CC-97540 in Patients With Antineutrophil Cytoplasmic Antibody-associated Vasculitis

NCT07388277 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: 2025P003179
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Antineutrophil Cytoplasmic Antibody-associated Vasculitis

Keywords

Refractory/Relapsing ANCA-associated vasculitis

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  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

  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

  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

  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

  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

  Study drug infusion (day 0) to 28 days post study drug infusion, assessed up to 12 months post study drug infusion.

Study Arms (1)

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.

Drug: CC-97540; Drug: Lymphodepletion Chemotherapy

Interventions

CC-97540 DRUG

Intravenous infusion

Also known as: BMS-986353, CD19-Targeted NEX-T CAR T Cells

CC-97540

Lymphodepletion Chemotherapy DRUG

Intravenous infusion of cyclophosphamide and fludarabine

CC-97540

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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:
• or more major BVAS/WG criteria or at least 3 minor BVASv3 items (see Appendix B), or
• 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%.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Marcela V. Maus, M.D.,Ph.D.: lead
• Bristol-Myers Squibb: collaborator

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

Condition Hierarchy (Ancestors)

Systemic Vasculitis; Vasculitis; Vascular Diseases; Cardiovascular Diseases; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Sebastian Unizony, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sebastian Unizony, MD

CONTACT

617-726-2000; SUNIZONY@mgh.harvard.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Sponsor-Investigator

Study Record Dates

• First Submitted: January 28, 2026
• First Posted: February 5, 2026
• Study Start (Estimated): July 9, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2029
• Last Updated: February 6, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Data can be shared no earlier than 1 year following the date of publication
• Access Criteria: Contact the Partners Innovations team at http://www.partners.org/innovation

Locations

US (1)