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

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

NCT07388277•Marcela V. Maus, M.D.,Ph.D.

View on ClinicalTrials.gov

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.

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.

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•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.
+13 more criteria

Exclusion Criteria

•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.
+6 more criteria

Locations (1)

Massachusetts General Hospital

Boston, Massachusetts, United States

Key Dates

Start Date

July 9, 2026

Primary Completion Date

December 31, 2028

Completion Date

December 31, 2029

Last Updated

February 6, 2026

Enrollment

ESTIMATED participants

Conditions

Antineutrophil Cytoplasmic Antibody-associated Vasculitis

Interventions

CC-97540

DRUG

Lymphodepletion Chemotherapy

DRUG

Contacts

Sebastian Unizony, MD

CONTACT

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