Treatment for autoimmune diseases using IMPT-514

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 study is testing a new treatment called IMPT-514 to see if it can help people with autoimmune diseases like lupus and vasculitis feel better after receiving a special infusion.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 36 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School (Academic / other)
• Drugs / interventions: CAR T, cyclophosphamide, fludarabine
• Locations: 3 sites (Nanjing, Jiangsu and 2 other locations)
• Trial ID: NCT06462144 on ClinicalTrials.gov

What this trial studies

This exploratory trial evaluates the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of a single dose of IMPT-514, an autologous anti-CD19/CD20 CAR T therapy, in patients with B cell driven autoimmune diseases such as Systemic Lupus Erythematosus, ANCA Associated Vasculitis, and Idiopathic Inflammatory Myopathy. Participants will receive an intravenous infusion of IMPT-514 following a lymphodepleting therapy regimen. The study aims to include up to 12 participants from each disease group and will monitor them for approximately two years post-treatment.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 75 with active, refractory Systemic Lupus Erythematosus, ANCA Associated Vasculitis, or Idiopathic Inflammatory Myopathy.

Not a fit: Patients with mild or well-controlled autoimmune diseases may not benefit from this aggressive treatment approach.

Why it matters

Potential benefit: If successful, this treatment could provide a novel therapeutic option for patients with refractory autoimmune diseases.

How similar studies have performed: While CAR T therapies have shown promise in hematological malignancies, their application in autoimmune diseases is still novel and largely untested.

Eligibility criteria

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.

Where this trial is running

Nanjing, Jiangsu and 2 other locations

• The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School — Nanjing, Jiangsu, China (Recruiting)
• The Affiliated Hospital Of Xuzhou Medical University — Xuzhou, Jiangsu, China (Recruiting)
• Zhongshan Hospital Fudan University. — Shanghai, Shanghai, China (Not_yet_recruiting)

Study contacts

• Study coordinator: Lingyun Sun, Doctor

Conditions Systemic Lupus Erythematosus; ANCA Associated Vasculitis; Idiopathic Inflammatory Myopathy; CD19/CD20 CART