Antineutrophil Cytoplasmic Antibody-associated VasculitisPhase 1/2NCT07388277
CC-97540 in Patients With Antineutrophil Cytoplasmic Antibody-associated Vasculitis
A Phase 1/2 study testing CC-97540 and Lymphodepletion Chemotherapy in people with Antineutrophil Cytoplasmic Antibody-associated Vasculitis, run by Marcela V. Maus, M.D.,Ph.D. at 1 site in United States.
Trial summary
The purpose of this study is to evaluate the safety of CC-97540 in relapsed or refractory severe antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Antineutrophil Cytoplasmic Antibody-associated Vasculitis
- What is being tested?
- CC-97540 and Lymphodepletion Chemotherapy
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Mar 2026to 31 Dec 2029
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Able to voluntarily provide written informed consent prior to the performance of any study-specific procedures.
- -≥18 years of age at the time of signing informed consent.
- Classification as granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) per the American College of Rheumatology (ACR) / European Alliance of Associations for Rheumatology (EULAR) 2022 definitions (Robson et al., 2022, Suppiah et al., 2022) (see Appendix A).
- Current or historical positive proteinase 3 (PR3) or myeloperoxidase (MPO) antibody testing or a cytoplasmic (cANCA) or perinuclear antineutrophil cytoplasmic antibody (pANCA) immunofluorescence pattern. (Antibodies and immunofluorescence may currently be negative.)
- Active ANCA-associated vasculitis within 6 weeks of screening presenting as either:
- Relapsed disease (BVASv3 > 0 following prior remission) despite standard-of-care treatment per the ACR/Vasculitis Foundation (VF) Guidelines for the Management of ANCA-Associated Vasculitis (Chung et al., 2021), or
Show all 23Show fewer
- Refractory disease (persistent BVASv3 positivity) despite standard-of-care treatment per the ACR/VF Guidelines (Chung et al., 2021). Refractory disease is defined as persistent BVASv3 positivity despite at least 6 weeks of appropriate guideline-indicated standard-of-care treatment per the ACR/VF Guidelines for the Management of ANCA-Associated Vasculitis.
- Appropriate guideline-indicated standard-of-care treatment per the ACR/VF Guidelines for the Management of ANCA-Associated Vasculitis includes:
- either rituximab or cyclophosphamide for severe disease;
- or, methotrexate or azathioprine for non-severe disease.
- -Severe disease activity defined as:
- 1 or more major BVAS/WG criteria or at least 3 minor BVASv3 items (see Appendix B), or
- 1 or more of cutaneous ulceration, retroorbital disease, sinonasal disease with bony or cartilage damage, subglottic stenosis, or renal involvement, or
- Unanimous expert committee consensus on severity (3/3 agreement).
- Left ventricular ejection fraction > 45%.
- Adequate hepatic function defined by aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) <2.5 × upper limit of normal (ULN) and direct bilirubin <1.5 × ULN. Elevation in bilirubin attributable to Gilbert's syndrome is permitted.
- Adequate renal function defined by creatinine clearance >30 ml/min using the Cockcroft-Gault formula. Measured creatinine clearance from a 24-hour urine collection is acceptable if clinically indicated. For select subjects, including those with short stature or low lean muscle mass, alternative calculators to Cockcroft-Gault such as Modification in Diet Renal Disease (MDRD), Chronic Kidney Disease Epidemiology (CKD-EPI), or Cystatin C measurements may be used.
- Absolute lymphocyte count of ≥ 300 cells/uL.
- Adequate organ function defined as:
- Hemoglobin ≥ 8 g/dL
- Platelet count ≥ 75,000/uL
- Willingness to adhere to contraceptive requirements. The effects of CC-97540 CAR T cells on the developing human fetus are unknown. Lymphodepleting chemotherapy poses genotoxic, fetotoxic, and infertility risks. For these reasons, women of child-bearing potential and men with partners of childbearing potential must use effective contraception beginning prior to leukapheresis and continuing for at least 12 months after CC-97540 infusion. Due to potential unknown interactions of CC-97540 with hormonal contraception, an additional barrier method should be used if hormonal contraception is chosen. Participants who become pregnant or suspect pregnancy during the study must notify the treating physician immediately.
- Ability and willingness to adhere to the study visit schedule and all protocol requirements.
Who cannot join
- Current or historical positivity for a glomerular basement membrane antibody.
- ANCA-associated vasculitis deemed drug-induced or cocaine/levamisole-associated.
- Treatment of relapsing disease with rituximab or other B cell-depleting therapy for the current episode of relapsed vasculitis. Prior rituximab use for a prior episode (flare) of vasculitis activity is permitted.
- Parkinson's disease, epilepsy, aphasia, cerebellar disease.
- Prior organ transplant currently requiring an immunosuppressive regimen.
- Active malignancy requiring treatment other than non-metastatic basal cell or squamous cell skin carcinoma.
Show all 16Show fewer
- Treatment with any prior CAR T cell therapy.
- Significant comorbid condition or disease which in the judgment of the Principal Investigator would place the subject at undue risk or interfere with the study.
- Active, uncontrolled, systemic bacterial, viral, or fungal infection.
- Subjects with a history of class III or IV congestive heart failure or with a history of non- ischemic cardiomyopathy.
- Subjects with unstable angina, myocardial infarction, or ventricular arrhythmia requiring medication or mechanical control within the previous 3 months.
- Subjects with high bleeding risk including INR or PTT greater than 1.5 times the upper limit of normal unless due to a stable dose of anticoagulation. Ongoing anticoagulation permitted if a stable regimen.
- Absolute neutrophil count < 500 cells/uL.
- Symptomatic cerebrovascular disease or peripheral vascular arterial disease requiring ongoing therapeutic anticoagulation or dual antiplatelet therapy or other vascular disease not allowing for holding of DAPT.
- Subjects with a history of pulmonary embolism (PE) or deep vein thrombosis (DVT) within 6 months of beginning lymphodepletion requiring ongoing anticoagulation.
- Pregnant or lactating women. Pregnant women are excluded from this study because CC-97540 CAR T cell drug products are agents with the potential for adverse effects for a fetus. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with CC-97540 T cell drug products, breastfeeding should be discontinued if the mother is treated with CC-97540 CAR T cell drug product.
Trial locations and contacts
1 site in 1 country.
United States1 site
Massachusetts General Hospital
Boston, Massachusetts, 02114
About this study
Official title: A Phase I/II Study of CC-97540 (BMS-986353), CD19-Targeted NEX-T CAR T Cells, in Patients With Antineutrophil Cytoplasmic Antibody-associated Vasculitis
The purpose of this study is to evaluate the safety of CC-97540 in relapsed or refractory severe antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis.
Read the detailed description
This is a two-part, non-randomized, open label, single site Phase I/II study to assess the safety and efficacy of CC-97540 CAR T cell products for treatment of relapsing or refractory ANCA-associated vasculitis. This study consists of 2 parts: Part A (Safety Lead-In), a verification of the safety of the infusion dose, and Part B (Expansion), in which participants receive CC-97540 CAR T cells at the dose confirmed in Part A.
This is a Phase I/II clinical trial. Phase I/II clinical trials test the safety and effectiveness of an investigational therapy to learn whether the therapy works in treating a specific disease. "Investigational" means that the therapy is being studied.
The U.S. Food and Drug Administration (FDA) has not approved CC-97540 as a treatment for any disease.
It is expected that about 12 people will take part in this research study.
- Being tested
- CC-97540, Lymphodepletion Chemotherapy
- Keywords
- Refractory/Relapsing ANCA-associated vasculitis
- Medical subject headings
- Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CC-97540Drug
Also called BMS-986353, CD19-Targeted NEX-T CAR T Cells
Intravenous infusion
Lymphodepletion ChemotherapyDrug
Intravenous infusion of cyclophosphamide and fludarabine
| Study group | Role | What they receive |
|---|---|---|
| CC-97540 | Experimental | Prior to receiving CC-97540, participants will undergo lymphodepleting chemotherapy. Lymphodepleting chemotherapy is the combination of cyclophosphamide and fludarabine and will be administered intravenously. CC-97540 will be infused intravenously on day 0 only. Receives: Drug: CC-97540; Drug: Lymphodepletion Chemotherapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of CC-97540 in ANCA-Associated Vasculitis
The occurrence of DLT is the primary endpoint and will be assessed for all patients treated at the expansion dose.
Measured: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 12 months post study drug infusion.
Efficacy of CC-97540
This will be determined by the percentage of participants in treatment-free remission defined as a BVASv3 of 0 off immunosuppressive therapy.
Measured: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 12 months post study drug infusion.
Secondary outcomes (6)
Number of Adverse Events
Adverse events will be classified and graded according to the CTCAE v.5.0. This will be determined by the proportion of participants experiencing treatment-related serious AEs.
Measured: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 12 months post study drug infusion.
6 Months Treatment-Free Remission
Determined by the percentage of participants in treatment-free remission.
Measured: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 6-months following study drug infusion.
6 Months Sustained Treatment-Free Remission
Determined by the percentage of participants in sustained treatment-free remission BVASv3 of 0 without use of immunosuppressive therapy since CAR T cell infusion.
Measured: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 6-months following study drug infusion.
12 Months Sustained Treatment-Free Remission
Determined by the percentage of participants in sustained treatment-free remission at 12 months following infusion of CC-97540.
Measured: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 12 months post study drug infusion.
Severe and Non-Severe ANCA-Associated Vasculitis
Determined by the percentage of participants with severe and non-severe ANCA-associated vasculitis relapse by 6 and 12 months following infusion of CC-97540 with severe relapse defined as 1 or more new or worsened major BVASv3 item or 3 or more minor BVASv3 items and non-severe relapse as 1 or 2 new or worsened minor BVASv3 items.
Measured: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 12 months post study drug infusion.
Time to Severe or Non-Severe Relapse
Determined by the time to severe or non-severe relapse following infusion of CC-97540.
Measured: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 12 months post study drug infusion.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2026
- Study start30 Mar 2026
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Antineutrophil Cytoplasmic Antibody-associated VasculitisStudies 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
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Marcela V. Maus, M.D.,Ph.D.
- Sponsor type
- University, hospital or other
- Collaborators
- Bristol-Myers Squibb
- Responsible party
- Sponsor-investigator, Marcela V. Maus, M.D.,Ph.D., Massachusetts General Hospital
- Investigators
- Sebastian Unizony, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07388277
- Sponsor's protocol ID
- 2025P003179
- Status last verified
- Feb 2026
- First submitted
- 28 Jan 2026
- Last updated
- 10 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: \[contact information for Sponsor Investigator\]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study of CC-97540 (BMS-986353), CD19-Targeted NEX-T CAR T Cells, in Patients With Antineutrophil Cytoplasmic Antibody-associated Vasculitis
- ClinicalTrials.gov ID
- NCT07388277
- Acronym / sponsor ID
- 2025P003179
- Keywords
- Refractory/Relapsing ANCA-associated vasculitis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Marcela V. Maus, M.D.,Ph.D.University, hospital or other
- Collaborators
- Bristol-Myers Squibb
- Study start
- 30 Mar 2026 (actual)
- Primary completion
- 31 Dec 2028 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 5 Feb 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 purpose of this study is to evaluate the safety of CC-97540 in relapsed or refractory severe antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis.
Detailed description
This is a two-part, non-randomized, open label, single site Phase I/II study to assess the safety and efficacy of CC-97540 CAR T cell products for treatment of relapsing or refractory ANCA-associated vasculitis. This study consists of 2 parts: Part A (Safety Lead-In), a verification of the safety of the infusion dose, and Part B (Expansion), in which participants receive CC-97540 CAR T cells at the dose confirmed in Part A.
This is a Phase I/II clinical trial. Phase I/II clinical trials test the safety and effectiveness of an investigational therapy to learn whether the therapy works in treating a specific disease. "Investigational" means that the therapy is being studied.
The U.S. Food and Drug Administration (FDA) has not approved CC-97540 as a treatment for any disease.
It is expected that about 12 people will take part in this research study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CC-97540 | Experimental | Drug: CC-97540, Drug: Lymphodepletion ChemotherapyPrior to receiving CC-97540, participants will undergo lymphodepleting chemotherapy. Lymphodepleting chemotherapy is the combination of cyclophosphamide and fludarabine and will be administered intravenously. CC-97540 will be infused intravenously on day 0 only. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (23 listed)
- Able to voluntarily provide written informed consent prior to the performance of any study-specific procedures.
- -≥18 years of age at the time of signing informed consent.
- Classification as granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) per the American College of Rheumatology (ACR) / European Alliance of Associations for Rheumatology (EULAR) 2022 definitions (Robson et al., 2022, Suppiah et al., 2022) (see Appendix A).
- Current or historical positive proteinase 3 (PR3) or myeloperoxidase (MPO) antibody testing or a cytoplasmic (cANCA) or perinuclear antineutrophil cytoplasmic antibody (pANCA) immunofluorescence pattern. (Antibodies and immunofluorescence may currently be negative.)
- Active ANCA-associated vasculitis within 6 weeks of screening presenting as either:
Key exclusion criteria (16 listed)
- Current or historical positivity for a glomerular basement membrane antibody.
- ANCA-associated vasculitis deemed drug-induced or cocaine/levamisole-associated.
- Treatment of relapsing disease with rituximab or other B cell-depleting therapy for the current episode of relapsed vasculitis. Prior rituximab use for a prior episode (flare) of vasculitis activity is permitted.
- Parkinson's disease, epilepsy, aphasia, cerebellar disease.
- Prior organ transplant currently requiring an immunosuppressive regimen.
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-97540Also: BMS-986353, CD19-Targeted NEX-T CAR T CellsIntravenous infusion | Drug | CC-97540 |
| Lymphodepletion ChemotherapyIntravenous infusion of cyclophosphamide and fludarabine | Drug | CC-97540 |
Primary outcome
Safety of CC-97540 in ANCA-Associated Vasculitis
Time frame: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 12 months post study drug infusion.
The occurrence of DLT is the primary endpoint and will be assessed for all patients treated at the expansion dose.
Efficacy of CC-97540
Time frame: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 12 months post study drug infusion.
This will be determined by the percentage of participants in treatment-free remission defined as a BVASv3 of 0 off immunosuppressive therapy.
6 secondary outcomes
Number of Adverse Events
Time frame: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 12 months post study drug infusion.
6 Months Treatment-Free Remission
Time frame: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 6-months following study drug infusion.
6 Months Sustained Treatment-Free Remission
Time frame: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 6-months following study drug infusion.
12 Months Sustained Treatment-Free Remission
Time frame: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 12 months post study drug infusion.
Severe and Non-Severe ANCA-Associated Vasculitis
Time frame: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 12 months post study drug infusion.
Time to Severe or Non-Severe Relapse
Time frame: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 12 months post study drug 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 10 Sep 2026.
- Individual participant dataSharing planned: Yes. The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: [contact information for Sponsor Investigator]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
Related trials for Antineutrophil Cytoplasmic Antibody-associated Vasculitis
Not yet recruiting
MYTP01 for Immune-Mediated Diseases
IgA Nephropathy, Kidney Transplant Recipients
Active, not recruitingPhase 2
Safety and Efficacy of Tarperprumig in Adult Participants With Anti-Neutrophil Cytoplasmic Antibody (ANCA)-Associated Vasculitis
Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
RecruitingPhase 1/2
A Phase 1/2 Study of NKX019 in Subjects With Immune-Mediated Diseases (Ntrust-2)
Systemic Sclerosis, Idiopathic Inflammatory Myopathies
Not yet recruitingPhase 2
Trial of VIS954 in the Treatment of Antineutrophil Cytoplasmic Antibody-associated Vasculitis
Antineutrophil Cytoplasmic Antibody-associated Vasculitis
RecruitingObservational study
Studies of the Natural History, Pathogenesis, and Outcome of Idiopathic Systemic Vasculitis
Takayasu's Arteritis, Giant Cell Arteritis
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. This study is not recruiting at the moment.
Sebastian Unizony, MD principal investigator
Massachusetts General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing CC-97540 and Lymphodepletion Chemotherapy in people with Antineutrophil Cytoplasmic Antibody-associated Vasculitis, run by Marcela V. Maus, M.D.,Ph.D. 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 23 inclusion and 16 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: under way, not looking for participants, as of the sponsor's last update on 10 Sep 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 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?
Safety of CC-97540 in ANCA-Associated Vasculitis (time frame: Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 12 months post study drug 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 10 Sep 2026.