Actively Recruiting
Anti-CD19 CAR T-Cell Therapy in Refractory Systemic Autoimmune Diseases
Led by Fondazione Policlinico Universitario Agostino Gemelli IRCCS · Updated on 2025-04-11
8
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are investigating the use of anti-CD19 CAR T-cell therapy as a new treatment approach for serious systemic autoimmune diseases that are difficult to treat, including Systemic Lupus Erythematosus (SLE), Systemic Sclerosis (SSc), Dermatomyositis/Polymyositis (DM/PM), and ANCA Associated Vasculitis (AAV). These conditions involve harmful B cell activity and often do not respond well to current treatments, leading to severe health risks. This open-label, phase I basket study aims to assess the safety, feasibility, and early effectiveness of this therapy by monitoring adverse effects, infection rates, and disease response over 24 weeks in eight participants. Participants will receive a single intravenous infusion of an investigational product called CD19-CAR_Lenti, which consists of their own T cells modified to target CD19-positive B cells. The T cells are collected through leukapheresis and processed using specialized equipment before infusion. The study includes pre-treatment procedures such as lymphodepletion, with all treatments and visits conducted at specialized hospital departments. The therapy targets a common disease mechanism across the four autoimmune conditions included in this basket trial. During the study, participants will have regular evaluations to monitor for side effects like cytokine release syndrome and neurotoxicity within the first 4 weeks, as well as infections and blood cell changes over the full 24-week period. Researchers will also measure how long the CAR T cells and B cell depletion persist in the blood and track changes in disease-related antibodies. The primary outcomes include safety events and overall disease response at 24 weeks. Participants will be closely followed through outpatient visits and laboratory tests throughout the study period.
CONDITIONS
Brief Title
Anti-CD19 CAR T-Cell Therapy in Refractory Systemic Autoimmune Diseases
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Able to understand and voluntarily sign informed consent, including data protection consent
- Adults aged 18 years or older and under 65 years at consent
- Male participants must use two contraception methods unless surgically sterile
- Females of childbearing potential must have negative pregnancy test and use effective contraception
- Able to follow study visit schedule and protocol requirements
- Double vaccination against SARS-CoV-2 or recent infection within last 6 months
- Diagnosed with SLE, SSc, DM/PM, or AAV according to specific classification criteria
- Active disease with insufficient response to standard treatments as defined per disease subgroup
You will not qualify if you...
- Suitable for less burdensome or approved treatments as judged by investigator
- Low blood counts (ANC < 1000/mm3, ALC < 500/mm3, hemoglobin < 8 g/dl, CD3+ T cells ≤100/μl)
- Significant uncontrolled diseases affecting compliance or results
- Serious cardiovascular disease or reduced heart function (ejection fraction < 50%)
- Impaired kidney function (eGFR < 30 ml/min)
- Advanced lung fibrosis with poor lung function (FVC < 40% or DLCO < 30%)
- Severe active infections including HIV, hepatitis B or C, COVID-19, or active tuberculosis
- Pregnant or breastfeeding females
- Known allergies to study drugs or components
- Cancer within last 5 years except certain fully resected carcinoma in situ
- Previous CAR T cell treatment
- Recent or concurrent participation in other investigational trials
- Requirement for live vaccination during study or shortly before leukapheresis
- Under 18 years or unable to give informed consent
- Recent substance abuse affecting safety or results
- Dependency on study staff or sponsor
- For SLE patients: history of severe central nervous system involvement
- Overlapping connective tissue diseases or mixed connective tissue disease diagnosis
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single treatment day with follow-up assessments
Participants receive a single intravenous infusion of autologous anti-CD19 CAR T cells targeting their systemic autoimmune disease.
Inpatient visits during treatment and immediate recovery period
Duration - 24 weeks
Participants are monitored for safety and efficacy outcomes including incidence of cytokine release syndrome, neurotoxicity, infections, and disease response over 24 weeks.
Regular outpatient visits throughout the 24-week period
Trial Site Locations
Total: 1 location
1
Fondazione Policlinico Universitario A. Gemelli IRCCS
Rome, Lazio, Italy, 00168
Actively Recruiting
Research Team
M
Maria Antonietta D'Agostino
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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