IMPT-514 in Systemic Lupus Erythematosus, Anca-associated Vasculitis, and Idiopathic Inflammatory Myopathy

An Exploartory Trial of IMPT-514, An Autologous Anti-CD19/20 CAR T Therapy in Participants With Active, Refractory Systemic Lupus Erythematosus, Anca-associated Vasculitis, and Idiopathic Inflammatory Myopathy

This is an exploratory trial to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of a single dose of IMPT-514, an autologous, anti-CD19/CD20 CAR T therapy, administered as an intravenous (IV) infusion, in participants with B cell driven autoimmune diseases, including active, refractory Systemic Lupus Erythematosus (SLE), ANCA Associated Vasculitis (AAV), and Idiopathic Inflammatory Myopathy (IIM).

Study Overview

• Status: Terminated
• Conditions: Systemic Lupus Erythematosus (SLE); ANCA Associated Vasculitis (AAV); Idiopathic Inflammatory Myopathy (IIM)
• Intervention / Treatment: Drug: IMPT-514 CART Cell Injection

Detailed Description

This study will explore the safety of IMPT-514 and will include up to two dose levels, with up to 12 total expected dosed participants for each disease group of SLE, AAV, and IIM.

IMPT-514 treatment consists of a single infusion of CAR-transduced autologous T cells administered intravenously after a lymphodepleting therapy regimen consisting of fludarabine and cyclophosphamide. Individual participants will remain in the active post-treatment period for approximately 2 year from IMPT-415 administration.

• Study Type: Interventional
• Enrollment (Actual): 3
• Phase: Early Phase 1

Contacts and Locations

Study Locations

• China
  • Jiangsu
    • Nanjing, Jiangsu, China, 210008
      • The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
    • Xuzhou, Jiangsu, China, 221004
      • The Affiliated Hospital of Xuzhou Medical University
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China, 200032
      • Zhongshan Hospital Fudan University.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

General Inclusion Criteria:

1. Willing and able to provide written informed consent.
2. Age 18 years of age or older and ≤75 years.
3. Weight > 35 kg at enrollment.
4. On stable background therapy (such as mycophenolate, cyclophosphamide, etc.) for autoimmune disease (SLE, AAV, IIM) with stable dose of immunosuppressants for at least 4 weeks prior to screening.

For SLE participants:

1. Diagnosis of SLE according to the 2019 European League Against Rheumatism (EULAR)/American College of Rheumatology (ACR) classification criteria or 2012 Systemic Lupus Collaborating Clinics (SLICC) criteria, including positive ANA >1:80 or positive anti-dsDNA.

For AAV participants:

1. Meets the diagnostic criteria for ANCA vasculitis in 2022 ACR/EULAR, including microscopic polyangiitis (MPA), granulomatous polyangiitis (GPA), and eosinophilic granulomatous polyangiitis (EGPA).

For IIM (Dermatomyositis, Anti-Synthetase Syndrome, and Immune-Mediated Necrotizing Myopathy) participants:

1. Diagnosis of probable or definite IIM with subgroup classification of dermatomyositis, or immune-mediated necrotizing myopathy (IMNM) subgroup of polymyositis.

Exclusion Criteria:

1. Any clinically significant underlying illness, other than systemic autoimmune disease (SLE, AAV, IIM), which would pose a safety risk or concern, as determined by the Investigator.
2. Rapidly progressive glomerulonephritis.
3. For SLE participants: Active central nervous system (CNS) lupus.
4. History of, or current significant neurologic disease.
5. History of allogeneic bone marrow or stem cell transplantation or solid organ transplantation (such as kidney, lung, heart transplant) or plans for such transplantation in future.
6. History of prior CAR T treatment, autologous or allogeneic.
7. Drug induced SLE, drug induced vasculitis or drug induced myopathy or myositis.
8. Any primary immunodeficiency.
9. History of, or current significant cardiovascular dysfunction.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Intervention; IMPT-514	Drug: IMPT-514 CART Cell Injection; CAR T-cell therapy administered intravenously after a lymphodepleting therapy regimen consisting of fludarabine and cyclophosphamide.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of dose limiting toxicities (DLTs), serious adverse events (SAE) and treatment-emergent adverse events (TEAEs).	28 days from cell infusion

Collaborators and Investigators

• Sponsor: The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School

Study record dates

Study Major Dates

• Study Start (Actual): June 13, 2024
• Primary Completion (Actual): October 21, 2025
• Study Completion (Actual): October 21, 2025

Study Registration Dates

• First Submitted: June 4, 2024
• First Submitted That Met QC Criteria: June 12, 2024
• First Posted (Actual): June 17, 2024

Study Record Updates

• Last Update Posted (Actual): January 28, 2026
• Last Update Submitted That Met QC Criteria: January 26, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Keywords: CD19/CD20 CART
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Vascular Diseases; Cardiovascular Diseases; Muscular Diseases; Neuromuscular Diseases; Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Skin Diseases; Skin Diseases, Vascular; Vasculitis; Systemic Vasculitis; Skin and Connective Tissue Diseases; Lupus Erythematosus, Systemic; Myositis; Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
• Other Study ID Numbers: RD17-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No