treatment with CC-97540
Measured: From infusion to 6 months post-infusion
Secondary outcomes (4)
Type, Frequency, and Severity of Treatment-Emergent Adverse Events
Adverse events will be assessed using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 5.0, as follows:
Grade 1: Mild adverse event Grade 2: Moderate adverse event Grade 3: Severe or medically significant but not immediately life-threatening Grade 4: Life-threatening consequences; urgent intervention indicated Grade 5: Death related to adverse event
Measured: From infusion to 52 weeks post-infusion
To evaluate the preliminary efficacy of CC-97540 in participants with anti-AQP4 antibody positive NMO
Proportion of patients relapse free at 6, 12 and 18 months after infusion with CC-97540
Measured: From treatment to 6 months post-treatment
Type, Frequency, and Severity of Treatment-Emergent Serious Adverse Events (SAEs)
Measured: From infusion to 52 weeks post-infusion
To assess the proportion of patients relapse-free following discontinuation of background therapies
To assess the duration of sero-reversion over 12 months and duration of sero- negativity over 12 months
Measured: From screening to 12 months post-infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 May 2026
- Study start1 Sep 2026 (estimated)
- Primary completion1 Aug 2030 (estimated)
- Study completion1 Aug 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AQP4+ NMOSDStudies of the same condition, open and under way.
- More trials run by University of Texas Southwestern Medical CenterThe sponsor's other registered studies.
- 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Texas Southwestern Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Bristol-Myers Squibb
- Responsible party
- Principal investigator, Benjamin Greenberg, University of Texas Southwestern Medical Center
- Investigators
- Benjamin Greenberg, MD, Principal investigator, UT Southwestern
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07573332
- Sponsor's protocol ID
- CA061-1031
- Status last verified
- Jul 2026
- First submitted
- 22 Apr 2026
- Last updated
- 17 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Phase 1, Safety and Tolerability Study of CC-97540 (BMS- 986353) in Anti-Aquaporin 4 Antibody Positive Neuromyelitis Optica Patients
- ClinicalTrials.gov ID
- NCT07573332
- Acronym / sponsor ID
- CA061-1031
- Conditions
- AQP4+ NMOSD
- Keywords
- anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder, AQP4+ NMOSD
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- University of Texas Southwestern Medical CenterUniversity, hospital or other
- Collaborators
- Bristol-Myers Squibb
- Study start
- 1 Sep 2026 (estimated)
- Primary completion
- 1 Aug 2030 (estimated)
- Study completion
- 1 Aug 2031 (estimated)
- First posted
- 7 May 2026 (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.
The goal of this clinical trial is to find out if the investigational medicine BMS-986353 is safe and well tolerated in adults with anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a long-term autoimmune condition that affects the optic nerves and spinal cord and can lead to relapses. Most people with NMOSD have antibodies against AQP4, which are linked to future disease activity.
The main questions this study aims to answer are:
"-" Is CC-97540 (BMS-986353) safe and well tolerated, based on how many participants experience serious side effects that limit dosing (called dose-limiting toxicities)?
"-" Does CC-97540 (BMS-986353) show early signs of benefit, based on how many participants no longer have detectable AQP4 antibodies in their blood (called sero-reversion)?
Participants are adults aged 18 to 60 years with AQP4 antibody-positive NMOSD who are currently clinically stable on ravulizumab or satralizumab. Approved NMOSD treatments reduce relapses by changing how the immune system works, but they do not remove the cells that make AQP4 antibodies. This study is designed to see whether BMS-986353 can target these cells without the need for long-term immune suppression.
Participants will:
"-" Receive CC-97540 (BMS-986353) as part of the study "-" Continue their current NMOSD therapy "-" Attend study visits for safety checks, exams, and lab tests
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants who are AQP4 antibody-positive NMOSD treated with CC-97540 (BMS- 986353) | Experimental | Biological: CC-97540 (BMS-986353), Drug: Fludarabine, Drug: Cyclophosphamide, Drug: Tocilizumab |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (13 listed)
- Signed written informed consent
- Age 18 to 60 inclusive
- Diagnosis of anti-AQP4 antibody positive NMOSD by 2015 Wingerchuk IPND criteria
- No relapses within last 12 months
- Stable ravulizumab or satralizumab dosing for at least 6 months
Key exclusion criteria (27 listed)
- History of active meningococcal disease
- Presence of active, clinically significant concomitant CNS pathology, other than NMOSD, that may confound the ability to interpret study results, including but not limited to, seizure disorder, traumatic brain injuries, delirium, Parkinson's disease, psychosis, Neuro-Behcet's disease, Guillain-Barré syndrome, metabolic or infectious cause of myelopathy, genetically-inherited progressive CNS disorder, ischemic cerebrovascular disorders, including, but not limited to, transient ischemic attack, subarachnoid hemorrhage, cerebral thrombosis, cerebral embolism, or cerebral hemorrhage; CNS sarcoidosis; history of anti- myelin oligodendrocyte glycoprotein antibody-associated disorder or a diagnosis of progressive multifocal leukoencephalopathy (PML) or history of PML; or of infectious or autoimmune encephalitis or meningitis under prior DMT.
- Any significant medical condition, laboratory test abnormality or psychiatric Active infection requiring treatment
- Hypersensitivity or allergy to fludarabine, cyclophosphamide, excipients of CC- 97540, ravulizumab, tocilizumab or satralizumab.
- condition that would pose a risk the participant's safety from participating in the study.
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 |
|---|---|---|
| CC-97540 (BMS-986353)CD19 CAR T therapy | Biological | Participants who are AQP4 antibody-positive NMOSD treated with CC-97540 (BMS- 986353) |
| FludarabineLymphodepleting Chemotherapy | Drug | Participants who are AQP4 antibody-positive NMOSD treated with CC-97540 (BMS- 986353) |
| CyclophosphamideLymphodepleting Chemotherapy | Drug | Participants who are AQP4 antibody-positive NMOSD treated with CC-97540 (BMS- 986353) |
| Tocilizumabmanagement of CRS | Drug | Participants who are AQP4 antibody-positive NMOSD treated with CC-97540 (BMS- 986353) |
Primary outcome
To evaluate the safety and tolerability of CC-97540 (BMS-986353) in participants with anti-AQP4 antibody positive NMO as determined by the proportion of patients experiencing DLTs
Time frame: From infusion to 52 weeks post-infusion
Proportion of patients experiencing DLTs at 4, 12 and 52 weeks post- infusion with CC-97540 (BMS-986353)
To evaluate the preliminary efficacy of CC-97540 (BMS-986353) in participants with anti-AQP4 antibody positive NMO as determined by the proportion of patients who sero-revert.
Time frame: From infusion to 6 months post-infusion
Proportion of patients seronegative for the anti-AQP4 antibody at 6 months post-infusion with treatment with CC-97540
4 secondary outcomes
Type, Frequency, and Severity of Treatment-Emergent Adverse Events
Time frame: From infusion to 52 weeks post-infusion
To evaluate the preliminary efficacy of CC-97540 in participants with anti-AQP4 antibody positive NMO
Time frame: From treatment to 6 months post-treatment
Type, Frequency, and Severity of Treatment-Emergent Serious Adverse Events (SAEs)
Time frame: From infusion to 52 weeks post-infusion
To assess the proportion of patients relapse-free following discontinuation of background therapies
Time frame: From screening to 12 months post-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 17 Jul 2026.
- Individual participant dataSharing planned: No
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A Study to Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, and Pharmacodynamics of Satralizumab in Pediatric Patients With Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD)
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Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD
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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.
Benjamin Greenberg, MD principal investigator
UT Southwestern
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CC-97540 (BMS-986353), Fludarabine and Cyclophosphamide in people with AQP4+ NMOSD, run by University of Texas Southwestern Medical Center at 1 site in United States.
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 13 inclusion and 27 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 17 Jul 2026. 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 United States. 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?
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.
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 Manuel Huichapa (214-645-8216, Manuel.Huichapa@UTSouthwestern.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the safety and tolerability of CC-97540 (BMS-986353) in participants with anti-AQP4 antibody positive NMO as determined by the proportion of patients experiencing DLTs (time frame: From infusion to 52 weeks post-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 17 Jul 2026.